Category Archives: Gene Medicine

When Novartis reported first-half Zolgensma sales this summer, the company said its spinal muscular atrophy (SMA) gene therapy was growing thanks to"expanding access" in Europe. This week at the Fierce Biotech Cell & Gene Therapy event, an exec outlined what's driving the uptake.

So far, Novartis has negotiated Zolgensma access for half of the SMA patient population in Europe, including for patients inEngland,Scotland, France, Germany, Italy, Belgium and other countries,Mike Fraser,general manager of Europe, Middle East and Africa at Novartis Gene Therapies, said in an interview.Novartis also has deals in place with six countries in the Middle East and Africa, he added.

Zolgensma scored its FDA approval in 2017 and its EU nod in 2020. Even ahead of the EU approval, Novartis was working behind the scenes to ensure a quick rollout in the region.

In some countries, it can take months or years for access negotiations to play out, Fraser said. Babies with SMA dont have time to wait, he added.

To hit the ground running, the company implemented a day one" access program. In government access negotiations, Novartis offered outcomes-based contracting, annuity payments or deferred payments, in which the company would cover the cost of treatment upfront and collect payments after access talks play out.

RELATED:New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

While Novartis has seen progress, Fraser still sees limitations with current healthcare systems. As he sees it, healthcare systems "are still not set up to recognize the value of these one-time therapies."That's despite the fact that expensive gene therapies can bring savings over a patient's life compared withconventional medicines.

By 2030, Fraser said he expects more than 20 gene therapies to be on the market.While Novartis is on the cutting edge of interacting with payers, he urged other companies to start getting involved in their own access talks.

One lesson for Novartis? Because healthcare and financialsituations vary from country to country, the pharmaceutical industry is going to have to get used to offering" a range of access options, Fraser said.

With these new and innovative treatments, bespoke and customized solutions are going to be the future, Fraser said.

Aside from access talks, Novartis is also participating in an effortto screen babies for SMA. Through a partnership with SMA Europe, the pharmaceutical industry aims to screen 100% of newborns across Europe by 2025.

RELATED:Novartis back with bid for 'multibillion-dollar' Zolgensma expansion in older SMA patients as FDA lifts clinical hold

The European approval of Zolgensma last year wasone "inflection point" that Novartis had been anticipating for the gene therapy since its original FDA nod. Meanwhile, the company is advancing aspinal injection formulation for patients between twoand 18 years. A potential approval for that use represents a "multibillion-dollar" opportunity, CEO Vas Narasimhan has said.

Zolgensma generated $634 millionduring the first half of the year, a 69% increase compared with 2020. The medicine is approved in more than 40 countries.

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Novartis has negotiated Zolgensma access for half of the European SMA population, exec says. How'd it get there? - FiercePharma

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SOUTH SAN FRANCISCO, Calif., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Senti Bio, a leading gene circuit company, today announced that its co-founder and chief executive officer, Tim Lu, MD, PhD, has been appointed to the Alliance for Regenerative Medicines (ARM) 2022 Board of Directors.

ARM envisions a world where advanced therapies are able to successfully alter current medical practices by treating the root causes of disease and disordersand I am honored to support this mission, said Dr. Lu. We are witnessing an era of unprecedented innovation and growth in this field, particularly in the areas of cell and gene therapy, and I look forward to working with ARMs board members of accomplished scientists and leaders to continue to support and inspire ARMs mission.

Lu added, I believe that cell and gene therapies have the potential to truly revolutionize the practice of medicine and, at Senti, we are proud to be a part of this exciting convergence of science and technology. 2021 has been an incredible year for our company so far: we initiated two partnerships in gene circuit-enabled cell and gene therapies, presented a suite of new data supporting our proprietary off-the-shelf NK cell programs at major conferences, and commenced the buildout of a wholly-owned cell therapy manufacturing facility. These are significant steps toward developing smarter medicines for patients using our gene circuit platform.

"We are pleased to welcome Tim Lu, CEO of Senti Biosciences, to the ARM Board of Directors," said Janet Lambert, CEO of ARM. "Our sector is poised to shape healthcare for years to come and our Board will be instrumental in advancing the delivery of transformative therapies for patients globally, while helping to eradicate barriers and legacy policies that could slow access.

About ARM and its Executive Committee and Board of DirectorsARMs Executive Committee and Board of Directors oversee the formation and execution of ARMs strategic priorities and focus areas over the coming year. Each group is held to an annual reelection or rotation process, with nominations and approval by the ARM membership and current Board. ARM promotes legislative, regulatory and reimbursement initiatives to advance regenerative medicines, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound benefits that are helping thousands of patients worldwide. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies.

About Senti BioOur mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that we believe may enable us to program next-generation cell and gene therapies with what we refer to as gene circuits. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are designed to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We aim to design gene circuits to improve the intelligence of cell and gene therapies in order to enhance their therapeutic effectiveness against a broad range of diseases that conventional medicines do not readily address. For more information, please visit the Senti Bio website at https://www.sentibio.com.

Find more information at sentibio.comFollow us on Linkedin: Senti BiosciencesFollow us on Twitter: @SentiBio

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Senti Bio CEO Appointed to The Alliance for Regenerative Medicine 2022 Board of Directors - StreetInsider.com

In genetic medicines, delivery is key. You want to make sure that the medicine reaches its destination in the body and doesnt cause any problems along the way. But before that, you want to ensure that the chosen delivery vehicle has enough room to carry everything it needs for the trip. ReCode Therapeutics is developing technology intended to improve on the capacity and the targeting of genetic medicines, and its first stops are the lungs.

ReCode has two programs on track toward clinical testing and the biotech has raised $80 million to advance those programs and others. Pfizer Ventures and EcoR1 co-led the Series B round of funding announced Thursday.

Many of the genetic medicines that are available as well as some still in development use engineered viruses for delivery. These viruses are very good at getting into a cell, but the body sees them as foreign, so the immune system creates antibodies against them. That means viral delivery can only be used once in a patient. Viruses can also spark dangerous side effects.

ReCode avoids the limitations of viruses by using lipid nanoparticles (LNPs). These particles are made from cholesterol and lipids, a type of fat, so the body is familiar with them, CEO David Lockhart said. That familiarity reduces the risk of an immune response. Furthermore, because LNPs dont cause the immune system to produce antibodies, they can be redosed if needed. Just as important, these particles can be designed to go to specific tissues in the body.

Weve only scratched the surface, Lockhart said. We can do even more with targeted delivery to more cell types, tissue types, beyond the liver, lung, and spleen.

LNPs have a preference for going to the liver. Thats great for genetic medicines for liver diseases, and companies such as Alnylam Pharmaceuticals have used that preference and optimized it for therapies that reach liver cells. Genetic medicines employ three or four lipids in their LNPs. ReCode adds a fifth one. The companys delivery technology comes from the lab of University of Texas Southwestern Medical Center Professor Daniel Siegwart, a co-founder of the company. Lockhart said that adding a fifth lipid changes how the LNP binds to proteins in the blood in way that removes the liver as a target, and enables delivery to other targets in the body. The technology is called Selective Organ Targeting, or SORT. In addition to the ability to target, Lockhart said that ReCodes LNPs offer greater capacity than viruses as well as the ability to carry mixed payloads of genetic cargo.

ReCode has two lead programs that are preclinical. One is for primary ciliary dyskinesia (PCD), a disease that causes dysfunction in cilia, organelles that extend from cells. The other is for cystic fibrosis (CF), a disease that leads to fluid buildup in the lungs. Both have defined genetic causes, but PCD has no treatments and while Vertex Pharmaceuticals has developed drugs that address various genetic mutations, Lockhart noted that about 10% to 13% of CF patients dont respond to those therapies.

ReCode aims to treat PCD and CF by delivering RNA and gene correction therapies. The first therapies that the company is developing are inhaled medicines for delivery to the epithelial cells that line the lung. These therapies wont need to be targeted as inhalation gets the medicine where it needs to go. But Lockhart noted that for some lung disorders, delivery to the endothelial cells that line blood vessels is needed. Those drugs, such as a gene correction CF therapy ReCode is developing, will employ SORT.

The new financing will be used to move both the PCD and CF programs closer to the clinic. Lockhart said that the preclinical research that supports an investigational new drug application (IND) will begin in the first half of next year, followed by an IND filing in the second half. Some of the capital will be used to develop additional programs that are also for respiratory disorders with defined genetic causes.

There are other companies trying to take LNPs beyond the liver. The technology of Georgia Tech spinout Guide Therapeutics screens for LNPs that can be used to deliver RNA to cells throughout the body. Beam Therapeutics saw enough promise in the approach to commit $120 million to acquire the startup earlier this year.

Though the delivery capability of ReCodes technology has applications beyond the lung, the company has no plans to pursue all of them. Lockhart said ReCode will keep respiratory disorders as a focus while exploring partnerships with other companies interested in applying the technology to diseases affecting other parts of the body. In the nearer term, ReCode will be building its own manufacturing capabilities to support its clinical trials.

ReCode raised $80 million in Series A funding last year. The latest financing added new investors Sanofi Ventures, funds managed by Tekla Capital Management, Superstring Capital, and NS Investment. Earlier investors participating in the new round include OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, and Osage University Partners. Though Pfizers venture arm did co-lead the financing, Lockhart said that the pharma giant only gets a board seat and has no inside track to SORT.

Theyre very excited about the technology, he said. They basically want to have a front row seat. We were very clear that there was no coupling of rights, no guarantees of access to the technology.

Photo by ReCode Therapeutics

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ReCode Therapeutics gets $80M to deliver on new RNA therapies for the lungs - MedCity News

Every human being is unique, not only in their mind but also in their body. Therefore, not all individuals will respond to the same healthcare treatments in the same way. For this reason, understanding everyones specific features and needs, and tailoring procedures and medications based on them, can make the difference between success and failure.

To achieve this goal, 11 research proposals bringing together Israeli top scientists in the fields of medicine, data analysis, artificial intelligence and more were awarded NIS 32 million by the Israel Precision Medicine Partnership, the organization announced Monday.

Every individual is different because we all have different genes that determine a different biology, and this presents us with the opportunity to treat them better, he said. For example, if 1,000 people have lung cancer, we know that for some of them, this is caused by a mutation in the gene A, which can be treated with drug A, while others have a different type of lung cancer that can be better addressed with a different drug.

It is funded by the Planning and Budgeting Committee of the Council of Higher Education, the Health Ministry, the National Digital Affairs Directorate of the Economy Ministry and two philanthropic organizations, Yad Hanadiv in Israel and the Klarman Family Foundation in the US.

Theres a lot of unused potential in biomedical research, Dor said. We are just starting to scratch the surface, and the system is starved. People typically have very low access to resources, compared with other Western countries. This program, supported by both government bodies and philanthropies, has started to close the gap, and its really pushing scientific discovery forward.

IN THE third round, the partnership received 99 applications, which were screened by an international committee led by American Nobel Laureate in chemistry Prof. Roger Kornberg and almost entirely made up of foreign scholars.

Typically, these proposals are the result of a collaboration between a scientist in the academic field and a clinician from a hospital or another healthcare organization, Dor said, stressing how this kind of interdisciplinary cooperation represents one of the goals of the program.

Each project aims to tackle a different medical challenge.

One of the most interesting proposals was presented by Prof. Aaron Ciechanover, who won the Nobel Prize in chemistry some 15 years ago together with some of his colleagues at the Technion [-Israel Institute of Technology], Dor said.

They are looking into a completely new type of cancer treatment, he said. They think they can manipulate cancer cells in such a way that the cells cannot tolerate the stress created by blocking the degradation of their proteins, and they die off.

Another proposal that is especially innovative is based on the cooperation of Hebrew University of Jerusalem computer scientist Prof. Nir Friedman and Hadassah-University Medical Center hepatologist Prof. Eithan Galun, Dor said.

They came up with a very original way of assessing the situation of the liver without actually performing a biopsy, he said.

Currently, the procedure to examine whether an individual has a liver disease is painful and involves some degree of risk.

They have a brilliant idea on how you can perform a blood test that will provide a lot of information on what is happening in the liver... without actually touching it, but rather finding out exactly which genes are active or dying in the liver or sending signals to the blood, Dor said. They have called it liquid biopsy.

Among the other selected projects, a duo from the Weizmann Institute of Science in Rehovot and the Rabin Medical Center in Petah Tikva aims to improve the sonographic diagnosis and monitoring of breast cancer and Crohns disease using artificial intelligence and super-resolution algorithms. Three experts from Shaare Zedek Medical Center in Jerusalem and the Hebrew University plan to use integrated computational and functional frameworks for optimized gene identification and therapy in rare hereditary diseases.

The NIS 210 million ($65m.) allocated to the partnership in 2018 was meant to last for four rounds of applications. In the previous two rounds, the partnership distributed around NIS 60m. ($18.6m.) each. If the program is not renewed, the next cycle might be the last one.

My hope is that in light of the incredible success of the program, it will persuade decision-makers to carry it on in some form, Dor said.

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From cancer to Crohn, Israel pushes precision medicine to the next level - The Jerusalem Post

Dr. Jay Kolls and Derek Pociask, PhD, in front of a scanned image of a 50-year-old lung tissue slide that was key to solving a medical mystery. Photo by Rusty Costanza.

When scientists were developing a vaccine against COVID-19, a key worry was the potential to cause disease enhancement. Its a complication that occurs when a vaccine does the opposite of what its intended to do and instead makes someone more vulnerable when exposed to a pathogen.

For COVID-19, the fear can be traced back to an infamous clinical trial in the 1960s for an experimental vaccine against respiratory syncytial virus (RSV), the most common cause of pneumonia in children. The vaccine used an inactivated form of the virus, but almost 80 percent of the children who were vaccinated and later exposed to the real virus were hospitalized for severe illness. Two toddlers died. The boys deaths stopped RSV vaccine development for decades and was one reason scientists pursued the novel MRNA vaccine design against COVID-19 instead of using an inactivated form of the respiratory virus.

The cause of these toddlers deaths was a medical mystery for decades until now, according to anew studyco-authored by Tulane University researchers in the journalScience Translational Medicine.

Scientists from Tulane University School of Medicine were able to delicately peel back the layers of 50-year-old medical slides containing lung tissue samples from the children to perform a detailed analysis of gene expression and study their immune response. Researchers compared the results to a control group using a similar analysis of more recent tissue slides from age and race-matched children who died from non-pulmonary causes.

After encountering RSV infection, the toddlers from the 1960s developed lung injury that damaged critical lung cells, said study co-corresponding authorDr. Jay Kolls, John W Deming Endowed Chair in Internal Medicine at Tulane School of Medicine.

These two children died likely due to injury to surfactant producing cells in the lung that are critical for transporting oxygen to the blood, Kolls said.Our study provides a comprehensive characterization of fatal enhanced respiratory disease, a tragedy that hampered RSV vaccine development for decades.

Kolls worked with co-corresponding author Fernando P. Polack of Vanderbilt University and Fundacion INFANT in Argentina and an international research team on the project.Derek Pociask, assistant professor of medicine at Tulane, devised the method for extracting material from the archived medical slides. It was no easy task considering the slides were only a centimeter wide and just six microns thick.

It took us four months of optimizing to get the method for getting RNA off of an old slide that had been cover-slipped, Pociask said. In this case, the only thing we had left from these kids were these slides from the 1960s. So, we practiced as much as we could.

The results of the study could make future attempts at developing an RSV vaccine safer by providing a molecular signature of enhanced RSV disease. Future vaccine developers can look for these markers when they test promising candidates in preclinical models.

They can look for the same gene signatures that we identified, Kolls said. If they can show that their vaccine doesn't elicit any of that kind of response, I think that would give both a potential funding sponsor or the FDA some comfort level that the vaccine is safe.

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Tulane scientists unravel 50-year-old medical mystery behind toddlers' deaths from RSV vaccine - Tulane News

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Dr. Helen Heslop, director of the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Childrens Hospital and Houston Methodist Hospital and interim director of the Dan L Duncan Comprehensive Cancer Center at Baylor, has been elected to the National Academy of Medicine. She is among 90 regular members and 10 international members announced today at the Academys annual meeting.

Election to the National Academy of Medicine is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service.

Dr. Heslop is a leader in innovative science. Her work is changing the way we treat cancer and improving patient care, said Dr. Paul Klotman, president, CEO and executive dean of Baylor. She also is helping to train future physician-scientists, and we are proud to have her leadership at Baylor. She is extremely deserving of this honor, and we look forward to her continued achievements.

Heslop is a professor of medicine and pediatrics and holds the Dan L Duncan Chair at Baylor. She is a physician-scientist engaged in translational research focusing on adoptive immunotherapy with gene-modified effector cells to improve hemopoietic stem cell transplantation and cancer therapy. She has extensive experience in developing and conducting transplant studies and cell and gene therapy studies and currently holds more than 20 Investigational New Drug Applications. She also has played a major role nationally as president of societies focusing on the intellectual and clinical development of cellular therapies and accreditation of facilities delivering cell therapies.

I am very honored to be elected to the National Academy of Medicine and want to acknowledge that this is based on team science with many superb collaborators in the Center for Cell and Gene Therapy at Baylor, Texas Childrens and Houston Methodist, Heslop said.

New academy members are elected by current members through a process that recognizes individuals who have made major contributions to the advancement of the medical sciences, health care and public health.

Heslop joins the following group of distinguished scientists from Baylor College of Medicine in the National Academy of Medicine:

Dr. Arthur L. BeaudetDr. Dennis M. BierDr. Malcolm K. BrennerDr. William R. BrinkleyDr. C. Thomas CaskeyDr. Mary K. EstesDr. Richard A. GibbsDr. Margaret A. GoodellDr. Vivian HoDr. Peter J. HotezDr. Brendan LeeDr. James R. LupskiDr. Bert W. OMalleyDr. Cheryl Lyn WalkerDr. Huda Y. Zoghbi

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Dr. Helen Heslop elected to the National Academy of Medicine - Baylor College of Medicine News

Boehringer Ingelheim has taken legal steps giving it the lead role in further development of a long-lasting, inhalation gene therapy for cystic fibrosis (CF), calledBI 3720931, that stems from a public-private research effort begun in 2018.

The 2018 agreement brought together Boehringer, the U.K. Cystic Fibrosis Gene Therapy Consortium (GTC; composed of university scientists), andOxford Biomedicain work to develop and conduct early tests on a potential first-in-class gene therapy for all with CF.

Now, Boehringer is exercising license options with these partners to acquire exclusive global rights to manufacture, register, and potentially market the therapy. Also involved is the IP Group, acting on behalf of the three U.K. universities in the GTC, Boehringer announced ina press release.

GTC will continue to contribute to research and testing, and Oxford Biomedica to the refinement andmanufacturing of lentiviral vectors stemming from its lentiviral vector delivery platform.

Since 2018, Boehringer Ingelheim has sponsored research and development activities with the GTC and [Oxford Biomedica]. The shared success achieved with our partners in this potentially revolutionary project makes us confident that we can now further accelerate this highly innovative therapeutic approach, Clive R. Wood, PhD, corporate senior vice president and global head of discovery research at Boehringer Ingelheim.

With our leadership in the discovery and development of therapies in respiratory diseases combined with the gene therapy and manufacturing knowledge of our partners, we aim to bring the next breakthrough to patients suffering from CF, who are desperately waiting for better options, Wood added.

Deliveredthrough a modified lentiviral vector (a virus that cannot replicate), BI 3720931 aims to carry a healthy copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is mutated in CF patients, to target cells. This could allow for greater production of the CFTR protein that is deficient in these people.

The therapy is being developed as an inhaled formulation that could make repeated administration possible. It also is designed to treat all CF patients, regardless of their specificCFTRmutations.

The novel lung-targeting technology we have developed has demonstrated high gene transfer efficiency in pre-clinical models and offers the possibility of repeated administration to maintain a therapeutic effect, a benefit that other viral-based gene therapies may not be able to provide, said Eric Alton, MD, coordinator of the GTC.

Our novel therapy has the potential to improve CFTR function and modify disease in all CF patients, independent of the more than 2,000 different known gene mutations, Alton added. The immediate target is those patients who are not eligible for CFTR modulators. The GTC is very excited to have reached this milestone after 21 years of focused effort.

Under the terms of the agreement, Boehringer will pay IP Group the intellectual property company working on behalf of the researchers with Imperial College London and the universities of Oxford and Edinburgh an undisclosed option exercise fee, plus future milestone payments and royalties based on potential sales. This option includes giving Boehringer rights to the lentiviral vector developed by GTC, the company reported in a related release.

Oxford Biomedica will receive an option exercise fee of 3.5 million (about $5 million), with additional payments of up to 27.5 million if various development, regulatory, and sales milestones are met.

We have enjoyed working with Boehringer Ingelheim, IP Group, and the GTC since 2018, said John Dawson, CEO of Oxford Biomedica. Building on the great progress made to date, we are delighted that Boehringer Ingelheim, one of the worlds leading respiratory medicine organizations, has chosen to exercise the option to license [Oxford Biomedicas] lentiviral vector manufacturing technology for this highly innovative inhaled cystic fibrosis gene therapy formulation developed by the GTC.

The partnerships past and continuing work, he added, has the potential to provide a new therapeutic option for many cystic fibrosis patients globally.

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Boehringer Acquires Rights to Potential CF Inhalation Gene Therapy - Cystic Fibrosis News Today

LEXINGTON, Mass., Oct. 21, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company, today is slated to present new preclinical data on its GeneRide platform at the European Society of Gene and Cell Therapy (ESGCT) Virtual Congress 2021, taking place October 19-22, 2021. The newly presented preclinical data further validate previous research in methylmalonic acidemia (MMA) and highlight selective advantage, a key feature of the GeneRide technology, in two additional indications characterized by intrinsic liver damage, hereditary tyrosinemia type 1 (HT1) and Wilson disease. Selective advantage enables edited hepatocytes carrying the corrective gene to survive and reproduce better than the endogenous mutated hepatocytes and to ultimately repopulate a part or whole of the diseased liver.

The data presented at ESGCT highlighted mouse models of the three liver indications treated with GeneRide vectors to deliver corrective genes. In all these models, expansion of the corrected healthy hepatocytes correlated with improved diseased markers.

In the HT1 models with acute liver damage, the data showed that GeneRide-corrected hepatocytes repopulated the entire liver within four weeks post-administration, replacing the diseased hepatocytes with corrected hepatocytes. HT1 mice are deficient in the gene encoding fumarylacetoacetate hydrolase (FAH), which is required to metabolize the amino acid tyrosine, resulting in the accumulation of toxic metabolites. HT1 mice that received the GeneRide-FAH vector were no longer reliant on the current standard of care for the disease, and demonstrated restored normal body growth, liver function, and undetectable succinyl acetone levels, one of the toxic metabolites that accumulates in patients with HT1. Compared to the current standard of care, treatment with the GeneRide vector resulted in superior succinyl acetone reduction and lower alfa-fetoprotein levels, a clinically validated biomarker for hepatocellular carcinoma and another risk factor for untreated HT1 patients.

Wilson disease results from a defect in copper transport, leading to toxic accumulation of copper and damage to tissues. In a Wilson disease mouse model, GeneRide-corrected hepatocytes repopulated the liver over time, and treated mice showed improvements in liver function, hepatomegaly, and urinary copper excretion.

"We are very excited to present these preclinical data in HT1 and Wilson disease. These data demonstrated repopulation of a diseased liver using our in vivo genome editing technology, resulting in GeneRide-edited corrected hepatocytes. The results in HT1 were particularly encouraging, demonstrating complete liver repopulation after treatment. These data further validate our technology and represent an important step as we continue on our mission to deliver the hope of genetic medicine to people impacted by devastating diseases," said Mariana Nacht, Ph.D., chief scientific officer of LogicBio.

Shengwen Zhang, director, molecular and cellular pharmacology at LogicBio, will give an oral presentation highlighting GeneRide's successful delivery of corrective genes in HT1, Wilson disease and MMA. Selective advantage and expansion of corrected hepatocytes was observed in these preclinical models, demonstrated by detection of increasing levels of a tagged albumin protein, albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression, as well as immunohistochemistry for the corrective protein in liver sections. Results also showed increasing levels of albumin-2A correlated with increased expression of the corrective gene and improved disease burden. The company believes that these data support the development of GeneRide vectors to durably treat multiple genetic diseases with liver dysfunction.

Additional posters presented at ESGCT highlight the Company's adeno-associated virus (AAV) technology platform advancements. One poster detailed the combination of LogicBio's proprietary plasmids and optimized transfection process in suspension HEK293 cells, which resulted in a 10- to 25-fold increase in titers using an LK03 capsid in 50L bioreactors. A separate poster highlighted recent development of anion exchange (AEX)-based high-pressure liquid chromatography, allowing LogicBio to use an analytical method to measure the percentage of full capsids in any given sample of AAV-LK03.

Additional information on the meeting can be found on the ESGCT website.

The oral presentation and posters will be made available on thePresentations section of the Company website at https://investor.logicbio.com/events-and-presentations/presentations.

AboutLogicBio Therapeutics

LogicBio Therapeuticsis a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The company's genome editing platform, GeneRide, is a new approach to precise gene insertion harnessing a cell's natural DNA repair process potentially leading to durable therapeutic protein expression levels. The company's gene delivery platform, sAAVy, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The company is based inLexington, MA.For more information, visitwww.logicbio.com, which does not form a part of this release.

Forward-Looking Statements

Statements in this press release regarding LogicBio's strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of theU.S.Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding validation of previous research; the potential of the GeneRide platform; and the company's belief that preclinical data supports the development of GeneRide vectors to durably treat multiple genetic diseases with liver dysfunction. The terms "believe," "validate" and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the risk that existing preclinical data may not be predictive of the results of ongoing or later preclinical and/or clinical results; the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators and vendors operate; manufacturing risks; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize product candidates, including for safety, tolerability, enrollment, manufacturing or economic reasons; the timing and content of decisions made by regulatory authorities; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape; changes in the economic and financial conditions of LogicBio; and LogicBio's ability to obtain, maintain and enforce patent and other intellectual property protection for LB-001 and any other product candidates. Other risks and uncertainties include those identified under the heading "Risk Factors" in LogicBio's Annual Report on Form 10-K for the year endedDecember 31, 2020and other filings that LogicBio may make with theU.S. Securities and Exchange Commissionin the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and LogicBio does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Investor Contacts: Laurence WattsGilmartin Group(619) 916-7620[emailprotected]

Stephen JasperGilmartin Group(858) 525-2047[emailprotected]

Media Contacts:Jenna UrbanBerry & Company Public RelationsW: 212-253-8881C: 203-218-9180[emailprotected]

Bill BerryBerry & Company Public RelationsW: 212-253-8881C: 917-846-3862[emailprotected]

SOURCE LogicBio Therapeutics, Inc.

https://www.logicbio.com

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LogicBio Therapeutics Announces Successful Repopulation of Diseased Livers in Mice with Healthy Corrected Hepatocytes in Two New Indications Using...

Dublin, Oct. 20, 2021 (GLOBE NEWSWIRE) -- The "Pharmaceutical Contract Development and Manufacturing Market (Pharmaceutical, Biologics, Active Pharma ingredients, tablet, Parenteral, Oral Liquid, Semi-Solids), End User (Big Pharma, Small Pharma, Generic Pharma, CRO)-Global Forecast to 2026" report has been added to ResearchAndMarkets.com's offering.

The global pharmaceutical contract development and manufacturing market is projected to reach USD 171.3 billion by 2026 from USD 120.6 billion in 2021, at a CAGR of 7.3%

Market growth is driven mainly by factors such as rising demand for generics, increasing investments in pharmaceutical R&D, and investments in advanced manufacturing technologies by CDMOs. The increasing demand for biological therapies, growing focus on specialty medicines, growth in the nuclear medicines sector, and advancements in cell and gene therapies are also expected to offer market growth opportunities in the coming years.

The small and medium-sized pharmaceutical companies segment accounted for the highest growth rate in the pharmaceutical contract development and manufacturing market, by end-user, during the forecast period

The pharmaceutical contract development and manufacturing market is segmented into big pharmaceutical companies, small & medium-sized pharmaceutical companies, generic pharmaceutical companies, and other end users. The small and medium-sized pharmaceutical companies segment accounted for the highest growth rate in the pharmaceutical contract development and manufacturing market in 2021. This segment's high growth can be attributed to the increasing number of emerging pharmaceutical companies that lack the in-house capabilities to manufacture and develop complex formulations and drug products.

Biologics manufacturing services segment accounted for the highest CAGR

Based on service, the pharmaceutical contract development and manufacturing market is segmented into pharmaceutical manufacturing, biologics manufacturing, and drug development services. In 2021, the biologics manufacturing services segment accounted for the highest growth rate. The major factor driving the growth of this segment is the growing demand for vaccines and biosimilars.

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Asia Pacific: The fastest-growing country in the pharmaceutical contract development and manufacturing market

The pharmaceutical contract development and manufacturing market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. Asia Pacific is projected to register the highest CAGR during the forecast period. This growth can be attributed to factors such as growth in the manufacturing sector, favorable government regulations, growing strategic expansions from leading companies, increasing emphasis on off-patent drugs, and the presence of a highly skilled workforce.

Competitive Landscape

The prominent players in this market are Thermo Fisher Scientific Inc. (US), Catalent, Inc. (US), Lonza Group Ltd. (Switzerland), Recipharm AB (Sweden), Vetter Pharma International GMBH (Germany), FAMAR Health Care Services (France), AbbVie Inc. (US), Aenova Group (Germany), Almac Group (UK), Siegfried Holding AG (Switzerland), Boehringer Ingelheim International GmbH (Germany), and Evonik Industries AG (Germany).

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Growing Demand for Generics is One of the Key Factors Driving Market Growth

Pharmaceutical Manufacturing Services to Command the Largest Share of the European Pharmaceutical Contract Development and Manufacturing Market

Small & Medium-Sized Pharmaceutical Companies to Grow at the Highest Rate During the Forecast Period

Developing Markets to Register Higher Growth Rates Between 2021 & 2026

Market Dynamics

Drivers

Patent Expiry and Increasing Demand for Generic Drugs

Increasing Investments in Pharmaceutical R&D

Investments in Advanced Manufacturing Technologies by Cdmos

Opportunities

Increasing Demand for Biological Therapies

Growth in the Nuclear Medicine Sector

Growing Demand for Cell and Gene Therapies

Challenges

Trends

Companies Mentioned

Abbvie

Aenova Group

Almac Group

Biovectra

Boehringer Ingelheim

Cambrex

Catalent

Cordenpharma

Curia (Formerly Albany Molecular Research)

Evonik Industries

Famar Health Care Services

Fujifilm Healthcare

Lonza Group

Piramal Pharma Solutions

Recipharm Ab

Samsung Biologics

Siegfried Holding Ag

Thermo Fisher Scientific Inc.

Vetter Pharma International

Wuxi Apptec (Wuxi Sta)

For more information about this report visit https://www.researchandmarkets.com/r/ajj3x3

Original post:
Global Pharmaceutical Contract Development and Manufacturing Markets, 2021-2026 - Increasing Demand for Biological & Cell and Gene Therapies /...

A commonly available oral diuretic pill approved by the U.S. Food and Drug Administration may be a potential candidate for an Alzheimers disease treatment for those who are at genetic risk, according to findings published inNature Aging.

The research included analysis showing that those whotook bumetanide a commonly used andpotent diuretic had a significantly lower prevalence of Alzheimers disease compared to those not taking the drug. The study, funded by the National Institute on Aging (NIA), part of the National Institutes of Health, advances aprecision medicineapproach for individuals at greater risk of the disease because of their genetic makeup.

The research team analyzed information in databases of brain tissue samples and FDA-approved drugs, performed mouse and human cell experiments and explored human population studies to identify bumetanide as a leading drug candidate that may potentially be repurposed to treat Alzheimers.

Though further tests and clinical trials are needed, this research underscores the value of big data-driven tactics combined with more traditional scientific approaches to identify existing FDA-approved drugs as candidates for drug repurposing to treat Alzheimers disease, said NIA Director Richard J. Hodes, M.D.

Knowing that one of the most significantgenetic risk factors for late-onset Alzheimers is a form of the apolipoprotein E gene called APOE4, researchers analyzed data derived from 213 brain tissue samples and identified the Alzheimers gene expression signatures, the levels to which genes are turned on or off, specific to APOE4 carriers. Next, they compared the APOE4-specific Alzheimers signatures against those of more than 1,300 known FDA-approved drugs.

Five drugs emerged with a gene expression signature that the researchers believed might help neutralize the disease. The strongest candidate wasbumetanide, which is used to treat fluid retention often caused by medical problems such as heart, kidney and liver disease.

The researchers validated the data-driven discoveries by testing bumetanide in both mouse models of Alzheimers andinduced pluripotent stem cell-derived human neurons. Researchers found that treating mice which expressed the human APOE4 gene reduced learning and memory deficits.

The neutralizing effects were also confirmed in the human cell-based models, which led to the hypothesis that people already taking bumetanide should have lower rates of Alzheimers. To test this, the team pared down electronic health record data sets from more than 5 million people to two groups: adults over 65 who took bumetanide and a matching group who did not take bumetanide. The analysis showed that those who had the genetic risk and took bumetanide had a ~35-percent to 75-percent lower prevalence of Alzheimers disease compared to those not taking the drug.

We know that Alzheimers disease will likely require specific types of treatments, perhaps multiple therapies, including some that may target an individuals unique genetic and disease characteristics much like cancer treatments that are available today, said Jean Yuan, M.D., Ph.D., Translational Bioinformatics and Drug Development program director in the NIA Division of Neuroscience. The data in this paper make a good case to conduct a proof-of-concept trial of bumetanide in people with genetic risk.

The research team was led by scientists at Gladstone Institutes, San Francisco, the University of California, San Francisco and the Icahn School of Medicine at Mount Sinai, New York City. This group is one of more than 20 teams supported by NIA through aprogramencouraging the researcher community to seek, through big data approaches, drugs that could potentially be repurposed.

The research was funded by NIH grants R01AG057683, R01AG048017, F31AG058439, R01AG061150, F31AG057150, R21TR001743 and K01ES028047.

NIA leads NIHs systematic planning, development and implementation ofresearch milestonesto achieve the goal of effectively treating and preventing Alzheimers and related dementias. This research is related to Milestone7.B, Initiate research programs for translational bioinformatics and network pharmacology to support rational drug repositioning and combination therapy from discovery through clinical development.

For more information about NIH and its programs, visitwww.nih.gov.

Original post:
Precision medicine data dive shows water pill may be viable to test as Alzheimer's treatment - The Highland County Press