Category Archives: Gene Medicine

DUBLIN--(BUSINESS WIRE)--The "The Market for Cell and Gene Therapy Manufacturing - The Rise of CMOs & CDMOs (2021)" report has been added to ResearchAndMarkets.com's offering.

Funding the Cell and Gene Therapy Boom

The rapid ascension of cell and gene therapies (CGT) has created a crucial turning point in the history of modern medicine. Not only have new therapies such as CAR-T produced a shift towards highly individualized medicine and a powerful new front in the war against cancer, but the rapidity of change with the CGT sector has taken the world by storm.

By 2025, the FDA has indicated that it will be reviewing an estimated 10 to 20 of CGTs per year, while the European Medicines Agency (EMA), has estimated that it will soon be approving as many as a dozen such therapies per year.

With the hard-earned marketing approvals gained by cell and gene therapy companies in recent years, biopharmaceutical companies have substantially increased their interest in the CGT sector. Some biopharma companies are resorting to increasing their manufacturing capacity by investing in expansion and new facilities, while others are partnering with third-party contract manufacturing organizations to outsource this work.

With the future in mind, contract manufacturing organizations (CMOs) and contract development and manufacturing organizations (CDMOs) are also investing heavily in expanding their manufacturing capacity to be able to take on new clients and larger, later-stage projects as the market expands.

Currently, the cell and gene therapy field is currently on track for a massive capacity shortage. In particular, there is likely to be a shortage of manufacturing capacity at the commercial scale. Although approximately 90% of cell and gene therapy developers would prefer to use CMOs, related CMO capacity is not available within the industry.

Moreover, the lead time for CMOs to begin cell and gene therapy projects averages over 18 months. Thus, CGT developers are often forced to expand their in-house capacity. Of course, CMOs are also investing heavily into the expansion of their cell and gene therapy manufacturing capabilities, but these large build-outs take time.

This report is tailed to the strategic needs of cell and gene therapy (CGT) companies, as well as the CMOs and CDMOs who are handling third-party manufacturing services for these clients. With the competitive nature of this global market, you don't have the time to do the research. Claim this report to become immediately informed, without sacrificing hours of unnecessary research or missing critical opportunities.

In compiling this report, the research analysts leveraged nearly a decade of historical data on the cell and gene therapy manufacturing industry. In addition to conducting extensive secondary research, our analysts interviewed dozens of highly regarded industry leaders.

Key Topics Covered:

1. Funding the Cell and Gene Therapy Boom

1.1 Recent CMO/CDMO Expansions in Cell and Gene Therapy Sector

1.1.1 Expected Capacity Crunch

1.1.2 Investments for CGT Capacity Expansion

1.1.3 Other CGT Manufacturing Investments

1.1.4 Events Driving Expansion for CGT Facilities and Infrastructure

2. Recent Merger and Acquisition (M&A) Deals within the Cell and Gene Therapy Sector

2.1 Charles River Laboratories International/Retrogenix

2.2 Charles River Laboratories International/Cognate BioServices

2.3 Beam Therapeutics/Guide Therapeutics

2.4 Thermo Fisher Scientific/Henogen

2.5 Eli Lilly/Prevail Therapeutics

2.6 Novartis/Vedere Bio

2.7 Bayer/Asklepios

2.8 AGC Biologics/MolMed

2.9 Sorrento Therapeutics/SmartPharm

2.10 Catalent/MaSTherCell

2.11 ElevateBio

2.12 Ixaka

3. Financing Rounds

3.1 Major Private Placements

3.1.1 Orchard Therapeutics

3.1.2 Amryt

3.1.3 BioNTech

3.1.4 Kiadis Pharma

3.2 Major Venture Capital Financings

3.2.1 ElevateBio

3.2.2 Century Therapeutics

3.2.3 Artiva Biotherapeutics, Inc.

3.2.4 Vineti, Inc.

3.2.5 Ori Biotech Ltd.

3.2.6 Neogene Therapeutics

3.2.7 Forge Biologics

3.2.8 Sana Biotechnology

3.2.9 Orca Bio

3.2.10 Freeline Therapeutics

3.2.11 Poseida Therapeutics

3.2.12 Kriya Therapeutics

3.2.13 Legend Biotech Corporation

3.2.14 Lyell Immunopharma

3.2.15 Mammoth Biosciences

3.2.16 Kyverna Therapeutics

3.3 Major Corporate Partnerships within the CGT Market

3.3.1 Bayer/Atara Biotherapeutics

3.3.2 Novartis/Mesoblast

3.3.3 Novartis/Sangamo

3.3.4 Janssen Biotech/Fate Therapeutics

3.3.5 Biogen/Sangamo

3.4 Follow-on Public Offerings by CGT Companies

3.4.1 Rocket Pharmaceuticals

3.4.2 Adaptimmune Therapeutics

3.4.3 Allogene Therapeutics

3.4.4 Iovance Biotherapeutics, Inc.

3.4.5 Bluebird bio

3.5 Initial Public Offerings (IPO)

3.5.1 Legend Biotech

3.5.2 Decibel Therapeutics

3.5.3 Sana Biotechnology

3.5.4 Rocket Pharmaceuticals

3.5.5 JW Therapeutics

3.5.6 AlloVir

3.5.7 Akouos

3.5.8 Generation Bio

3.5.9 Passage Bio

3.5.10 Beam Therapeutics

4. Market Size for Cell and Gene Therapy CMO/CDMOs

5. Profiles of Cell and Gene Therapy CMOs and CDMOs

5.1 3P Biopharmaceuticals

5.2 ABL, Inc.

5.3 AGC Biologics

5.4 Advent BioServices Ltd.

5.5 Akron Biotech

5.6 Aldevron

5.7 Anemocyte S.r.l

5.8 Applied Viromics

5.9 ATVIO Biotech, Ltd./Orgenesis Biotech Israel, Ltd.

5.10 Austrianova

5.11 Avid Bioservices, Inc.

5.12 Batavia Biosciences B.V.

5.13 Bio Elpida

5.14 BioCentriq

5.15 BioNTech IMFS GmbH

5.16 BioReliance Corporation/Merck Millipore

5.17 Bio-Techne

5.18 Biovian Oy

5.19 Boehringer Ingelheim BioXcellence

5.20 Brammer Bio/Thermo Fisher Scientific

5.21 C3i

5.22 Catalent Biologics

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World Cell and Gene Therapy Manufacturing Market Report 2021: The Rise of CMOs & CDMOs - Funding the Cell and Gene Therapy Boom -...

On the surface, it may seem that the higher the price of a new medicine, the harder it is for its developer to get a thumbs-up from drug-price watchdog Institute for Clinical and Economic Review (ICER). For companies in the rapidly growing gene and cell therapy field, that would make ICERs review a tough hurdle to overcome, particularly if the agencys opinions take on more weight in determining insurance coverage decisions in the future.

But analysts at Mizuho Securities dug into most of ICERs verdicts on gene and cell therapies to date and found a surprising trend: The institutes reviewers have deemed more than half the products they reviewed so far to be cost-effective. And the evidence they used to reach those positive conclusions offers important lessons on research, development and pricing to developers of the next generation of gene and cell therapies, said analyst Difei Yang, Ph.D., the lead author of two reports Mizuho sent to clients last week, in an interview.

Clearly, theyre looking at the amount of time the patient gainsbut also the quality of life, Yang said. Companies that put in the effort to provide robust data proving a gene or cell therapy could outperform the standard of care on both measureshead-to-head trials, solid evidence of long-term benefit and so forthhad a higher likelihood of a positive ICER verdict on cost-effectiveness, Mizuho found.

Makers of CAR-T cancer cell therapies have so far been much more successful at proving superiority over the standard of care than have manufacturers of gene therapies for rare diseases, Mizuho noted. ICER approved of the cost-effectiveness of the first two CAR-Ts on the market, Gileads Yescarta for lymphoma and Novartis Kymriah for lymphoma and leukemia, both approved in 2017 and launched at $373,000 and $475,000, respectively. And the agency has already said it will give the thumbs-up to the yet-to-be-approved multiple myeloma CAR-T cilta-cel from Janssen and Legend Biotechprovided that it is priced at $475,000 or less.

Only one of four CAR-Ts that ICER reviewed did not hit the cost-effectiveness threshold: Bristol Myers Squibbs ide-cel for multiple myeloma, which was launched this spring with the brand name Abecma at a price of $419,500. A spokesperson for BMS griped at the time that ICER relied on inappropriate modeling of ide-cels overall survival data and a misguided view of retreatment.

ICERs president Steve Pearson, M.D.,cited important evidence gaps in the data available to the agency while it was reviewing the cost-effectiveness of multiple myeloma treatments.

Pearson said in an interview that ICERs reviewers sometimes struggle to determine how data from what are often brief studies will translate to real-world outcomes. We need to see longer-term data that we can connect to what were seeing in the short term, Pearson said. What we really want to see are patient-relevant outcomes, like how well theyre functioning and their quality of life.

Often, companies fall short when it comes to providing data that provide a clear contrast between patients who receive gene or cell therapies and those who do not, Pearson said. They may say [non-treated] patients would only have six months to live, but sometimes the comparison is just not that clean, he said. [Or] the [standard-of-care] data may be 10 years old. We know that care changes over 10 years.

BMS took a step in that direction with another CAR-T in its portfolio, Breyanzi, which was approved in February for large B-cell lymphoma. Last week, it released trial data showing an improvement in event-free survival in patients who received the CAR-T treatment versus those who were treated with the gold standard, Roches Rituxan, high-dose chemotherapy and stem cell transplant.

It is too early to define Breyanzis ability to extend lives, but BMS hopes the data will ultimately move the CAR-T earlier in the treatment regimen and set the product apart from lymphoma rivals Yescarta and Kymriah. Breyanzi was introduced at a list price of $410,300. ICER has not yet reviewed the product.

Mizuhos Yang says being able to getthe green light for earlier treatment may be the motivation for such head-to-head trials, and, ultimately, these data would likely bring ICER on board with high-priced cell and gene therapies, too. The earlier you treat the patient, the better off they are. And the better off they are, the lower the net cost will be to the healthcare system, she says.

RELATED: Bristol's new myeloma CAR-T needs a hefty discount to be cost-effective, watchdogs say while endorsing GSK's Blenrep

Gene therapies, on the other hand, have received mixed reviews from ICER on cost-effectiveness. Take Spark Therapeutics Luxturna, which is approved for a rareinherited disease that causes blindness for those aged one year and older. ICER found that the product, whichlaunched at $850,000, would be cost-effective if given to patients at the age of threebut not if patients were dosed at age 15 or later, because by that time they would have already lost a significant amount of eyesight.

Yang attributed the verdict to the cost of care. ICER tries to quantify how much a caregiver costs, so if the child is treated earlier, the idea would be that theyre more independent, they require less care, Yang says. If theyre treated at 15, a lot of that cost has already happened. And this message is consistent with gene therapy in general.

But Pearson said ICERs reviewers placed more weight on the overall value to society of preventing blindness. There really are no significant healthcare costs for being blind, he said. But society saves money to a certain extent, because educational costs are lower, patients have full careers and higher earnings potential if theyre fully sighted.

ICER initially balked at Novartis' $2.1 million gene therapy for spinal muscular atrophy (SMA), Zolgensma, but it didn't approve of Biogen's SMA drug Spinraza either. Spinraza launched at up to $750,000 for the first year and half that every year thereafter, prompting ICER to ultimatelydetermine Zolgensma is the lesser of two evils on cost.

BioMarins Roctavian, a hemophilia gene therapy, has hit some bumps on the road to approval, but ICER has already determined how it would be cost-effective. Using a placeholder price of $2.5 million, ICER estimated if the gene therapy is durable for a dozen years, it would save the healthcare system about $5 million per patient compared to chronically dosed factor VIII. Thats an example of where gene therapy can really bring down [costs], Yang said.

RELATED: ICER's blasted pharma pricing for years, but now drugmakers are 'rolling up their sleeves' to cooperate

After delving into ICERs methodology, Yang said she came away with one bone to pick with the agency. Right now, their judgments are based on clinical valuation, but there should be some sort of consideration for the value of innovation, she said. We learn something along the way, and there has to be long-lasting value in that. I dont think thats being captured.

Pearsons response? We are sensitive to the idea that theres value in having treatments that take different approaches, but we dont consider innovation as something thats separate from the benefits to patients and families, he said. Innovation matters to patients to the degree it helps them live better lives. If its not better for patients, Im not sure we should be paying more for it.

ICER has recently boosted its efforts to maintain an ongoing dialogue with biopharma developers and insurance companies. The agency adopted a formal 12-month checkup policy for each of its published reports, Pearson said. If any new data have been released during that time, reviewers may revisit their original verdict.

It also introduced ICER Analytics, a cloud-based portal that houses all of its economic models. Life sciences companies and insurers can subscribe to ICER Analytics. If [a drugmaker] has new evidence, or if they disagreed with our assumptions the first time around, they can put in their own information and create a different result on fair price, Pearson said. Well put that side by side with our original results so payers can see it. More than 50 companies have signed up for trial subscriptions since ICER Analytics launched in November, he said.

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Cost watchdog ICER will bless some high-priced gene and cell therapies, but only with solid proof of benefit: Analyst - FiercePharma

- Collaboration leverages expertise and capabilities of both companies towards developing life-changing treatments for severe neurological diseases -

ZUG, Switzerland and CAMBRIDGE, Mass. and THOUSAND OAKS, Calif., June 15, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and Capsida Biotherapeutics Inc., a biotechnology company dedicated to developing breakthrough gene therapies using fully integrated adeno-associated virus (AAV) engineering, cargo development and manufacturing, today announced a strategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreichs ataxia.

Under the agreement, CRISPR Therapeutics will lead research and development of the Friedreichs ataxia program and perform gene-editing activities for both programs, and Capsida will lead research and development of the ALS program and conduct capsid engineering for both programs. Capsidas high-throughput AAV engineering platform generates capsids optimized to target specific tissue types and limits transduction of tissues and cell types that are not relevant to the target disease, potentially allowing for improved efficacy and safety. CRISPR Therapeutics and Capsida will each have the option to co-develop and co-commercialize the program that the other company leads. Following such option, the companies would equally share all research, development and commercialization costs and profits worldwide related to the collaboration product. As part of the collaboration, Capsida will also be responsible for process development and clinical manufacture of both programs and have the option to manufacture commercial products generated under the agreement.

We are excited to enter this collaboration with Capsida. The combination of Capsidas AAV engineering platform and CRISPR Therapeutics gene-editing platform has the potential to enable transformative gene-edited therapies for patients with neurological diseases, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. This new partnership is one more step in our overall strategy of bringing together innovative and complementary technologies to unlock the full potential of our core platform.

Bringing together Capsidas fully integrated, tissue targeting gene therapy platform with CRISPR Therapeutics leading gene-editing capabilities gives us the potential to develop first-in-class gene therapies for patients with severe neurological disorders and expand the reach of Capsidas broadly enabling capabilities, said Robert Cuddihy, M.D., Chief Executive Officer of Capsida Biotherapeutics.

AboutCRISPR Therapeutics

CRISPR Therapeuticsis a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA.CRISPR Therapeuticshas established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts,CRISPR Therapeuticshas established strategic collaborations with leading companies includingBayer, Vertex Pharmaceuticals andViaCyte, Inc.CRISPR Therapeutics AGis headquartered inZug, Switzerland, with its wholly-ownedU.S.subsidiary,CRISPR Therapeutics, Inc., and R&D operations based inCambridge, Massachusetts, and business offices inSan Francisco, CaliforniaandLondon, United Kingdom. For more information, please visitwww.crisprtx.com.

CRISPR THERAPEUTICS word mark and design logo are registered trademarks ofCRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni and Dr. Cuddihy in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the future activities of the parties pursuant to the collaboration and the expected benefits of CRISPR Therapeutics collaboration with Capsida; and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: CRISPR Therapeutics may not realize the potential benefits of the collaboration; uncertainties inherent in the initiation and completion of preclinical studies, including availability and timing of results from preclinical studies; whether results from a preclinical study will be favorable and predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals and that future competitive or other market factors may adversely affect the commercial potential for product candidates; potential impacts due to the coronavirus pandemic, such as the timing and progress of preclinical studies; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is developing tissue-targeted gene therapies using its biologically driven, high-throughput adeno-associated virus (AAV) engineering and proprietary cargo development platform. As a fully integrated gene therapy company, Capsida is combining its differentiated AAV engineering and screening capabilities with cargo development and state-of-the-art manufacturing to establish a proprietary pipeline of groundbreaking gene therapies across a range of therapeutic areas for indications that are unreachable with current technologies. The companys leadership is backed by decades of successful biologics manufacturing experience and deep AAV biology expertise. Visit us at http://www.capsida.com to learn more.

CRISPR Therapeutics Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

Capsida Media Contact:Greig Communications, Inc.Kathy Vincent+1-310-403-8951kathy@greigcommunications.com

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CRISPR Therapeutics and Capsida Biotherapeutics Announce - GlobeNewswire

John Tlumacki/The Boston Globe via Getty Images

Pivoting from conflicting andcontroversial pressover its recently approved Alzheimers disease treatment,Biogenhas announced yesterday that its gene therapy drug timrepigene emparvovec has failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial.

The late-stage STAR study enrolled 169 adult males with choroideremia, an inherited retinal disease characterized by progressive vision loss and ultimate blindness. The investigators evaluated the efficacy and safety of a single subretinal injection of the investigational gene therapy timrepigene emparvovec. This drug was designed to deliver a functional human choroideremia gene into photoreceptor cells and the retinal pigment epithelium to address the conditions underlying genetic causative mechanisms.

In astatement on the STAR findings, Biogen stated the study failed to meet the primary endpoint of the proportion of patients who experienced a 15-letter improvement in the best corrected visual acuity (BCVA) at one year in the intervention arm versus the control group. The primary endpoint was assessed using the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart.

Also, Biogen reported that the trial failed to show efficacy regarding the key secondary endpoints. However, the safety findings from the study were generally consistent with previous research trials.

We extend our deepest gratitude to all those who contributed to the STAR study, including the participants, investigators, site staff and the broader choroideremia community, said Katherine Dawson, M.D., Biogens Head of the Therapeutics Development Unit.

While we are disappointed by the results of the STAR study, we are hopeful that the clinical insights gleaned from this study may help to shape therapeutic innovation for inherited retinal diseases including choroideremia, so that in the future there may be treatment options for the community affected by these debilitating disorders.

The company noted it would continue to assess the STAR studys complete data set before confirming its future plans for clinical development of timrepigene emparvovec.

These topline results from the STAR trial follow a controversial approval of Biogens Alzheimers drug Aduhelm (aducanumab). Asreported by the companyon June 7, the U.S. Food and Drug Administration (FDA) granted accelerated approval to the therapy to address the accumulation of amyloid beta plaques in the brain associated with the neurologic disease.

The approval was based on clinical trial data showing the therapy could reduce these plaques, but these studies did not demonstrate an overall clinically meaningful reduction in cognitive decline. Instead, the plaques serve as a biomarker for cognitive dysfunction in patients with Alzheimers disease, and both Biogen and some regulators at the FDA surmise the reduction in these plaques could possibly provide a beneficial effect.

But as reported in opinion pieces in theNew York TimesandBloomberg, amongother publications, the approval of the dementia drug is based on spotty evidence, which serves to threaten the FDAs reputation.

As such, three-panel members of the FDAs Peripheral and Central Nervous System Drugs Advisory Committee have resigned from their posts in protest over the Aduhelm approval.

My rationale was that the FDA needs to re-evaluate how it solicits and uses the advisory committees because I didnt think that the firm recommendations from the committee in this case were appropriately integrated into the decision-making process, said one of the resigning members, Aaron Kesselheim, a professor of Medicine at Harvard Medical School, in aninterview withReuters.

More here:
After Controversial AD Drug Win, Biogen's Retinal Gene Therapy Flops - BioSpace

Written by AZoNanoJun 15 2021

The rare lung disease is so complicated that its acronym is difficult to pronounce. However, for infants who were unfortunate enough to be born with this disorder, the outcome is generally fatal.

Thedisease is known as alveolar capillary dysplasia with misalignment of the pulmonary veins, or ACDMPV for short. According to a study, the disease is associated with mutations in the FOXF1 gene. Medical experts throughout the world have recorded around 200 cases, but an unestimated number of infants could have died without the disorder ever being diagnosed, stated the National Organization for Rare Disorders.

The disease is induced by genetic changes that inhibit the formation of proper blood vessels in the lungs. Within a few days or weeks post birth, infants turn blue because of the absence of oxygen while blood pressure increases inside their lungs. The few infants who get to live do so by receiving exceptionally rare infant-sized lung transplants.

Now, a new study headed by experts from Cincinnati Childrens and the University of Cincinnati has reported about helping mice (with a FOXF1 mutation that is identical to human ACDMPV patients) live longer with this fatal disease. This was done by applying high-tech nanoparticles to send a STAT3 gene into the lungs to trigger the growth of blood vessels.

STAT3 is a crucial downstream target of the FOXF1 gene and its delivery can rectify the vascular deficiency in ACDMPV mice. The study results were published online in the Circulation journal on June 11th, 2021.

If these findings can be matched in human analyses in the future, this success can potentially boost the speed of development for other nanoparticle-based treatments for many medical conditions, stated the study co-authors.

Nanoparticle carriers have shown minimal toxicity and have accelerated the development of novel therapies for human cancers, diabetes, and chronic inflammatory disorders. We have developed a unique nanoparticle delivery system that can deliver genes capable of stimulating micro-vessel growth in the newborn lung.

Vlad Kalinichenko, MD, PhD, Study Senior Author and Member of the Center for Lung Regenerative Medicine and Perinatal Institute, Cincinnati Childrens Hospital Medical Center

Kalinichenko added, This study shows that a single injection of the nanoparticles with the STAT3 gene vector was sufficient to increase alveolar-capillary density, prevent excessively high blood pressuresand dramatically improve survival.

Around 70% of mice born with ACDMPV die in less than 28 days of birth without treatment. The new therapy reduced this mortality rate to 35%, stated Fei Sun, PhD, the first author of the study and a member of the Center for Lung Regenerative Medicine at Cincinnati Childrens.

This nanoparticle method is different from gene replacement therapies that can cause permanent changes in the body. It involves materials that do not remain in the body for more than seven days. Yet, in the mice examined so far, a single therapy early after the birth was sufficient to divert a whole stream of later-developing issues that take place with ACDMPV.

The treatment works by sending an engineered nanoparticle composed of many fatty acids, polymersand a small amount of cholesterol that transports the non-integrating STAT3 gene, which consequently encourages the growth of blood vessels in the lung tissue.

Kalinichenko and collaborators also noted the molecular processes involved as part of their ongoing analyses of lung development. The nanoparticle was designed with support from Zicheng Deng and Andrew Dunn, who are both graduate students mentored by Donglu Shi, PhD, from the Materials Science and Engineering Program at the University of Cincinnati.

With the presence of more blood vessels, the rapidly growing lungs in the newborns developed in a closer-to-normal fashion, without triggering harmful molecular remodeling signals that can lead to permanent malformations and even death from lung failure.

The study explains how the therapy enhanced various measures of heart, lungand blood vessel health, such as the ratio of pulmonary acceleration time to pulmonary ejection time (PAT/PET), blood pressure in the right ventricle, arterial oxygenation levels, the diameter of pulmonary arteriesand also the thickness of their walls.

More studies need to be completed before nanoparticles can be tested in human newborns with ACDMPV, such as safety tests and establishing whether recurrent treatments would be required.

Sun, F., et al. (2021) Nanoparticle Delivery of STAT3 Alleviates Pulmonary Hypertension in a Mouse Model of Alveolar Capillary Dysplasia. Circulation. doi.org/10.1161/CIRCULATIONAHA.121.053980.

Source: https://www.cincinnatichildrens.org/

Continued here:
Nanoparticle-Based Therapy Could Treat Fatal Lung Disease in Newborns - AZoNano

DUBLIN--(BUSINESS WIRE)--The "Regenerative Medicine Market - Global Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

The regenerative medicine market size to grow at a CAGR of around 34% during the period 2020-2026.

Increased R&D investments by pharmaceutical companies will drive the demand for regenerative medicines. Europe plays a significant role in supporting the development and authorization of these products for several genetic and rare disorders. Increased funding via several venture capitalists and governments, and private institutions contribute significantly to the global regenerative medicine market growth.

The increased prevalence of diseases such as cardiovascular diseases and diabetes can drive cell and gene therapy and tissue-engineered products. With the rise in thermal burns, occupational burn accidents, and chronic wounds, regenerative medicine products will experience steady growth. Novartis and Gilead Sciences are the key companies offering various therapies to treat cancer, genetic, and rare disorders.

The report considers the present scenario of the regenerative medicine market and its market dynamics for 2019-2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The study covers both the demand and supply sides of the market. It also profiles and analyzes leading companies and several other prominent companies operating in the market.

REGENERATIVE MEDICINE MARKET SEGMENTATION

The regenerative medicine market research report includes a detailed segmentation by application, products, end-users, geography. Oncology constitutes the largest portion of the global regenerative medicine market share. The development of curative therapies by CAR-T and cell and gene therapies is widely popularized in the oncology therapeutic area. The increasing global prevalence rates and the increasing rates of different types of life-threatening cancers are the most important key factors that drive the oncology segment.

Consistent innovations in gene therapies due to the increased number of clinical trials and pipeline products are driving the growth prospects. Hence, the increased inflow of funding for the development of gene therapy is one of the driving factors for the sector growth. Cell therapy is the major revenue contributor. The increasing prevalence of diabetes and foot ulcers is the primary factor contributing to the growth of tissue-engineered products. The tissue-engineered product segment to grow at a CAGR of 8% by 2026.

Hospitals are likely to remain a dominant revenue contributor to the global regenerative medicine market. Around 50% of therapeutic surgeries performed in the US annually, including cardiovascular and musculoskeletal, occur in hospitals. Cancer care centers are likely to witness an incremental growth of approx. USD 10 billion by 2026.

As cancer is the second leading cause of death across the globe, which is responsible for approx. 10 million deaths annually, the scope of cancer centers is growing. Key vendors are focusing more on cancer care centers than hospitals to promote their products. As the cancer centers are being covered under reimbursement schemes, the growth of these facilities is likely to increase during the forecast period.

KEY QUESTIONS ANSWERED:

1. How big is the regenerative medicine market?

2. What are the critical applications of regenerative medicine products?

3. Who are the key players in the regenerative medicine market?

4. Which segment accounted for the largest regenerative medicine market share?

5. Which region holds the largest share in the global regenerative medicine market?

6. How has the COVID-19 pandemic affected the regenerative medicine industry?

Key Vendors

Other Prominent Vendors

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage

5 Report Assumptions & Caveats

6 Market at a Glance

7 Introduction

8 Market Opportunities & Trends

8.1 Latest Advances In Tissue-Engineering Therapies

8.2 Robust Product Pipeline Of Regenerative Medicine Companies

8.3 Strategic Acquisitions By Regenerative Medicine Companies

9 Market Growth Enablers

9.1 Increase In Acute, Chronic, & Genetic Disorders

9.2 Increasing Demand For Car T-Cell Therapies

9.3 Faster Regulatory Approvals & Special Designations Of Rm Products

10 Market Restraints

10.1 Manufacturing, Operational, & Ethical Challenges

10.2 High Cost of Regenerative Therapies

10.3 Outbreak of The COVID-19 Pandemic

11 Market Landscape

11.1 Market Overview

11.2 Market Size & Forecast

11.3 Five Forces Analysis

12 Application

12.1 Market Snapshot & Growth Engine

12.2 Market Overview

12.3 Dermatology

12.4 Musculoskeletal

12.5 Oncology

12.6 Genetic Disorders

13 Product

13.1 Market Snapshot & Growth Engine

13.2 Market Overview

13.3 Gene Therapy

13.4 Cell Therapy

13.5 Tissue-Engineering

14 End-Users

14.1 Market Snapshot & Growth Engine

14.2 Market Overview

14.3 Hospitals

14.4 Cancer Care Centers

14.5 Wound Care Centers

14.6 Ambulatory Surgical Centers

For more information about this report visit https://www.researchandmarkets.com/r/cfc68n

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Regenerative Medicine Market - Global Outlook and Forecast 2021-2026 - Increasing Demand For Car T-Cell Therapies - ResearchAndMarkets.com - Business...

June 16, 2021

The Discovery Labs of King of Prussia now boasts another major research and development organization within its growing "Cellicon Valley." The innovation hub announced a multi-year lease with the University of Pennsylvania's Gene Therapy Program, a cutting-edge research and development organization, last month. It will focus its endeavors in Montgomery County on the development of medicine for rare and orphan diseases and treatments for contagious pathogens such as COVID-19.

Currently located in the University City neighborhood of Philadelphia, the Gene Therapy Program has outgrown its space at Penn's Translational Research Center. It's located in an area where space is at a premium. Rather than pay University City real estate's steep priceswhich have remained resilient amid the pandemicor commission new construction, Director James M. Wilson, M.D., Ph.D., has decided to expand outward. The space at the Discovery Lab, formerly occupied by pharmaceutical giant GSK, provides the necessary infrastructure and size to house an expanding and technologically advanced research and development (R&D) program,

"The past few years have been a pivotal time in the development of gene therapies, and our new space at Discovery Labs will propel us even further in charting the future of the field," Wilson said. "With additional state-of-the-art equipment and increased operational capabilities, we are well-positioned to enhance the design and execution of our innovative, transformative therapies."

Largely driven by the lack of research and drug development dedicated to patients with rare, inherited diseases, the Gene Therapy Program has been at the forefront of research into Adeno-Associated Virus (AAV) Vectors. AAVs are largely benign viruses that have proven modifiable by gene therapy. By reconstructing or repairing the genetic material within the viruses, scientists have been able to produce therapeutic effects in patients with rare, genetic diseases. Wilson and others at the Gene Therapy Program have also expanded their research to include similar approaches to pathogenic viruses such as COVID-19.

Moving to the Discovery Labs enhances the program's ability to conduct such advanced research. The new space includes two full floors for the Penn Vector Core, a major technological resource for investigators and therapy designers that advances the understanding of gene function, allowing for the development of new vector medicines. The location also provides ample space for every stage of drug R&D, from discovery research all the way through applications for clinical trials. All told, the multi-year lease includes over 150,000 square feet of lab space and two buildings on the Discovery Lab's campus.

With innovative R&D organizations such as Penn's Gene Therapy Program flocking to the Discovery Labs, King of Prussia will find itself in an enviable position post-pandemic. Penn joins Thomas Jefferson University Health, CHOP, the Centers for Breakthrough Medicine, GSK, and numerous other major healthcare companies and institutions at the Discovery Labs, positioning the company as the host for some of the most innovative and groundbreaking medical research in the United States.

Led by founder and chairman Brian O'Neill, the Discovery Labs have sought to leverage its more than one million square feet of lab space in conjunction with the amenities of their immediate location and proximity to Philadelphia's "Cellicon Valley," to attract some of the largest and most impactful healthcare companies and organizations to King of Prussia. So far, it appears to be working.

"Dr. Wilson and his incredible team at Penn have helped establish Philadelphia as a leader in gene therapies, and we are deeply honored to have the opportunity to work with him and his incredible team of scientists through the expansion of their world-class research and development labs," O'Neill said. "To welcome Penn's Gene Therapy Program as the anchor tenant for our planned life sciences cluster here in King of Prussia is strong validation for the incredible life science ecosystem that is emerging in Greater Philadelphia."

It seems as if, for the foreseeable future, King of Prussia will stand as one of the brighter extensions of the Delaware Valley's expanding "Cellicon Valley," where medical innovation, scientific research, and treatment development are in the midst of an ongoing renaissance. The advent and growth of the Discovery Labs underscore the increased demand for top-flight R&D real estate, and offers promising returns for both the area and the greater medical community through its latest partnership with the University of Pennsylvania.

The Delaware Valley Journal provides unbiased, local reporting for the Philadelphia suburbs of Bucks, Chester, Delaware and Montgomery Counties. For more stories from the Delaware Valley Journal, visit DelawareValleyJournal.com

Link:
Penn's Gene Therapy Program Moves To Innovation Hub In King Of Prussia - Patch.com

LAS VEGAS, June 17, 2021 (GLOBE NEWSWIRE) -- Zhittya Genesis Medicine, Inc. (a private company) (Zhittya or the Company), is proud to announce that Mr. Daniel C. Montano, CEO, and founder of Zhittya Genesis Medicine Inc. has received the award of Biotech Entrepreneur of the Year from the Nevada Biotechnology & Health Science Consortium. We also congratulate Dr. Marc Kahn, Dean of the Kirk Kerkorian School of Medicine at the University of Nevada, Las Vegas (UNLV), for receiving the Medical Professional of the Year Award from the same institution.

The Nevada Biotechnology & Health Science Consortium (NevBio) was founded in 2007 to encourage the development and expansion of the Health Sciences and Biotechnology in Southern Nevada. NevBio is part of the national biotechnology association, the Biotechnology Innovation Organization (BIO) and the Council of Bioscience Associations. Thousands of people have attended the monthly presentations sponsored by NevBio over the last 14 years on topics such as immunotherapy, nuclear medicine, gene editing, sepsis, cancer, C-Diff, pain, biofilm, angiogenesis, clinical trials, patents, raising capital, and much more. NevBio has given legislative advice both in Carson City & Washington DC, on assisting the development of health sciences in Southern Nevada. Senator Harry Reid and the late Nevada Chancellor Jim Rogers have spoken at the award dinners.

Daniel C. Montano, CEO of Zhittya Genesis Medicine Inc., is a biotech pioneer leading the creation of a revolutionary biopharmaceutical treatment termed Therapeutic Angiogenesis and is the recipient of the Biotech Entrepreneur of the Year Award. Daniel Montano moved to Las Vegas in 2002 from California to advance biotechnology here. Therapeutic Angiogenesis has demonstrated in US FDA clinical trials that it can trigger the growth of new blood vessels, thereby treating heart disease, the number one cause of death in the world. In animal experiments, monkeys given experimental Parkinsons disease, then treated with Therapeutic Angiogenesis, reversed their disease progression. Heart disease, strokes, peripheral artery disease, Parkinsons disease, multiple sclerosis and Alzheimers disease are all diseases he is applying Therapeutic Angiogenesis to treat. If successful, Las Vegas could become the biotech center of the world for Regenerative Medicine through Therapeutic Angiogenesis.

Dr. Marc Kahn will be awarded the Medical Professional of the Year, as the Dean of the new Kirk Kerkorian School of Medicine. Dr. Kahns experience is in both medicine and teaching medical professionals the business of medicine. His introduction of a joint MD-MBA program was very well received. His leadership of the School of Medicine is critical to the long-term success of making Las Vegas a biomedical center.

The Las Vegas Valley is developing into a biomedical center, with three medical schools and biotech companies that have either started here or have moved here from California. Biotech and health science companies are moving to Las Vegas to take advantage of its low costs, world-class airport, and hospitality facilities. The Las Vegas Valleys population has grown to almost 2.8 million people and is still growing. The Awards Dinner will be on Thursday, July 15th at 6 pm at the Ahern Hotel in Las Vegas. Register at nevbio.org.

The Awards Dinner will be:

Contact Information: Daniel Montano, CEO Zhittya Genesis Medicine, Inc. Phone: (1) 702-790-9980 E-mail: dan@zhittyamedicine.com Website: zgm.care

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Mr. Daniel C. Montano, CEO, is recognized as Biotech Entrepreneur of the Year and Dr. Marc - GlobeNewswire

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats at the LifeSci Genetics Medicine Conference and LSX Biotech Growth CEO Forum.

Conferences Details:

Event:

LifeSci Genetics Medicine Conference

Date:

Tuesday, June 22, 2021

Time:

10:00 am ET

Format:

Fireside chat

Participants:

RA Session II, President, Founder and CEO

Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

Kamran Alam, Chief Financial Officer

Event:

LSX Biotech Growth CEO Forum

Topic:

Too Much of a Good Thing Can be Wonderful: Optimizing Value From Broad Discovery Platforms Through Strategy and Strategic Partnering

Date:

Tuesday, June 22, 2021

Time:

1:10 pm ET

Format:

Fireside chat

Participants:

RA Session II, President, Founder and CEO

Webcasts for these conferences will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

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.

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Taysha Gene Therapies to Participate in Upcoming Investor Healthcare Conference and CEO Forum - Business Wire

Scientists led by UNC School of Medicine researchers Silvia Kreda, PhD, and Rudolph Juliano, PhD, created an improved oligonucleotide therapy strategy with the potential for treating other pulmonary diseases, such as COPD and asthma.

CHAPEL HILL, NC UNC School of Medicine scientists led a collaboration of researchers to demonstrate a potentially powerful new strategy for treating cystic fibrosis (CF) and potentially a wide range of other diseases. It involves small, nucleic acid molecules called oligonucleotides that can correct some of the gene defects that underlie CF but are not addressed by existing modulator therapies. The researchers used a new delivery method that overcomes traditional obstacles of getting oligonucleotides into lung cells.

As the scientists reported in the journal Nucleic Acids Research, they demonstrated the striking effectiveness of their approach in cells derived from a CF patient and in mice.

With our oligonucleotide delivery platform, we were able to restore the activity of the protein that does not work normally in CF, and we saw a prolonged effect with just one modest dose, so were really excited about the potential of this strategy, said study senior author Silvia Kreda, PhD, an associate professor in the UNC Department of Medicine and the UNC Department Biochemistry & Biophysics, and a member of the Marsico Lung Institute at the UNC School of Medicine.

Kreda and her lab collaborated on the study with a team headed by Rudolph Juliano, PhD, Boshamer Distinguished Professor Emeritus in the UNC Department of Pharmacology, and co-founder and Chief Scientific Officer of the biotech startup Initos Pharmaceuticals.

About 30,000 people in the United States have CF, an inherited disorder in which gene mutations cause the functional absence of an important protein called CFTR. Absent CFTR, the mucus lining the lungs and upper airways becomes dehydrated and highly susceptible to bacterial infections, which occur frequently and lead to progressive lung damage.

Treatments for CF now include CFTR modulator drugs, which effectively restore partial CFTR function in many cases. However, CFTR modulators cannot help roughly ten percent of CF patients, often because the underlying gene defect is of the type known as a splicing defect.

CF and splicing defects

Splicing is a process that occurs when genes are copied out or transcribed into temporary strands of RNA. A complex of enzymes and other molecules then chops up the RNA strand and re-assembles them, typically after deleting certain unwanted segments. Splicing occurs for most human genes, and cells can re-assemble the RNA segments in different ways so different versions of a protein can be made from a single gene. However, defects in splicing can lead to many diseases including CF when CFTRs gene transcript is mis-spliced.

In principle, properly designed oligonucleotides can correct some kinds of splicing defects. In recent years the U.S. Food and Drug Administration has approved two splice switching oligonucleotide therapies for inherited muscular diseases.

In practice, though, getting oligonucleotides into cells, and to the locations within cells where they can correct RNA splicing defects, has been extremely challenging for some organs.

It has been especially difficult to get significant concentrations of oligonucleotides into the lungs to target pulmonary diseases, Kreda said.

Therapeutic oligonucleotides, when injected into the blood, have to run a long gauntlet of biological systems that are designed to keep the body safe from viruses and other unwanted molecules. Even when oligonucleotides get into cells, the most usually are trapped within vesicles called endosomes, and are sent back outside the cell or degraded by enzymes before they can ever do their work.

A new delivery strategy

The strategy developed by Kreda, Juliano, and their colleagues overcomes these obstacles by adding two new features to splice switching oligonucleotides: Firstly, the oligonucleotides are connected to short, protein-like molecules called peptides that are designed to help them to distribute in the body and get into cells. Secondly, there is a separate treatment with small molecules called OECs, developed by Juliano and Initos, which help the therapeutic oligonucleotides escape their entrapment within endosomes.

The researchers demonstrated this combined approach in cultured airway cells from a human CF patient with a common splicing-defect mutation.

Adding it just once to these cells, at a relatively low concentration, essentially corrected CFTR to a normal level of functioning, with no evidence of toxicity to the cells, Kreda said.

The results were much better with than without OECs, and improved with OEC dose.

There is no mouse model for splicing-defect CF, but the researchers successfully tested their general approach using a different oligonucleotide in a mouse model of a splicing defect affecting a reporter gene. In these experiments, the researchers observed that the correction of the splicing defect in the mouse lungs lasted for at least three weeks after a single treatment hinting that patients taking such therapies might need only sporadic dosing.

The researchers now plan further preclinical studies of their potential CF treatment in preparation for possible clinical trials.

Yan Dang, Catharina van Heusden, Veronica Nickerson, Felicity Chung, Yang Wang, Nancy Quinney, Martina Gentzsch, and Scott Randell were other contributors to this study from the Marsico Lung Institute; Ryszard Kole a co-author from the UNC Department of Pharmacology.

The Cystic Fibrosis Foundation and the National Institutes of Health supported this work.

Media contact: Mark Derewicz, UNC School of Medicine, 919-923-0959

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Scientists Demonstrate Promising New Approach for Treating Cystic Fibrosis | Newsroom - UNC Health and UNC School of Medicine