SAN DIEGO, Oct. 19, 2020 (GLOBE NEWSWIRE) -- Histogen, Inc.(NASDAQ: HSTO), a clinical-stage therapeutics company focused on developing potential first-in-class restorative therapeutics that ignite the bodys natural process to repair and maintain healthy biological function, today announced the appointment of Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations.

I am pleased to have Moya join the Histogen team at this important time as we continue to progress our pipeline candidates in the clinic, said Richard W. Pascoe, President and Chief Executive Officer of Histogen. Moya has over 30 years of experience in the biotechnology and pharmaceutical industries and most notably has extensive expertise in the cell and gene therapy field. Moreover, Moya brings an impressive track-record to Histogen as an industry leader which will complement our existing senior management team.

Ms. Daniels most recently served as Senior Vice President of GMP Quality at SanBio, where she successfully led the CMC regulatory development and GMP Quality function in support of the planned Japan commercialization of their lead product candidate. Prior to SanBio, Ms. Daniels was the Senior Vice President of Regulatory Affairs and Global Quality Assurance at Orchard Therapeutics, where she led the companys CMC regulatory and GXP quality strategy and was part of the team that led the in-licensing of GSKs rare disease gene therapy portfolio. Prior to joining Orchard, Ms. Daniels served as Vice President of Regulatory Affairs and Quality Assurance at Fate Therapeutics, Inc, where she led the development of the global regulatory strategy and quality assurance function and was interim head of Clinical Operations. Moya led development of the global regulatory strategy, quality, and global clinical operations at Osiris Therapeutics, which led to the approval of Prochymal, the first approved allogeneic cell therapy indicated for the treatment of pediatric steroid refractory acute Graft Versus Host Disease in Canada and New Zealand. Ms. Daniels also held a senior leadership position at Macrocure as Vice President of Global Regulatory Affairs and currently serves as a Scientific Advisory Board member for Indapta Therapeutics.

I am energized by Histogens leadership team and scientists and look forward to working with them to advance the companys technology through clinical development. Histogens proprietary manufacturing process that generate biological materials that stimulate a patients own stem cells to activate and regenerate tissue is truly unique, said Ms. Daniels. Histogens focus on developing potential first-in-class restorative therapeutics is perfectly aligned with my industry experience and scientific passion.

About HistogenHistogen Inc. is a clinical-stage therapeutics company focused on developing potential first-in-class restorative therapeutics that ignite the bodys natural process to repair and maintain healthy biological function. Histogens innovative technology platform utilizes cell conditioned media and extracellular matrix materials produced by hypoxia-induced multipotent cells. Histogens proprietary, reproducible manufacturing process provides targeted solutions across a broad range of therapeutic indications including hair growth, dermal rejuvenation, joint cartilage regeneration and spinal disk repair. For more information, please visit http://www.histogen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward-looking statements when we discuss our future operations and our ability to successfully initiate and complete clinical trials, obtain clinical trial data and achieve regulatory milestones and related timing; the nature, strategy and focus of our business; the sufficiency of our cash resources and ability to achieve value for our stockholders; and the development and commercial potential and potential benefits of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on our current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of ours that could differ materially from those described in or implied by the statements in this press release, including: the uncertainties associated with the clinical development and regulatory approval of our product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the potential that earlier clinical trials and studies of Histogens product candidates may not be predictive of future results; risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm our financial condition and increase its costs and expenses; the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all; and the ability to attract and retain key personnel. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including those risks discussed in our filings with the Securities and Exchange Commission. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events, or circumstances or otherwise.

CONTACT:

Susan A. KnudsonExecutive Vice President & CFOHistogen Inc.ir@histogen.com

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Histogen Appoints Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations - BioSpace

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

PLEASANTON, Calif., Oct. 6, 2020 /PRNewswire/ -- Unchained Labs, the life sciences company that's all about getting researchers the right tool for the job, launched its new gene therapy quantification application on Stunner today. Stunner is the first platform that gives researchers a full read-out on how many AAV capsids they have, how many are packed with DNA and if there is aggregation in less than a minute using only 2 L of sample.

Adeno-associated viruses (AAVs) used in gene therapies are hard to manufacture and tough to characterize. Researchers need to be able to quickly and accurately measure their capsid titer, and how many of them are full of DNA they are supposed to deliver to cells. Other methods that gather this info require labels, dyes, reagents, standard curves or use a lot of sample. Pulling it all together is a big lift and takes way too long with existing technologies.

Stunner makes AAV titer a snap. Researchers just pipette 2 L of their sample, up to 96 samples per run, and then Stunner takes it from there. UV/Vis measures the total protein and DNA in the sample. Dynamic light scattering identifies AAV capsids based on their size and checks for aggregation. Stunner then combines all this information to provide an accurate titer and empty/full ratio.

"Gene therapy researchers are hungry for better tools and Stunner delivers all the data they need," said Taegen Clary, VP of Marketing at Unchained Labs. "No other instrument quantifies AAV capsids, both full and empty, and can see aggregates. These complex molecules demand that traditional techniques be reimagined or combined to provide better insights, and with Stunner's AAV application we've done both."

About Unchained LabsHere's the deal. We're all about helping researchers break free from tools that just don't cut it. Unleashing problem-tackling products that make a huge difference in the real science they do every day. That's our mantra, our promise and we own it. We're located in Pleasanton, CA and can be found online at http://www.unchainedlabs.com.

Contact:Taegen ClaryVP of Marketing, Unchained Labs[emailprotected]925.587.9806

SOURCE Unchained Labs

http://www.unchainedlabs.com

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Unchained Labs launches the ultimate gene therapy tool; tackle AAV titer, empty/full ratio and more with Stunner! - PRNewswire

CSO C. David Pauza, Ph.D. Presents on Cell and Gene Therapy for HIV Disease

Chief Science Officer C. David Pauza, Ph.D. Presents At the Intersection of Genetic Medicine and Immunotherapy: Clinical Experience with a Cell and Gene Therapy for HIV Disease

ROCKVILLE, Md., Oct. 08, 2020 (GLOBE NEWSWIRE) -- AmericanGene Technologies(AGT)a cutting-edge cell and gene therapy company in Rockville, Maryland announced today that CEO Jeff Galvin will present at the annual Cell & Gene Meeting on the Mesa. The meeting will be held virtually October 12-16. Galvins presentation will highlight the company's technology, including AGT103-T, a therapeutic intended to cure HIV, which is scheduled to begin Phase 1 clinical trials this month.

Specifics of AGTs clinical trial can be found at https://clinicaltrials.gov/ct2/show/NCT04561258, and details of the HIV therapy intended to cure the disease are on the AGT web site.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a five-day virtual conference featuring more than 120 presentations from the leading public and private companies. These presentations will highlight the most exciting technical and clinical achievements from the past 12 months in cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies. The meeting also includes over 100 panelists and features speakers taking part in 20 in-depth sessions covering all aspects of cell and gene therapy commercialization.

The following are specific details regarding AmericanGene Technologies presentation at the conference:

Please visit http://www.meetingonthemesa.com for full information including registration. Complimentary attendance at this event is available for accredited investors and members of the media. Investors should contact Laura Stringham at lstringham@alliancerm.org and interested media should contact Kaitlyn Dupont at kdupont@alliancerm.org. The event hashtag is #CGMOM20.

About American Gene Technologies (AGT)

AmericanGene Technologies(AGT)is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies. AGTs mission is to transform peoples lives by designing highly effective therapeutics to cure infectious diseases, cancers, and inherited disorders. AGT has received three patents for the technology used to make the AGT103-T cell product and ten patents for its uniqueimmuno-oncology approachto stimulategamma-delta () T cellsto destroy a variety of solid tumors. The company has also developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT's treatment for PKU has been grantedOrphan Drug Designationby the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2021.

More information is available on:

Website - http://www.americangene.comLinkedIn - LinkedInTwitter -@americangeneFacebook -@amerigeneInstagram -@americangenetechnologies

American Gene Technologies Contacts:

C. Neil Lyons, Chief Financial OfficerPhone: (301) 337-2269Email:info@americangene.comwww.americangene.com

Sasha Whitaker, Digital Marketing and CommunicationsPhone: (301) 337-2100Email:swhitaker@americangene.comwww.americangene.com

A video is available at the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/2623868c-a33f-49c1-bc46-0c114cbd1d83

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American Gene Technologies to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

As with many conferences, the Cleveland Clinics 2020 Medical Innovation Summit went virtual this year. Still, the event featured the hotly anticipated top 10 list of innovations for 2021 that saw a range of therapies, including options for arrhythmias and gene therapy for hemoglobinopathies.

Usually, the event sees all innovations presented in a single session, but the top three were highlighted on their own.

Ranked in order of expected importance, the list was led by gene therapy for hemoglobinopathies, which are genetic disorders affecting the structure or production of the hemoglobin molecule. The most common hemoglobinopathies include sickle cell disease and thalassemia. Now, there is an experimental gene therapy that could help patients make functional hemoglobin molecules.

This is a genetic disease that affects more than 20 million people worldwide, said Rabi Hanna, department chair, pediatric hematology, oncology, & blood & marrow transplantation in highlighting the problem. Of note, sickle cell disproportionally affects the Black community. Only three medications have been approved in the past 50 years.

If adopted, this therapy could lead to a cure a very dramatic development. Hanna noted that many companies are in phase III, with one applying for approval and receiving breakthrough designation. He hopes the U.S. FDA could recognize such candidates in the next year.

That was followed by a medication for primary-progressive multiple sclerosis (MS), which affects about 15% of people with the disease. The FDA has approved a therapeutic monoclonal antibody with a novel target, namely the primary-progressive population.

For the first time we have treatment for all forms of MS, explained Adrienne Boissy, chief experience officer at the Cleveland Clinic.

Patients with this form of MS have not had a treatment in the past. This specific treatment is meant to impact the disease itself, both the MRI appearance in this disease as well as functional measures that matter for the patient, like walking and being able to maintain the life that they want to have at the fullest with this treatment.

Coming in third was smartphone-connected pacemaker devices, which are used to prevent or correct arrhythmias. These innovations aim to help in the disconnect between patients and their cardiac treatment. When paired with a mobile app, patients can gain greater insight into health data from the pacemakers. At the same time, the devices can transmit health information to physicians.

Around the world, we have millions of patients walking with pacemakers and defibrillators, and its important to know that remote monitoring of these devices is an essential part of the care of these patients, explained Khaldoun Tarakji. Much data shows that having this leads to better outcomes, perhaps through the early detection of arrhythmias. Still, adoption and adherence have remained suboptimal, he noted.

Typically, remote monitoring has taken place via bedside monitor. Patients must be in close contact with these devices. Now, a smartphone or tablet can act as that monitor, transmitting data from anywhere.

Following that innovation was a treatment for cystic fibrosis, which is caused by a defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. A class of drugs called CFTR modulators correct the proteins action, but medications developed before 2019 only were effective in a subset of people with certain mutations. However, last October, the FDA approved a new option that provides relief for patients with the most common CF gene mutation (F508 del), which is estimated to represent 90% of individuals living with the disease.

Elexacaftor/tezacaftor/ivacaftor is a fixed-dose combination medication of CFTR modulators and chloride channel openers approved for use in those patients with CF who have at least one F508del mutation in the CFTR gene.

Definitely a game changer, confirmed Elliott Dasenbrook, director, Adult Cystic Fibrosis Program, Cleveland Clinic, when asked about the class of drugs. This new class of medications the modulators help the protein channel actually, its misfolded as a result of the genetic mutations, so it helps correct that misfolding and it also helps move it throughout the cell so that it can get to the membrane, where it actually functions.

Holding the number five spot was a universal hepatitis C treatment. While this disease has serious consequences, there is no vaccine, and patients must take medication. Many treatments led to adverse side effects or only were effective for certain genotypes of the disease. However, a fixed-dose combination medication holds hope for these patients. More than 90% effective for hepatitis C genotypes one through six, the therapy represents an effective option for a wider scope of patients.

Coming in next is bubble CPAP (b-CPAP) for increased lung function in premature babies. Premature babies with infant respiratory distress syndrome (IRDS) often require ventilation. Those with IRDS commonly are administered a surfactant during mechanical ventilation, a practice that can cause lasting lung injury. B-CPAP is a noninvasive ventilation strategy, delivering continuous positive airway pressure to newborns to maintain lung volumes during exhalation.

The procedure can stimulate the lung to grow while minimizing the injury to the lung, explained Hany Aly, department chair, neonatology, Cleveland Clinic.

Unsurprisingly, telemedicine, which played a big role during the ongoing COVID-19 pandemic, made the list. Specifically, the clinic highlighted state and national regulators actions in reducing telehealth adoption barriers. Their actions permitted more widespread telehealth adoption.

COVID was a complete disruptor, and it was a disruptor that we needed, said Beri Ridgeway, associate chief of staff & chair, obstetrics, Gynecology & Women's Health Institute, Cleveland Clinic. Policy is so critical, and I believe it was one of the barriers that prevented us from taking telehealth to the next level. By taking steps on the state and federal levels in March to remove these barriers, patients benefitted. Changes came in terms of who could see patients, by what platform to include chat functions on the phone and coverage. By April, 90% of clinicians were using some sort of telehealth, it was noted.

The number eight spot was held by a vacuum-induced uterine tamponade device for postpartum hemorrhage, which affects about 1% to 5% of women who give birth. Those who experience this condition face possible blood transfusions and emergency hysterectomy. Nonsurgical interventions have involved balloon devices that expand the uterus while compressing the site of bleeding. However, a vacuum-induced uterine tamponade has caught the attention of the medical community. It is a low-tech solution that is potentially translatable to developing countries with low resource availability.

I think this is going to replace the use of the balloon compression devices for control of hemorrhage, said Edward Chien, department chair, obstetrics & gynecology, Cleveland Clinic.

In September, Alydia Health said it had received U.S. FDA clearance of the Jada system, which is designed to rapidly control and treat postpartum hemorrhage and abnormal postpartum uterine bleeding. At the same time, it announced the publication of its pivotal PEARLE study in Obstetrics & Gynecology, which demonstrated that Jada is safe and highly effective in using vacuum to control postpartum bleeding.

Holding down the ninth spot is PARP inhibitors for prostate cancer, a condition one in nine men will be diagnosed with in his lifetime. PARP inhibitors pharmacological inhibitors for cancer treatment block proteins called PARP that help repair damaged tumor DNA in individuals with BRCA1 and BRCA2 gene mutations.

The PARP inhibitors rucaparib and olaparib have been demonstrated to delay the progression of prostate cancer in men with refractory cancer and BRCA mutations. Both were approved for the prostate cancer indication in May.

While not a new treatment in cancer overall, PARP inhibitors could prove very helpful for men with this condition. And while the gynecological field has led the way, this option has been considered for years in prostate cancer, said Moshe Ornstein, associate staff, Hematology & Medical Oncology Cleveland Clinic. They will be appropriate for patients who have specific mutations in their DNA damage repair pathways.

Finally, coming in at number 10 was immunologics for migraine prophylaxis. While a host of products have tried to help this condition, nothing was directed solely for migraines. In 2018, new medications were developed for migraine sufferers that blocked the activity of a molecule called calcitonin gene-related peptide. This medication is the first to be specifically designed for the preventive treatment of migraine.

The first three medications approved for the prevention of both chronic and episodic migraines are erenumab, galcanezumab, and fremanezumab. These options were approved by the FDA in May and September 2018, respectively. They are given as once-monthly injections and are extremely well-tolerated. For most people, there are few adverse reactions aside from some pain at the injection site.

Its an exciting time to be in headache medicine. Its been years since weve had any type of new therapeutics to treat this disorder, MaryAnn Mays, director, Neurology Residency Program at the Cleveland Clinic, said.

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Smartphone-connected pacemaker devices, experimental gene therapy among top 10 innovations at annual event - BioWorld Online

FREIBURG, Germany, October 08, 2020 / B3C newswire / -- CellGenix GmbH, a pioneer for GMP grade reagents and a leading global supplier of high quality raw and ancillary materials for cell and gene therapies, announces the completion of the second phase of their facility expansion. CellGenix has reconstructed and expanded their production facility for cytokines and implemented a state-of-the art automated filling and freeze-drying line.

By implementing an automated filling and freeze-drying line and increasing bulkware production footprint, finished product capacity for cytokines is substantially increased. This continues to enable CellGenix to meet the fast-growing demand for critical raw and ancillary materials as more customers reach late-stage clinical development and commercialization of their cell and gene therapies. Scaling up from phase I or II clinical trials to large-scale commercial manufacturing, the demand for GMP cytokines often rises by several magnitudes. Consequently, a secure supply chain is imperative. A delay in delivery or change in product quality could lead to delays in production, not only increasing costs but also putting precious patient samples in jeopardy.

The newly designed and expanded facility enables CellGenix to streamline their production processes. The automated filling process also provides increased accuracy and reduced risks, which further increases the safety of their preclinical and GMP cytokines. The automated filling, stoppering and capping system (operates under an Open Restricted Area Barrier System (ORABS) and) is compliant with EU GMP Annex 1 for the manufacture of sterile medicinal products, also taking into account the new version which is currently under revision.

In a first expansion phase in 2018 CellGenix added additional space and personnel in its quality control, R&D, logistics and warehouse departments. By finishing both expansion phases, CellGenix has further strengthened their position as a leading supplier for large scale manufacturing in the cell and gene therapy space.

The expansion of our facility is a result of the robust, sustainable, and profitable growth, generated by long standing and trustful customer relationships we achieved over many years. CellGenix is now well prepared for the future growth of the cell and gene therapy market. It supports our goal to further establish CellGenix as a key provider in the fight against disease, preferred supplier or raw and ancillary materials and trusted partner for large scale manufacturing of cell and gene therapies. Felicia Rosenthal, Chief Executive Officer at CellGenix.

About CellGenixCellGenix is a leading global supplier of high quality raw and ancillary materials for the expanding market of cell and gene therapy and regenerative medicine. CellGenix develops, manufactures and markets human cytokines, growth factors, and other recombinant cell culture components in preclinical and GMP quality as well as proprietary serum-free media for further manufacturing of ATMPs. As a former ATMP developer and manufacturer, in more than two decades CellGenix gained in-depth cell processing knowledge and superior regulatory expertise. With this unique background, CellGenix understands the high requirements their customers are facing during product development and the regulatory approval process. To meet the increasing demand of GMP quality raw materials for ATMP manufacturing CellGenix has recently expanded manufacturing capacity, built additional R&D and QC laboratories, and warehouse space. The upgrade also introduced state-of-the-art, automated, large-scale capacities for recombinant protein products in the existing GMP facilities. CellGenix is headquartered in Freiburg, Germany and operates a subsidiary near Boston in Portsmouth, USA.

Contact

CellGenixFelicia M. RosenthalCEOThis email address is being protected from spambots. You need JavaScript enabled to view it. Am Flughafen 1679108 Freiburg, Germany

Keywords: Cytokines; Genetic Therapy; Cell- and Tissue-Based Therapy; Regenerative Medicine; Culture Media, Serum-Free; Indicators and Reagents; Manufactured Materials; Cell Culture Techniques; Freeze Drying

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CellGenix Completes Facility Expansion to Stay Ahead of the Increasing Demand for GMP Raw Materials - b3c newswire

The innovation experts at Cleveland Clinic have peered into their crystal ball and pronounced the most profound medical advancements that will impact the care and treatment of patients in the coming year.

During the closing session of the 2020 Cleveland Clinic Medical Innovation Summit, the 18th year the conference has been held, Cleveland Clinic Innovations shared its annual list of top 10 medical innovations. The breakthrough technologies were selected by a committee of Cleveland Clinic subject matter experts, led by Will Morris, MD, executive medical director, Cleveland Clinic Innovations, and Akhil Saklecha, MD, managing director, Cleveland Clinic Ventures.

1. Gene Therapy for Hemoglobinopathies

Experimental gene therapy gives new hope to those suffering from hemoglobinopathies, genetic disorders responsible for sickle cell disease and thalassemia. Through treatment, those with these conditions have the potential ability to make functional hemoglobin molecules, reduce the presence of sickled blood cells or ineffective red blood cells in thalassemia, and prevent associated complications.

2. Novel Drug for Primary-Progressive Multiple SclerosisA new, U.S. Food and Drug Administration (FDA)-approved therapeutic monoclonal antibody with a novel target is the first and only multiple sclerosis (MS) treatment for 15% of primary-progressive patients, who experience gradual onset and steady progression of signs and symptoms. In those with MS, the immune system attacks the fatty protective myelin sheath that covers the nerve fibers, causing communication problems between the brain and the rest of the body that can result in permanent damage or deterioration and eventual death.

3. Smartphone-Connected Pacemaker Devices

Remote monitoring of implantable pacemakers and defibrillators traditionally occurs through a bedside console that transmits data to the physician. Yet many patients don't understand how it functions, and adherence to remote monitoring has been suboptimal. New Bluetooth-enabled pacemaker devices used in conjunction with a mobile app, offer a convenient new method to transmit data, while also providing patients greater insight into their health status.

4. New Medication for Cystic Fibrosis

A new combination drug, approved by the FDA in October 2019, provides relief for patients with the most common cystic fibrosis gene mutation (F508 del), estimated to represent 90% of individuals living with the disease. Medications developed prior to last year had only been effective in a subset of people with certain mutations.

5. Universal Hepatitis C Treatment

A new, approved fixed-dose combination medication has vastly improved hepatitis C treatment. More than 90% effective for hepatitis C genotypes one through six, the therapy represents an effective option for a wider scope of patients. With no vaccine for the hepatitis C virus, patients have been limited to medication, but many treatments were accompanied by adverse side effects or only effective for certain genotypes of the disease.

6. Bubble CPAP for Increased Lung Function in Premature Babies

Newborns with infant respiratory distress syndrome now have access to a safer method of ventilation though b-CPAP, a non-invasive ventilation strategy. Unlike mechanical ventilation, which administers a surfactant that can cause lasting lung injury, b-CPAP maintains lung volumes during exhalation through oscillating, rather than constant pressure, minimizes physical trauma, and stimulates lung growth when administered over a prolonged period.

7. Increased Access to Telemedicine through Novel Practice and Policy Changes

While telehealth technologies and initiatives have been around for years, COVID-19 forced them into the spotlight and stimulated widespread adoption. "Since March, state and federal regulators have moved quickly to reduce telehealth adoption barriers, understanding that these tools can speed access to care while protecting healthcare workers and community members," according to Cleveland Clinic. "These measures opened the floodgates for telehealth, allowing for new programs and the expansion of existing networks."

8. Vacuum-Induced Uterine Tamponade Device for Postpartum Hemorrhage

A low-tech solution is now part of the arsenal to fight postpartum hemorrhage (PPH). Vacuum-induced uterine tamponade uses negative pressure created inside the uterus to collapse the bleeding cavity, causing the muscle to close off the hemorrhaging vessels. The device represents another minimally invasive tool that is potentially translatable to developing countries with minimal resources.

9. Prostate Cancer PARP Inhibitors

Previously known for their success in treating womens cancers, two PARP inhibitors, which block proteins that help repair damaged tumor DNA in people with BRCA1 and BRCA2 gene mutations, were approved for prostate cancer treatment in May. These pharmacological agents have been demonstrated to delay the progression of prostate cancer in men with refractory cancer and DNA repair pathway mutations.

10. Immunologics for Migraine Prophylaxis

New medications were developed in 2018 help head off migraine pain by blocking activity of the calcitonin gene-related peptide (CGRP) molecule, which spikes during these headaches. In 2020 this FDA-approved class of medication became the first actively prescribed therapeutic for preventative treatment of migraines, which affect an estimated 12% of the U.S. adult population.

More information about the these innovations, including videos and year-by-year comparisons is available on the website.

Mandy Roth is the innovations editor at HealthLeaders.

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Cleveland Clinic Names Top 10 Medical Innovations; Sickle Cell Therapy Tops the List - HealthLeaders Media

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).

BMN 307 is an adeno-associated virus type 5 (AAV5)-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells.

The Company recently initiated an open-label phase 1/2 study (PHEARLESS) to evaluate the safety, efficacy and tolerability of a single intravenous administration of BMN 307 in adults with PKU with PAH deficiency. The study will assess whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit biomarin.com.

BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track designation for PKU investigational gene therapy, BMN 307. https://www.prnewswire.com/news-releases/biomarin-pioneer-in-phenylketonuria-pku-and-gene-therapy-receives-fda-fast-track-designation-for-pku-investigational-gene-therapy-bmn-307-301144678.html. Accessed October 5, 2020.

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Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference

BURLINGTON, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc.(Nasdaq:FLXN) announced today thatAdam Muzikant, Ph.D., Senior Vice President, Business Development, will present at the annual 2020 Cell & Gene Virtual Meeting on the Mesa. Dr. Muzikant will provide a review of FX201, an investigational, intra-articular, IL-1Ra gene therapy product candidate in clinical development for the treatment of osteoarthritis (OA).

The Company is conducting an open-label, Phase 1 dose-escalation trial evaluating the safety and tolerability of FX201 in patients with knee OA. The trial is intended to test low, mid and high doses of FX201 in cohorts of five to eight patients. Following the completion of the low-dose cohort and data review by an independent Drug Monitoring Committee, the trial has advanced to enrolling the mid-dose cohort. Data from the study are anticipated in 2021.

Organized by theAlliance for Regenerative Medicine, the 2020 Cell & Gene Meeting on the Mesa will be delivered in a virtual format over the course of five days beginning on October 12. The conference will feature more than 120 presentations by leading public and private companies highlighting the technical and clinical achievements in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

About FX201FX201 (humantakinogene hadenovec) is a novel, clinical-stage, intra-articular gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector based on human serotype 5 (Ad5) that is designed to transfer a gene to cells in the joint to produce an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), under the control of an inflammation-sensitive promoter. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of OA. By persistently suppressing inflammation, Flexion believes FX201 holds the potential to provide long-term pain relief and functional improvement, and to modify disease progression.

About Flexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with OA, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; timing and plans with respect to the Phase 1 clinical trial of FX201; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on managements expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, the fact that the impacts and expected duration of the COVID-19 pandemic are uncertain and rapidly changing; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to FX201; risks related to clinical trials, including potential delays, safety issues or negative results; and other risks and uncertainties described in our filings with the Securities and Exchange Commission (SEC), including under the heading Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 filed with the SEC on August 5, 2020 and subsequent filings with the SEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contacts:

Scott YoungVice President, Corporate Communications & Investor RelationsFlexion Therapeutics, Inc.T: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor Relations Flexion Therapeutics, Inc.T: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics to Present at the 2020 Cell & Gene Virtual Meeting on the Mesa - GlobeNewswire

Detect Lysosomal Storage Diseases program reduces barriers to genetic diagnosis through sponsored testing for lysosomal storage disorders, including GM2 gangliosidosis

Behind the Seizure program can help accelerate genetic epilepsy diagnosis in children experiencing unprovoked seizures

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced a partnership with Invitae, a leading medical genetics company, to support Invitaes Detect Lysosomal Storage Diseases (Detect LSDs) and Behind the Seizure programs. The Detect LSDs program enables the rapid diagnosis of lysosomal storage disorders (LSDs), including GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff disease). The Behind the Seizure program is an innovative, cross-company collaboration that supports faster diagnosis for children with epilepsy. The Behind the Seizure program will also support patient identification across Tayshas broad pipeline of gene therapies for which a number of indications have an underlying seizure phenotype.

"Through both initiatives, we are supporting the rapid identification of patients with debilitating diseases, allowing them to gain access to earlier therapeutic interventions. For LSDs, there are more than 50 different disorders with overlapping symptoms, making misdiagnosis common," said RA Session II, Tayshas President, CEO and Founder. "Likewise, more than 50% of epilepsies have a genetic basis. When a patient presents with seizures, genetic testing may help identify more than 100 underlying, often rare conditions. We are proud to support these initiatives to help patients gain timely access to natural history studies, clinical trials, and ultimately disease-modifying therapies."

Eligible individuals suspected of having an LSD or epilepsy will gain access to genetic testing and counseling at no charge through these programs. The Detect LSDs program will help identify individuals who are eligible for Tayshas study evaluating TSHA-101 in patients with GM2 gangliosidosis, expected to enter the clinic later this year. The Behind the Seizure program will enable patient identification across Tayshas broad pipeline of indications, some of which have an underlying seizure phenotype, and rapid enrollment into natural history studies and clinical trials.

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"Increasing access to genetic testing can support earlier diagnosis of neurodegenerative diseases, which in turn enables clinicians to provide precision therapies sooner and better overall outcomes," said Robert Nussbaum, M.D., Chief Medical Officer of Invitae. "These unique, cross-company collaborations have been shown to help increase access to testing and reduce time to diagnosis. We are pleased Taysha has joined us in helping increase access to testing for children impacted by neurodegenerative conditions."

Additional details, as well as terms and conditions of the Detect LSDs program, can be found at https://www.invitae.com/en/detectLSDs/. To learn more about the Behind the Seizure program, please visit https://www.invitae.com/en/behindtheseizure/.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our partnership with Invitae and our planned clinical trial of TSHA-101 for the treatment of GM2 gangliosidosis, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission ("SEC") on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201006005450/en/

Contacts

Company Contact: Niren Shah, PharmD, MBATaysha Gene TherapiesNshah@tayshagtx.com

Media Contact: Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com

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Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and...

Cancer Gene Therapy Market report offers in-depth analysis of the industry size, share, major segments, and different geographic regions, forecast for the next five years, key market players, and premium industry trends. It also focuses on the key drivers, restraints, opportunities and industry challenges.

Growing prevalence of cancer with rising mortality rates will augment cancer gene therapy industry forecast in the coming years. Cancer therapies incorporated with genetically modified genes ai in blocking the growth of the tumors.

Efficient PCR and isothermal amplification technologies for detecting mutations and CRISPR gene editing tools are some technical developments. These advancements have led to innovations and ensured availability of advanced cancer gene therapies driving the industry trends.

Request for a sample copy of this report @https://www.decresearch.com/request-sample/detail/763

Estimates have stated that the global cancer gene therapy market value is likely to cross an annual valuation of USD 2.5 billion by 2025.

Technological developments are majorly fueling cancer gene therapy growth. Genetically modified genes which block tumor growth have been incorporated into recent cancer therapies. Efficient isothermal amplification techniques and PCR technology are transforming the way gene mutations are detected. Recent launch of CRISPR gene editing tools is claimed to help enhance the process of gene therapy development. These innovations and advancements in technology are anticipated to propel cancer gene therapy industry size.

The in-vivo segment is estimated to witness about 22% growth over the forecast period owing to its multiple offered benefits. In-vivo gene therapy consists of direct delivery of therapeutic genes into the target cell, a process which has shown effective results in cancer treatment. Viral vectors are delivered using in-vivo gene therapy which help in stopping the activity of tumor inducing genes and has exhibited positive results in clinical trials.

Biopharmaceutical firms held approximately 48% of the industry revenue share in 2018. Major firms such as Roche and Novartis are working on cancer gene therapies that have high adoption rate of both non-viral and viral vectors. These firms are also carrying out clinical trials that are favoring the demand for such vectors, consequently driving market growth.

Broadening awareness regarding the availability of advanced cancer therapies have stimulated the market for cancer gene therapy in China. Government initiatives and funds have encouraged researchers to carrying out extensive R&D activities linked to cancer gene therapy.

Meanwhile, on a global landscape, companies like Vigene Biosciences, Cobra, Uniqure, Sirion Biotech, Bluebird Bio, Caribou, Ziopharm, Finvector, Cellectis and Sarepta Therapeutics are leading the cancer gene therapy market. These firms are focusing on enhancing their market position through business strategies such as product launches, mergers and acquisitions, among others.

Browse full table of contents (TOC) of this report @ https://www.decresearch.com/toc/detail/cancer-gene-therapy-market

Partial Chapter of the Table of Content

Chapter 4. Cancer Gene Therapy Market, By Type

4.1. Key segment trends

4.2. Ex-vivo

4.2.1. Market size, by region, 2014 2025 (USD Million)

4.3. In-vivo

4.3.1. Market size, by region, 2014 2025 (USD Million)

Chapter 5. Cancer Gene Therapy Market, By Product

5.1. Key segment trends

5.2. Viral vectors

5.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.2. Adenoviruses

5.2.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.3. Lentiviruses

5.2.3.1. Market size, by region, 2014 2025 (USD Million)

5.2.4. Retrovirus

5.2.4.1. Market size, by region, 2014 2025 (USD Million)

5.2.5. Adeno associated virus

5.2.5.1. Market size, by region, 2014 2025 (USD Million)

5.2.6. Herpes simplex virus

5.2.6.1. Market size, by region, 2014 2025 (USD Million)

5.2.7. Vaccinia virus

5.2.7.1. Market size, by region, 2014 2025 (USD Million)

5.2.8. Others

5.2.8.1. Market size, by region, 2014 2025 (USD Million)

5.3. Non-viral vectors

5.3.1. Market size, by region, 2014 2025 (USD Million)

5.4. Others

5.4.1. Market size, by region, 2014 2025 (USD Million)

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Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch

Gosselies, Belgium, 5 October 2020, 7am CEST BONE THERAPEUTICS(Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, Link Health Pharma Co., Ltd (Link Health) and Shenzhen Pregene Biopharma Company, Ltd (Pregene) today announce the signing of an exclusive license agreement for the manufacturing, clinical development and commercialization of Bone Therapeutics allogeneic, off-the-shelf, bone cell therapy platform ALLOB in China (including Hong Kong and Macau), Taiwan, Singapore, South Korea, and Thailand.

Under the agreement, Bone Therapeutics is eligible to receive up to 55 million in development, regulatory and commercial milestone payments including 10 million in upfront and milestone payments anticipated in the next 24 months. Bone Therapeutics is also entitled to receive tiered double-digit royalties on annual net sales of ALLOB. Bone Therapeutics retains development and commercialization rights to ALLOB in all other geographies outside of those covered by this agreement. As a result, Bone Therapeutics will continue to concentrate on its development and commercialization plans for ALLOB in the US and Europe and novel innovative cell-based products globally.

This collaboration between Bone Therapeutics, Link Health and Pregene expands our geographic reach and demonstrates the global commercial potential of ALLOB,said Miguel Forte, MD, PhD, Chief Executive Officer of Bone Therapeutics. We already have operational experience in Asia with the Phase III clinical trial of our lead product JTA-004 in Hong Kong. We selected Link Health and Pregene to partner with us in Asia as a result of their expertise in advanced therapeutics and cell therapies, their proven track record of development and commercial implementation in Chinese and Asian markets, and Pregenes well established cell therapy manufacturing capacity. Bone Therapeutics will continue to develop the ALLOB cell therapy platform for other markets while exploring additional partnership opportunities in the U.S. and Europe.

The agreement grants Link Health and Pregene exclusive rights to clinically develop and commercialize ALLOB for the treatment of human bone disorders in Greater China, Taiwan, Singapore, South Korea, and Thailand. All rights for China will be transferred to Pregene and Link Health will gain rights for the remaining countries Bone Therapeutics will share its patented proprietary manufacturing expertise for the expansion and differentiation of bone-forming cells and has the option to sell clinical supplies to Link Health and Pregene in preparation for their clinical development of ALLOB.

This collaboration and license agreement for Bone Therapeutics ALLOB provides a strong addition to our pipeline. ALLOB has demonstrated the potential to reduce the recovery time and stimulate bone growth for a variety of bone conditions, and to have a considerable impact on patients lives,said Yan Song, PhD, Chief Executive Officer of Link Health. It is important for Link Health to collaborate with companies that have strong therapeutic product portfolios and entrepreneurial management. This partnership with Bone Therapeutics is a direct result of our shared commitment to appreciate the enormous potential of cell therapy and regenerative medicine.

Pregene now has a flourishing portfolio of CAR-T cell therapy-based cancer treatments. Bone Therapeutics ALLOB provides anallogeneic, off-the-shelf cell therapy that expands our portfolio of cell therapies to include the sizable commercial potential of orthopedics,said Hongjian Li, Co-founder and Chief Executive Officer of Pregene. We expect to be able to leverage our extensive international cell and gene therapy experience to develop Bone Therapeutics ALLOB platform and subsequently launch products in China and Southeast Asian markets.

ALLOB, an allogeneic and off-the-shelf cell therapy product manufactured through a proprietary, scalable production process, consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors. In preclinical studies ALLOB has shown to reduce healing time in a delayed-union fracture model by half, and has demonstrated good tolerability and signs of efficacy in two Phase IIa studies for two separate indications. The Companys randomized, placebo-controlled, double-blind Phase IIb clinical trial in patients with difficult tibial fractures has received approval from regulatory authorities in six of the seven planned European countries to date, and is expected to enroll the first patient later this year.

About Link Health Pharma Co., Ltd

Link Health is a leading Chinese pharmaceutical company based in Guangzhou, Southern China, focusing on the development of innovative drugs for unmet medical needs.

Link Health has created a highly professional team with diverse expertise in drug development, medical affairs and regulatory affairs. Leveraging deep understanding of China market, regulatory environment and strong network with global biopharmaceutical companies, Link Health is well positioned to bring innovative drugs to the market efficiently. The company has a drug development pipeline of 5 clinical stage assets and 1 under NDA reviewing in China.

The company has also established a fully owned subsidiary in Amsterdam, the Netherlands. The Dutch office builds and further strengthen collaborations with global pharma/biotech partners and research institutes.

About Pregene Biopharma Co., Ltd

Shenzhen Pregene Biopharma Co. Ltd is a leading enterprise in the cell and gene therapy field with the core technology for industrialization. The companys core team comes from well-known institutions and companies including the Academy of Military Medical Sciences, the University of Toronto, and the US FDA.

Pregene has established the gene editing platform, viral vector and cell production platform, nanobody selection platform and other small to pilot trial manufacturing system, with total investment over 100 million CNY. It has the laboratories and GMP plants for cell and gene therapy of over 10,000 square meter.

The company focuses on the research and development of cell and gene therapy drugs, and participated in the drafting the national standard Considerations for CAR-T Cell Quality Study and Non-clinical Evaluation issued by the National Institutes for Food and Drug Control in June 2018. The CAR-T cell therapy for the treatment of multiple myeloma have obtained NMPA IND clearance as the Class I new drug, which is the first in China and fastest in the world using the humanized single domain antibody in CAR construct, and phase I clinical trials are now in progress. Other pipelines such as CAR-T, TCR-T and mRNA drugs for tumors, autoimmune diseases and other indications are in the development at different stages. The company has broad development prospects with the abundant backup technologies.

Looking forward to the future, the company will build the core capacity in one-stop solution for cell and gene therapy drugs, and fulfill the Express of innovative medicine development from drug discovery to clinical products.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company is ready to start the phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available atwww.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

For US Media and Investor Enquiries:LHA Investor RelationsYvonne BriggsTel: +1 310 691 7100ybriggs@lhai.com

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -...

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinsons Disease R&D Day on Friday, October 30.

Information on the upcoming presentations can be found below:

Chardan 4th Annual Genetic Medicines Conference

2020 ARM Virtual Cell and Gene Meeting on the Mesa

AXO-Lenti-PD Parkinsons Disease R&D Day

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com.Replays will be available for approximately 30 days following the conferences.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - GlobeNewswire

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).

PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.

The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit pfizer.com.

Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https://www.businesswire.com/news/home/20201001005382/en/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy. Accessed October 2, 2020.

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Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced that it will be presenting at the Alliance for Regenerative Medicines (ARM) virtual Cell and Gene Meeting on the Mesa, taking place October 12-16, 2020. Michael Redman, Executive Vice President and Chief Operating Officer of Genprex, will lead the companys presentation.

The 2020 Cell and Gene Meeting on the Mesa will be delivered in a virtual format over the course of five days where attendees will be able to watch company presentations on-demand, in addition to two live-streaming panels each day. The Cell and Gene Meeting on the Mesa is the sectors foremost annual conference, bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies.

For more information on the conference, or to register, please visit https://www.meetingonthemesa.com.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, GPX-001 (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). GPX-001 has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. GPX-001 has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for GPX-001 for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of GPX-001, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of GPX-001 and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates and whether patents will ever be issued under patent applications that are the subject of our license agreements. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex to Present at the Alliance for Regenerative Medicine's Virtual Cell and Gene Meeting on the Mesa - Business Wire

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa. The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com.

Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery. That discussion will be available to view on demand on the conference website starting Oct. 13.

About Generation Bio

Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.For more information, please visitgenerationbio.com.

Contact:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

Gyros Protein Technologies AB has announced the introduction of Gyrolab AAVX Titer Kit for adeno-associated virus (AAV) vector titer determination in AAV vector manufacturing. The new Gyrolab AAVX Titer Kit for physical titer determination, one of the critical quality attributes (CQAs) of the FDA process validation guidelines, has a broad applicability across numerous AAV serotypes and adds to the Companys wide range of ready-to-use kits that are already used by scientists and bioengineers in the rapidly growing cell and gene therapy market, where AAV vectors are commonly used.

Gyrolab AAVX Titer Kit supports scientists in the development and production of cell and gene therapies to maximize performance and productivity, and reduces the time needed to produce patient treatments. With compressed development timelines for these therapies, the new kit generates results 4 times faster and requires 10 times less sample compared to traditional ELISA methods, accelerating AAV vector workflows and enabling high quality data to be produced from small sample volumes. Featuring an analytical range of >3 logs, Gyrolab AAVX Titer Kit also minimizes repeat testing compared to ELISA.

Gyrolab AAVX Titer Kit is based on a highly selective AAVX affinity ligand developed with the Thermo Scientific CaptureSelect technology by Thermo Fisher Scientific. These ligands are also the basis of POROS CaptureSelect AAVX Affinity Resin, which is frequently used to purify AAV viral vectors. The AAVX ligand binds with high affinity and selectivity to native and recombinant AAV particles of various serotypes (AAV1 to AAV8, AAVrh10).

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Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced the appointment of its executive leadership team. This group has significant experience in gene therapy drug development and commercialization, and will enable Taysha to build the corporate culture and infrastructure necessary to advance its extensive pipeline of 18 gene therapy programs, with exclusive options to acquire four additional programs from UT Southwestern Gene Therapy Program. In addition, Sukumar Nagendran, M.D., former Chief Medical Officer of AveXis, and Phillip Donenberg, former Chief Financial Officer of AveXis, have joined the companys Board of Directors. Mr. Donenberg will also serve as the companys Audit Committee Chairman.

Joining the Taysha Board is a unique opportunity to contribute to scientific advancements in CNS gene therapy, said Sukumar Nagendran, M.D., Taysha Board of Directors. In partnership with UT Southwestern, Taysha has built an extensive pipeline of gene therapy candidates for life-threatening CNS diseases with significant unmet medical need.

It is a distinct pleasure to be reunited with many of my former AveXis colleagues that enabled the development and successful commercialization of Zolgensma, said Phillip Donenberg, Taysha Board of Directors. I am excited to contribute to Tayshas efforts to deliver therapies with the potential to improve the lives of patients with devastating CNS disease.

Each member of the Taysha leadership team has significant gene therapy expertise, with an unrelenting, patient-first focus guiding their individual areas of focus. Joining RA Session II, Founder, President and CEO of Taysha, on the management team are the following individuals:

From day one, we set out to build a team that has the passion, experience and talent to achieve our mission of eradicating monogenic CNS disease. Today, we are proud to announce a highly experienced team of CNS gene therapy experts, said Mr. Session. We are also pleased Dr. Nagendran and Mr. Donenberg have joined our Board and will contribute their several years of gene therapy expertise. Their experience in building AveXis will be invaluable as we continue to grow and advance several programs into the clinic.

About Taysha Gene Therapies

Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our management teams proven experience in gene therapy drug development and commercialization with UT Southwesterns world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies to dramatically improve patients lives. More information is available at http://www.tayshagtx.com.

Excerpt from:

Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and...

Brazil has become the first among the Latin American countries to approve the marketing of gene therapy products. Specifically, the Brazilian Health Regulatory Agency (ANVISA) recently granted marketing authorization for Novartis gene therapy products Luxturna and Zolgensma. As published in the Brazilian Federal Register, Luxturna received its marketing authorization on August 6th and a few days later, on August 17th, Zolgensma received its marketing authorization. Prior to ANVISAs final decision, both products had been approved by the Brazilian Technical Commission of Biosafety (CTNBio), which is responsible for evaluating biosafety of Genetically Modified Organisms (GMOs) in Brazil.

Luxturna

Luxturna (voretigene neparvovec) is indicated for the treatment of adult and pediatric (children over 12 months) patients with vision loss caused by bi-allelic retinal pigment epithelium (RPE) mutation-associated hereditary retinal dystrophies. In Brazil, no other therapeutic option available for the treatment of this rare disease until approval of Novartis gene therapy.

It took 232 business days from the submission of the marketing authorization application for ANVISA to provide its decision. As part of the administrative approval procedure, ANVISA and Novartis signed a Term of Commitment to ensure that the company will conduct additional trials with Brazilian patients to continue to monitor the safety and effectiveness of Luxturna on a long-term basis.

Zolgensma

Zolgensma (onasemnogene abeparvovec) is indicated for the treatment of pediatric patients less than 2 years of age with type 1 spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron (SMN1) gene and with bi-allelic mutations in the SMN1 gene and up to 3 copies of the SMN2 gene.

After 149 business days of analysis, ANVISA concluded that the products benefits surpassed its risks and granted the marketing authorization. However, because it is a new gene therapy, the authorization was granted on a exceptional basis, meaning that Novartis is required to send data on Zolgensmas ongoing trials as agreed to in the signed Term of Commitment.

Novartis also commited to conduct additional studies with Brazilian patients to enable the evaluation of whether Zolgensma maintains a positive benefit-risk balance. Additionally, Novartis also agreed to train (i) healthcare professionals who will perform infusions of the drug; and (ii) Brazilian hospitals in the appropriate storage and handling of Zolgensma.

Regulatory framework and next steps

The Brazilian regulatory framework on advanced therapeutic products is very new. At the end of 2018, ANVISAs Collegiate Board of Directors approved Rule #260/2018, which defines the technical standards for conducting clinical trials with experimental advanced therapy products. On February 26, 2020, ANVISAs Rule #338/2020 was published which provides the requirements for obtaining a marketing authorization for advanced therapy products. The regulation classifies advanced therapy products into three different categories: (i) advanced cell therapy; (ii) gene therapy; and (iii) tissue engineering. Both Luxturna and Zolgensma fall into the second category, gene therapy.

The next step for Novartis to introduce Luxturna and Zolgensma into the Brazilian market involves receiving price approval from the Brazilian Drugs Market Regulation Chamber (CMED). This approval is expected by the end of 2020. There is much anticipation regarding the pricing of these drugs, particularly since Zolgensma is currently considered to be the worlds most expensive drug (at USD $2.125 million).

The approval of the first gene therapy products in Brazil also brings up the issue of whether these drugs will become available in the Public Healthcare System (SUS). The analysis of whether a drug is incorporated into the SUS is conducted by the National Commission for Incorporation of Health Technologies (CONITEC), which issues a recommendation to the Ministry of Health. While Luxturna and Zolgensma are currently not in the SUS, the Brazilian population can obtain free access to these products through a court order.

Although there are still some a few steps to be taken until Luxturna and Zolgensma truly get into the hands of patients in Brazil, the approval itself and the consolidation of a regulatory framework on advanced therapeutic products is expected to promote the development of this pharmaceutical in Brazil.

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First Gene Therapy Products Approved in Brazil - Lexology

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - The News Brok