New York, June 18, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy in Oncology - Thematic Research" - https://www.reportlinker.com/p06096403/?utm_source=GNW There are currently just 3 gene therapies marketed for oncology indications in the eight major pharmaceutical markets (8MM) (US, France, Germany, Italy, Spain, UK, Japan, and China).

Oncolytic viruses lead the category with 2 products, followed by viral vector gene therapies with 1 approved drug.

Gene therapies are in development for melanoma and other various solid tumors. As of April 2021, there are 252 clinical trials investigating in vivo gene therapies across the 8MM with 81 drugs in development.

Sales of products that comprise the categories of in vivo gene therapy are forecast to reach over $7B by 2027. The therapeutic oligonucleotide market, which will be galvanized by the success of COVID-19 messenger ribonucleic acid (mRNA) vaccines, is forecast to reach $4.5B by 2027 globally.

Key Highlights - The main drivers of growth include significant industrial investment in the field, as well as regulatory bodies recognition of gene therapy potential by creating specialized designations for gene therapy products. - The main barriers to growth include market access issues such as high cost of therapy/lack of reimbursement and lack of expertise as well as clinical issues such as safety concerns. - The most important unmet needs in the oncology gene therapy market include high cost of therapy, improving the safety of delivery systems and providing a valid treatment option to treat several cancer types. - The 8MM have set up programs to expedite gene therapy approvals, especially when they address an unmet need or are targeting orphan indications.

KEY QUESTIONS ANSWERED

- What are the late-stage gene therapy pipeline agents in development for cancer? Which class of gene therapy drugs will have the highest peak sales, and why? - What are the current unmet needs that gene therapies can address in the treatment of cancer? What are the opportunities for R&D? - How is the field of gene therapy going to move forward? Which technologies are the most promising for combinations? - What is the regulatory landscape for gene therapies in the US, the 5EU, Japan and China?

Scope - Overview of gene therapy including classification of therapy and technologies, regulatory and market access details, product & company profiles. - Quotes from US- and 5EU-based key opinion leaders and researchers. - Key topics covered for gene therapy in oncology in the 8MM include trends, value chain, market analysis, opportunities, challenges and unmet needs and high-value deals. - Pipeline analysis: Comprehensive data split across different phases and indications, emerging novel trends under development, and analysis of the most promising late-stage pipeline drugs for classes of gene therapy (Phase III and pre-registration). - Analysis of the key dynamics of the gene therapy market. Insightful review of the key industry drivers and challenges. Deals and R&D strategies covered in detail to highlight business opportunities.

Reasons to Buy The report will enable you to - - Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current deals and technologies to identify companies with the highest potential. - Develop business strategies by understanding the trends shaping and driving the global oncology gene therapy market. - Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global oncology gene therapy market in the future. - Design your development strategy through a review of potential novel targets or combinations across indications. - Understand the challenges and strategies impacting the development of oncology gene therapy agents in preclinical studies and clinical trials. - Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage. - Organize your sales and marketing efforts by identifying the classes of gene therapy and indications that present maximum opportunities for consolidations, investments and strategic partnerships.Read the full report: https://www.reportlinker.com/p06096403/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Gene Therapy in Oncology - Thematic Research - GlobeNewswire

Multipronged approach: New Rett syndrome therapies eye the same target from different angles.

Photograph by William Mebane

In theory, researchers know how to reverse many of the debilitating effects of Rett syndrome: Boost expression of the protein MECP2.

People with the condition typically have a mutated copy of the MECP2 gene and, as a result, deficient levels of its protein. This loss leads to intellectual disability, autism, seizures and trouble walking, talking and breathing.

But in practice, getting peoples cells to express the right level of MECP2 protein has proved complicated. Unlike with protein deficiencies in some other genetic syndromes, MECP2 levels must be carefully titrated: Too little causes Rett syndrome; too much also leads to autism, seizures and developmental delay.

The fact that MECP2 falls on the X chromosome throws up yet another curve ball for potential therapies: Most girls with Rett syndrome, who have two X chromosomes, have one mutated and one functional copy of the gene so half their cells already have sufficient levels of the protein. That creates a dosage problem: Ramping up MECP2 levels indiscriminately can overdose those cells in an attempt to treat the others. (Boys with mutations in MECP2 and only one copy of the gene rarely survive infancy.)

Traditional gene-replacement therapy which involves swapping out the mutated copy of MECP2 for a functional one has been successful in mice, but its not obvious how to safely translate that approach for people, says Matthew Lyst, research fellow in the School of Biological Sciences at the University of Edinburgh in Scotland.

For these reasons, a cadre of researchers is looking for alternative gene-therapy approaches, involving RNA or microRNA. These various treatments all take slightly different tacks to try to sidestep Retts dosage problem.

Its going to need a clever solution, Lyst says.

Photograph by William Mebane

One potential treatment targets RNA the single strands of genetic material that translate the DNA code to build MECP2 protein. In Rett syndrome, MECP2 mutations are repeated in the RNA, resulting in a faulty translation and no protein. But editing the RNA sequence can ensure that MECP2 still gets made.

One of the benefits of RNA editing is that, unlike gene editing, its potentially reversible, says John Sinnamon, research assistant professor at Oregon Health & Science University in Portland, who presented the work at the 2021 International Society for Autism Research (INSAR) annual meeting in May. Not every type of RNA mutation is amenable to editing, he says. Still, he and his colleagues have combined multiple techniques to target about 45 percent of the mutations seen in people with Rett syndrome.

One technique enabled Sinnamon and his colleagues to edit half of the MECP2 RNA in mice that carry a particular Rett mutation. It also increased MECP2 protein across multiple cell types, the team found.

This was a proof of principle that this type of editing works in an animals brain, Sinnamon says. The next step is to see [if we can] achieve sufficient levels of editing to see a behavioral effect, and whether there are any negative outcomes, he says.

A related approach called RNA transsplicing involves targeting the part of the RNA that carries the mutation and replacing it with a non-mutated version.

Like RNA editing, RNA transsplicing affects only those cells that carry a MECP2 mutation. But transsplicing also has the benefit of being able to target a greater diversity of Rett variants than RNA editing can, said Stuart Cobb, reader in neuroscience and a research fellow at the University of Edinburgh, who presented the work at INSAR.

Newborn wildtype mice injected with a virus carrying an RNA transsplicing treatment show transspliced RNA throughout the brain, Cobb and his colleagues have found, suggesting the approach is effective.

At the moment, the process remains inefficient for altering MECP2 RNA, Cobb said during his talk, but the team is working on finding better target locations for delivering the corrected bit of gene sequence.

Taysha Gene Therapies is using yet another strategy to control dosing. They are building on standard gene-replacement therapy, adding in a mechanism that regulates MECP2 expression.

The companys new treatment, called TSHA-102, takes advantage of microRNAs short strands of genetic material to tamp down production of MECP2 protein before it gets too high, said Suyash Prasad, chief medical officer and head of research and development for the company, during an INSAR presentation.

The treatment uses a virus to carry a functional copy of MECP2 into a cells nucleus, as well as an untranslated sequence that binds the microRNAs and slows production of the protein when it increases too much. As a result, each cells MECP2 levels remain within the Goldilocks range, Prasad said: Not too high, not too low.

Wildtype mice that received this gene-therapy treatment did not have an increased risk of premature death or show any adverse behavioral effects, whereas 4 out of 10 wildtype mice that received a therapy that increased MECP2 levels indiscriminately died prematurely.

And mice that have no MECP2 in the brain usually die at 10 to 12 weeks of age, but those treated with Tayshas gene therapy survive another 5 weeks, the team found.

As with any gene therapy, these approaches carry risks, such as adverse immune reactions and long-term damage to DNA. Such outcomes can block people from participating in later trials. And as the science advances, researchers, clinicians, families and regulatory agencies will need to evaluate if those risks are worth taking.

One way to understand how the families of people with Rett syndrome would weigh risks and benefits is to ask, as Taysha did in focus groups. Caregivers have the most concern about their childrens inability to speak, gastrointestinal problems and breathing difficulties, suggesting that they would especially value a treatment that could improve these traits. The company plans to use this feedback to inform the upcoming clinical trial, Prasad said.

Ideally, TSHA-102, or the next treatment that makes it to trial, will elevate MECP2 to ideal levels and ease all Rett traits. But getting to those levels in all cells may not be possible or necessary, Sinnamon said at INSAR.

In mice, any little increase in the amount of MECP2 re-expression seems to be beneficial to the animals, and small improvements could also have real benefits for people with Rett syndrome, he said.

One promising fact for any potential Rett therapy is that neurons lacking MECP2 dont die they just dont function typically. That means that a successful treatment has the potential to not just slow the progression of Rett traits, but to reverse them altogether. And it also lessens the pressure to intervene as early as possible to make a difference a pressure that exists for many neurodevelopmental conditions.

Until clinical trials are underway, its unclear how much MECP2 needs to be expressed, and in how many cells, for the improvements to be worth the risks, the researchers say.

Of the alternative therapies discussed, TSHA-102 is furthest along in the pipeline; the company plans to begin clinical trials later this year, Prasad said. Researchers working on the other approaches say it is difficult to predict when their therapies might be at that stage they still need to fine-tune the techniques in mice.

But, Sinnamon says, theres only so far that animal models can take you.

Read the original here:
Alternative gene-therapy approaches take aim at Rett syndrome - Spectrum

Jesse Owens was just 17 when he decided to devote his life to gene therapy research.

Now the youngest full-time faculty member at John A. Burns School of Medicines Institute for Biogenesis Research, Owens who grew up in Keaukaha is working to make gene therapy safer with the help of funding from the National Institutes of Health.

During his senior year of high school, the 2003 Hilo High graduate, now 36, sailed around the world, taking classes aboard a sailboat as part of a study program.

It was then that Owens became interested in the idea of adding new genes into a genome.

I was really interested in the idea that you could deliver a gene into an animal, whether that be a mouse, a pig or a person. I thought that was really cool, something that could be a very powerful tool.

He received his bachelors degree in molecular, cellular and developmental biology from University of California, Santa Cruz, and earned a doctorate in cell and molecular biology from the University of Hawaii at Manoa in 2014.

At UH-Manoa, he was able to work with Dr. Stefan Moisyadi, a specialist in transgenic mice.

So from there, I kind of refocused my goal into making it so when you deliver a gene into a person or organism, the gene goes in safely and not randomly, he said. It goes into a specific location.

Owens recently was awarded $2.3 million over the next five years from the NIH to develop a new and safer tool for gene therapy.

Gene therapy delivers a gene into a person to replace a mutated or broken gene to cure genetic disease, Owens explained.

According to a UH news release, the grant addresses drawbacks to current genome editing technologies that randomly insert a therapeutic gene.

Sometimes, that randomly inserted gene might disrupt other genes, which could cause cancer, Owens explained.

Currently, the process is not thought of as very safe and is only used on diseases that are terminal, he said, but one of his major goals is to make gene therapy less risky, then use gene therapy to treat more diseases.

By the end of the five years, Owens said he hopes to have developed a new tool to deliver large pieces of DNA to a specific location in the genome and nowhere else.

The genome we have is able to make every single protein in our body, he said. So the ability to bring in new pieces of DNA gives us a lot of power because we are able to potentially correct any genetic disease. So were not talking about curing one disease with this tool. You might be able to cure all genetic diseases. So by making a tool, you kind of have a broader impact than if I studied one disease.

Owens shares his alma mater with biochemist Jennifer Doudna, a 1981 Hilo High graduate and a professor at the University of California, Berkeley, who along with her research partner, Emmanuelle Charpentier, a French microbiologist, received the 2020 Nobel Prize in chemistry for their development of the CRISPR-Cas9 gene-editing tool.

I feel like people in Hilo often dont think they have that many opportunities, and I think that Jennifer Doudna is an example of why thats not true, Owens said.

Not only is he faculty here, he got his (doctorate) here, he was trained here, he was raised on the Big Island, Dr. Steven Ward, director of the UH Institute for Biogenesis Research, said in the news release. Hes the product of that, and it just shows you that Hawaii can do some of the worlds greatest biomedical research.

In addition to the NIH funding, Owens recently signed a sponsored research agreement with a private company called SalioGen Therapeutics that is specializing in nonviral gene therapies, UH said. The goal of this collaboration is to advance the tools Owens is developing in the lab into clinical-stage gene therapy candidates.

Owens is the son of David Owens of Keaukaha and Peach McGuffey of Puna.

Email Stephanie Salmons at ssalmons@hawaiitribune-herald.com.

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Big Islands Owens gets $2.3M grant for genetic therapy research - West Hawaii Today

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Holdings plc (Gyroscope), a clinical-stage gene therapy company focused on treating diseases of the eye, today announced the company has entered a research collaboration with Childrens Medical Research Institute (CMRI) in Australia to develop next-generation clinical capsids, the protein shells of viral vectors used to deliver gene therapies.

A team of researchers from CMRI and Gyroscope will work together in the design and screening of capsid libraries to identify novel capsids for enhanced delivery of ocular gene therapies. Under the agreement, Gyroscope has an option to obtain an exclusive licence for ocular uses of capsids developed through the partnership. The CMRI team is led by Associate Professor Leszek Lisowski, Ph.D., MBA, a recognised expert in viral vector-based gene therapy, vectorology and genotoxicity, with more than 15 years of experience in capsid generation and discovery.

Capsids are one of the most critical components of a gene therapy, however, there are some limitations with the capsids available today, said Jane Hughes, Ph.D., Chief Scientific Officer, Gyroscope. We are excited to collaborate with Associate Professor Lisowksi and the team at CMRI to engineer next- generation capsids supporting our goal of developing a pipeline of differentiated ocular gene therapies that have the potential to be administered in the convenience of a doctors office.

Gene therapies are being studied in many diseases of the eye and capsids play an important role in maximising the potential benefit of these therapies for patients, said Dr. Lisowski. We look forward to working with the team at Gyroscope to identify novel capsids that may improve upon the current standard for gene therapies for treatment of diseases of the eye.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. Our lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), a leading cause of blindness. GT005 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of people with GA.

Supported by leading life sciences investors, Gyroscope has built a global organisation combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness.

For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

About Childrens Medical Research Institute

Childrens Medical Research Institute (CMRI) pioneered microsurgery, immunisations against lethal childhood illnesses and care for premature babies, all of which has improved the lives of countless Australian children over the last 61 years. Today, CMRI is an independent institute and the site of world-leading research in the areas of cancer, neurobiology, embryology, proteogenomics and gene therapy. CMRI is affiliated with the University of Sydney and is a founding partner of Luminesce Alliance and the Westmead Research Hub.

CMRIs scientists undertake research collaborations worldwide. CMRI is also home to high- technology research facilities available to researchers and organisations across Australia and internationally. It operates CellBank Australia, the only national repository of cell cultures in Australia; is home to advanced gene therapy and gene engineering facilities--creating treatments of the future. In addition, CMRI houses two Australian Cancer Research Foundation Centres, including the ACRF Telomere Analysis Centre and ProCan, which combined aim to substantially increase understanding about the biology of cancer and to produce technologies to improve the diagnosis and treatment of all types of cancer.

CMRIs achievements are made possible by a network of devoted community supporters, and via the iconic Jeans for Genes fundraising campaign. For more information visit: http://www.cmrijeansforgenes.org.au.

Original post:
Gyroscope Therapeutics Announces Research Collaboration Agreement with Children's Medical Research Institute to Develop Novel Gene Therapy Capsids -...

ARLINGTON, Va., June 18, 2021 /PRNewswire/ --Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, announces the publication of Cell and Gene Therapy Business Outlook.

"With thousands of potential therapies on the market, everyone knows there is potential," said Bruce Carlson, Senior VP of Publications for Science and Medicine Group. "The question that will continue to be asked is - how much money are companies making selling cell and gene therapy products, and what will be the future potential?"

This new twice-monthly publication dedicated to cell and gene therapy, Cell and Gene Therapy Business Outlook offers the following:

Unique features of this new newsletter include market analysis in each issue and a table that tracks important deals in the cell and gene industry.

"We'll handle science, we'll explain it, we'll inform on important developments," Carlson said. "But we'll focus on that science that is near-term relevant."

Edited by Blake Middleton, a professional CGT researcher and former Staff Research Associate at UCLA Department of Pharmacology, Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. Included is news that could affect business decisions near-term. Cell and Gene Therapy Business Outlook also explains the relevant science behind partnerships, product launches and business deals.

Science and Medicine Group also publishes the bimonthly Instrument Business Outlook newsletter.

Information is available at: http://www.cellandgenetherapybusinessoutlook.com

About Science and Medicine GroupScience and Medicine Group supports companies seeking to commercialize products for the rapidly changing marketplace at the intersection of science, medicine and technology. Comprised of industry leading brands, Science and Medicine Group serves analytical instrument, life science, imaging and clinical diagnostic companies by helping them create strategies and products to win markets and provide platforms to digitally engage their markets through a variety of innovative solutions. Our clients, including some of the top Fortune 500 companies in the world, use our business intelligence, market research, publications and marketing platforms to grow their businesses globally.

SOURCE Science and Medicine Group Inc

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New Cell and Gene Therapy Business Outlook Newsletter Launched - PRNewswire

Nuevocor wants to restore cardiac function to diseased hearts through gene therapy and now has the backing of Boehringer Ingelheim through a $24 million series A.

The preclinical biotech's lead candidate is an adeno-associated virus-based gene therapy for patients with dilated cardiomyopathy caused by mutations in the lamin A/C gene.

Dilated cardiomyopathy causes the hearts main pumping chamber to stretch and expand, limiting the hearts ability to pump blood. The condition is often undetectedbut can cause irregular heartbeats, blood clots or sudden death, according to the Mayo Clinic. Patients with the lamin A/C mutation have an increased risk of irregular heartbeats and sudden cardiac arrest and are not eligible for conventional gene therapies.

RELATED: Boehringer more than doubles VC fund with boosted focus, reach

The current standard of care for dilated cardiomyopathy only serves to delay disease progression, and the only cure is to have a heart transplant, Nuevocor CEO Dr. Yann Chong Tan said in a statement. At Nuevocor, we hope to give patients a new lease of life through our technology."

Singapore-based Nuevocor also plans to apply itsnovel target discovery platformto other untreatable cardiomyopathies.

The series A round was led by EVX Ventures and Boehringer Ingelheim Venture Fund (BIVF). Other investors were EDBI, Xora Innovation and SEEDS Capital.

EVX Ventures Chairman XQ Lin said Nuevocor is the latest biotech to emerge from the firms venture creation ecosystem, which also includes Carmine Therapeutics. The firm also invests in other companies, such as the recently relaunchedAltruBio.

RELATED: New name, new board, new focus: AltruBio relaunches with $63M series A

BIVF has invested in 45 companies, including three that have gone on to be acquired by Boehringer Ingelheim, the largest private pharmaceutical company and ownerof the venture fund.

"Nuevocor is trying to apply a very innovative and challenging approach using gene therapy for the treatment of genetically-driven, dilated cardiomyopathies, said Dr. Weiyi Zhang, Managing Director of BIVF Asia. We are excited to see that the company is exploiting the potential of genetic suppressors to change the course of these diseases and hopefully to significantly extend the life span and improve the life quality of DCM patients.

See the rest here:
Backed by Boehringer Ingelheim fund, Nuevocor snags $24M to tackle diseased hearts with gene therapy - FierceBiotech

SINGAPORE, June 18, 2021 /PRNewswire/ -- Nuevocor, a preclinical-stage biotech company specializing in gene therapy for cardiomyopathies, has announced the completion of an oversubscribed $24 million Series A financing round. The round was co-led by EVX Ventures and Boehringer Ingelheim Venture Fund (BIVF), with EDBI, Xora Innovation, SEEDS Capital and other investors joining the syndicate.

The funds will be used to accelerate the preclinical development of its lead programme, an adeno-associated virus (AAV) based gene therapy for patients suffering from dilated cardiomyopathy (DCM) due to mutations in the lamin A/C (LMNA) gene. The company also plans to address other untreatable cardiomyopathies using their novel target discovery platform.

Nuevocor's founding CEO, Dr Yann Chong Tan stated: "We aredelightedto have this strong group of investors join us in developing gene therapy-based treatmentsthat have the potential to restore cardiac function in diseased hearts. The current standard of care fordilated cardiomyopathyonly serves to delay disease progression, and the only cure is to have a heart transplant. AtNuevocor, wehopeto givepatients a new lease of lifethrough our technology."

XQ Lin, Chairman of EVX Ventures, commented, "We are thrilled to co-lead Nuevocor's Series A round alongside other top tier investors. Nuevocor is the latest company to emerge from our venture creation ecosystem. We look forward to supporting Nuevocor's path towards the clinic and bringing new medicines to patients in need."

"Nuevocor is trying to apply a very innovative and challenging approach using gene therapy for the treatment of genetically-driven, dilated cardiomyopathies. We are excited to see that the company is exploiting the potential of genetic suppressors to change the course of these diseases and hopefully to significantly extend the life span and improve the life quality of DCM patients," said Dr. Weiyi Zhang, Managing Director of BIVF Asia.

Headquartered in Singapore, Nuevocor isa privately held preclinical-stage biopharmaceutical company focused ondeveloping AAV gene therapy for the treatment of genetically defined cardiac diseases with high unmet need. Nuevocor's lead program, lamin A/C dilated cardiomyopathy (LMNA DCM) gene therapy is based on decades of research into fundamental mechanisms underlying LMNA-associated disease enabled by development of novel technologies on the part of scientific co-founders Colin Stewart and Brian Burkeof the Agency for Science, Technology and Research (A*STAR). Together with AAV gene therapy and cardiac disease modelling expertise of scientific co-founders Mark Kay from Stanford University and Jianming Jiang from the National University of Singapore respectively, Nuevocor is well-positioned to treat LMNA and other geneticcardiomyopathies, amongst other cardiac diseases.

"Singapore has made long-term R&D investments to drive health outcomes and economic growth, and to build the local biotech ecosystem. A*STAR is proud to have supported our spin-off Nuevocor in translating excellent science from bench to bedside so they can develop more effective gene therapies for hard-to-treat cardiac diseases, for better patient outcomes," said Professor Ng Huck Hui, Assistant Chief Executive, Biomedical Research Council, A*STAR.

About LMNAdilated cardiomyopathy

LMNA mutations are the #2 cause of familial DCM, affecting approximately 60,000 people in the US and EU alone. The mutation confers amongst the worst prognosis of all DCM, with increased risk of arrhythmogenic DCM and sudden cardiac death. Being an autosomal dominant disease characterized by gain-of-function of the mutant Lmnaprotein, conventional gene replacement therapies would be ineffective for LMNA DCM.Nuevocor'sinnovative approach to gene therapy circumvents this roadblock.

About EVX Ventures

EVX Ventures is a global VC that builds, incubates, and invests in biotech companies. With a focus on disruptive therapeutics platform technologies and novel therapeutic modalities, they invest in global technologies to redefine the therapeutics of tomorrow. Learn more at http://www.evx.ventures

About Boehringer Ingelheim Venture Fund

Created in 2010, the Boehringer Ingelheim Venture Fund GmbH (BIVF) invests in groundbreaking therapeutics-focused biotechnology companies to drive innovation in biomedical research. BIVF is searching for significant enhancements in patient care through pioneering science and its clinical translation by building long-term relationships with scientists and entrepreneurs. BIVF's focus is to target unprecedented therapeutic concepts addressing high medical needs in immuno-oncology, regenerative medicine, infectious diseases and digital health. For more information, visit http://www.boehringer-ingelheim-venture.com.

Contact:[emailprotected]

SOURCE Nuevocor

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Nuevocor closes US$24M Series A Financing to Advance Novel Gene Therapies for Cardiomyopathies - PRNewswire

DUBLIN, June 21, 2021 /PRNewswire/ -- 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 Boom1.1 Recent CMO/CDMO Expansions in Cell and Gene Therapy Sector1.1.1 Expected Capacity Crunch1.1.2 Investments for CGT Capacity Expansion1.1.3 Other CGT Manufacturing Investments1.1.4 Events Driving Expansion for CGT Facilities and Infrastructure

2. Recent Merger and Acquisition (M&A) Deals within the Cell and Gene Therapy Sector2.1 Charles River Laboratories International/Retrogenix2.2 Charles River Laboratories International/Cognate BioServices2.3 Beam Therapeutics/Guide Therapeutics2.4 Thermo Fisher Scientific/Henogen2.5 Eli Lilly/Prevail Therapeutics2.6 Novartis/Vedere Bio2.7 Bayer/Asklepios2.8 AGC Biologics/MolMed2.9 Sorrento Therapeutics/SmartPharm2.10 Catalent/MaSTherCell2.11 ElevateBio2.12 Ixaka

3. Financing Rounds3.1 Major Private Placements3.1.1 Orchard Therapeutics3.1.2 Amryt3.1.3 BioNTech3.1.4 Kiadis Pharma3.2 Major Venture Capital Financings3.2.1 ElevateBio3.2.2 Century Therapeutics3.2.3 Artiva Biotherapeutics, Inc.3.2.4 Vineti, Inc.3.2.5 Ori Biotech Ltd.3.2.6 Neogene Therapeutics3.2.7 Forge Biologics3.2.8 Sana Biotechnology3.2.9 Orca Bio3.2.10 Freeline Therapeutics3.2.11 Poseida Therapeutics3.2.12 Kriya Therapeutics3.2.13 Legend Biotech Corporation3.2.14 Lyell Immunopharma3.2.15 Mammoth Biosciences3.2.16 Kyverna Therapeutics3.3 Major Corporate Partnerships within the CGT Market3.3.1 Bayer/Atara Biotherapeutics3.3.2 Novartis/Mesoblast3.3.3 Novartis/Sangamo3.3.4 Janssen Biotech/Fate Therapeutics3.3.5 Biogen/Sangamo3.4 Follow-on Public Offerings by CGT Companies3.4.1 Rocket Pharmaceuticals3.4.2 Adaptimmune Therapeutics3.4.3 Allogene Therapeutics3.4.4 Iovance Biotherapeutics, Inc.3.4.5 Bluebird bio3.5 Initial Public Offerings (IPO)3.5.1 Legend Biotech3.5.2 Decibel Therapeutics3.5.3 Sana Biotechnology3.5.4 Rocket Pharmaceuticals3.5.5 JW Therapeutics3.5.6 AlloVir3.5.7 Akouos3.5.8 Generation Bio3.5.9 Passage Bio3.5.10 Beam Therapeutics

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

5. Profiles of Cell and Gene Therapy CMOs and CDMOs5.1 3P Biopharmaceuticals5.2 ABL, Inc.5.3 AGC Biologics5.4 Advent BioServices Ltd.5.5 Akron Biotech5.6 Aldevron5.7 Anemocyte S.r.l5.8 Applied Viromics5.9 ATVIO Biotech, Ltd./Orgenesis Biotech Israel, Ltd.5.10 Austrianova5.11 Avid Bioservices, Inc.5.12 Batavia Biosciences B.V.5.13 Bio Elpida5.14 BioCentriq5.15 BioNTech IMFS GmbH5.16 BioReliance Corporation/Merck Millipore5.17 Bio-Techne5.18 Biovian Oy5.19 Boehringer Ingelheim BioXcellence5.20 Brammer Bio/Thermo Fisher Scientific5.21 C3i5.22 Catalent Biologics5.23 CATAPULT5.24 CCRM5.25 Cell Therapies Pty Ltd.5.26 CELLforCURE (Novartis)5.27 Celonic AG5.28 Cellular Therapeutics Ltd.5.29 Center for Breakthrough Medicines5.30 Charles River Laboratories International, Inc.5.31 Cobra Biologics5.32 Cognate BioServices5.33 Delphi Genetics S.A.5.34 ElevateBio5.35 Emergent BioSolutions5.36 Eurogentec5.37 Exothera5.38 FinVector5.39 Flash Therapeutics5.40 Fraunhofer Institute for Cell Therapy and Immunology5.41 FUJIFILM Cellular Dynamics, Inc.5.42 FUJIFILM Diosynth Biotechnologies5.43 GenScript Biotech Corporation5.44 KBI Biopharma5.45 Lonza Group Ltd.5.45.2 Services5.47 Matica Biotechnology, Inc.5.48 Medinet Co., Ltd.5.49 Minaris Regenerative Medicine, LLC5.50 MolMed S.p.A5.51 NECSTGEN5.52 NEOBIOSIS, LLC5.53 NIKON CeLL Innovation Co., Ltd.5.54 Ology Bioservices, Inc.5.55 OrganaBio5.56 Orgenesis5.57 OxfordBiomedica plc5.58 Patheon/Thermo Fisher Scientific5.59 Performance Cell Manufacturing5.60 Q-Gen Cell Therapeutics5.61 RoslinCT5.62 Samsung Biologics5.63 SK biotek5.64 Stanford Laboratory for Cell and Gene Medicine5.65 Stemmatters, Biotechnologia e Medicina Regenerativa S.A.5.66 Takara Bio Europe SAS5.67 TUM Cells5.68 Interdisciplinary Stem Cell Institute/Miami University5.69 Upstate Stem Cell cGMP Facility5.70 VGXI, Inc.5.71 Vigene Biosciences, Inc.5.72 Waisman Biomanufacturing5.73 Yposkesi

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Global Cell and Gene Therapy Manufacturing Market Report 2021-2025: Expected Capacity Crunch, Investments for CGT Capacity Expansion, Events Driving...

MENLO PARK, Calif.--(BUSINESS WIRE)--Grace Science, LLC, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GS-100, an investigational AAV9 gene replacement therapy for the treatment of NGLY1 deficiency.

The FDA grants orphan drug designation to investigational medicines and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare disease or conditions affecting fewer than 200,000 people in the United States.

Orphan drug designation provides certain benefits and incentives to Grace Science for developing a gene therapy for NGLY1 deficiency, including seven years of U.S. marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain clinical research costs, eligibility for orphan drug grants, and a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees.

NGLY1 deficiency is an ultra-rare genetic disease caused by loss of function mutations in the NGLY1 gene. The disease primarily affects children and manifests as a complex neurological syndrome that includes global developmental delay, cognitive impairment, movement disorders, abnormal tear production (alacrima), as well as other neurological symptoms. There is currently no approved therapy for NGLY1 deficiency.

Matt Wilsey, founder and CEO of Grace Science stated: We are pleased the FDA has granted orphan drug designation to our gene therapy program for NGLY1 deficiency. This action highlights the significant need that exists for the development of safe and effective treatments for children suffering from this condition, including my own daughter, Grace. Our recent interactions with the FDA have been highly productive and we are making rapid progress on our plans to file an investigational new drug application (IND) in the third quarter of Q3 2022, which would allow us to initiate our first clinical trial by the end of next year.

Grace Science recently closed its Series A financing, which will fund the advancement of GS-100 into a first-in-human clinical trial for NGLY1 deficiency. The viral vector development and clinical manufacturing services for GS-100 will be provided by Thermo Fisher Scientific at its site in Alachua, FL.

About Grace Science

Grace Science, LLC is a biotechnology company founded in 2017 to develop novel therapies based on the function of NGLY1, a key enzyme involved in proteostasis. The companys deep knowledge of the NGLY1 pathway is key to its ability to identify new ways of treating NGLY1 deficiency as well as more common diseases. For more information about Grace Science, LLC and NGLY1 deficiency, please visit gracescience.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210622005487/en/

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Grace Science Receives US FDA Orphan Drug Designation for GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency - BioSpace

Cell and gene therapies (CGTs), also classified as Advanced Therapy Medicinal Products (ATMPs), have been hailed as revolutionary, but the path towards realising their potential has also seen pitfalls and disappointments along the way since the first attempts some decades ago.

Today, CGTs offer new horizons for people living with some rare, lifelong diseases. However, despite their promise, these therapies entail a range of scientific, logistic, regulatory and economic challenges. There is a clear need for inclusive dialogue to define a way forward in ensuring safe and affordable access to these novel treatments.

The current landscape: hopes and hurdles

In several disease areas, CGTs already exist and have transformed patients lives. Speaking at a debate convened by the European Health Forum Gastein and CSL Behring on 15th of June, Avril Daly (EURORDIS) stressed that we often think about CGTs as futuristic products, while they are already being administered in the now.

Thus, a main objective should be providing access for patients and their families and turning hope into reality. Bertalan Mesk (Medical Futurist Institute) reinforced this notion by urging to make patients the point of care and involve them at the highest level of decision-making.

However, where there are expectations and hope, there are also concerns and uncertainties.

Speaking in a personal capacity, Ilona G. Reischl (Austrian Medicines and Medical Devices Agency), commented that she sees a trivialisation of CGTs in Europe: we are discussing complex medicinal products and CGTs should be treated as such, she emphasised, underlining that there is a need for appropriate regulation and for clinical trials in order to provide product specific information.

It is also crucial to collect real-world data to help answer important questions about the durability of treatment responses to CGTs and to inform novel payment approaches for potentially curative, one-off therapies.

Further, health systems must adapt to accommodate this paradigm shift in care as they have done previously with ground-breaking, disruptive medical innovations.

Pooling expertise, setting up clinical frameworks and providing the means and infrastructures to enable the specialist care needed to deliver CGTs is now top of the agenda.

Equally important is ensuring appropriate infrastructure and processes to access diagnosis and genetic testing, as a first step for patients in receiving these transformative therapies.

However, time is of the essence for patients with rare diseases lacking treatment options or with poor quality of life. Opportunities to provide therapies that save, change or enhance these patients lives cannot be missed.

What may be seen as a small improvement can in fact have a huge impact of patients quality of lives, and the way we measure improvements need to change to reflect properly patients experiences and expertise, noted Avril Daly.

If indeed CGTs will transform tomorrows healthcare paradigm, we are not far from tomorrow, explained Dan Hart (Royal London Hospital Haemophilia Centre). The scientists have done the work, and it is now up to us to overcome some of the ethical implications linked with ensuring equal access across Europe.

Several EU policy initiatives offer possibilities to define a European policy and regulatory environment that addresses current challenges and is future-proof with respect to embracing medical innovation and ensuring patient access while maintaining European leadership in this field.

These include the upcoming EU Pharmaceutical Strategy, the European Health Data Space (EHDS), and the evaluation/revision of existing legislation including the EU Cross-border Healthcare Directive and the EU Blood, Tissues and Cells Directive. Andrzej Ry (European Commission Directorate General for Health and Food Safety), stressed the need for continuity and for legislation that is adaptable and reduces administrative burden.

Alignment between the relevant EU legislation and between European and national frameworks will be paramount, said Ilona G. Reischl, including rules on hospital exemption (HE) which allow for the use of an ATMP without a marketing authorisation under certain circumstances.

The willingness of all to realise ATMPs is there, and the stars are aligned, said Ry, while MEP Cristian Silviu Buoi outlined that by 2025 the European Medicines Agency shall approve 10-20 CGTs annually.

Paige Bischoff (Alliance for Regenerative Medicine) reflected on the fact that the EU Pharmaceutical Strategy refers to ATMPs as a major milestone of innovation. What is key now is greater harmonisation across Europe to support the Strategy, especially in legislation regulating genetically modified organisms (GMOs).

The EHFG debate revealed a consensus that allowing creativity and flexibility at all levels can help provide an environment where patients can best benefit from CGT. For example, legislative frameworks need to be adaptable to innovation, and COVID-19 served as an example of how to adapt quickly to change.

From a pricing and reimbursement perspective, new payment models are needed to reflect the novel features of CGTs. One size will not fit all, as every CGT is different. Diane Kleinermans (Belgian National Institute for Health and Disability Insurance) highlighted that thinking out of the box will be important to answer to the needs inherent to new technologies and therapies.

Further, international cooperation is the way forward, and the BeNeLuxA initiative is one example of this, she explained, as a platform for collaboration between five countries to monitor the whole life spectrum of medicines, organise joint Health Technology Assessments and conduct horizon scanning.

EU-level support for the continuous monitoring of quality and harmonised data, including via the EHDS, will be a building block to guarantee patient access to safe and effective CGTs across Europe and to address burning questions such as how safe is safe enough, or when health systems should pay for treatment given uncertainties in the durability of patient responses.

Reducing fragmentation, standardising data across borders and the increased use of real-world evidence will be key to supporting the acquisition of longer-term data, Bischoff asserted.

Cooperation between medical experts, policymakers, regulators, hospitals and academic developers, patients, health technology assessors, payers and industry will be decisive in transforming healthcare. Advancing CGTs requires collaboration as well as patient-centricity, working with and for patients.

The field of CGTs is deeply complex, as outlined by Vivienne Parry, science writer and broadcaster, and facilitator of the debate but the fact is that CGTs are real and being administered now.

They disrupt traditional ways of providing healthcare, of developing, assessing and paying for treatments, and challenge us all to rethink existing policies and practices or, as Bertalan Mesk pointed out at the beginning of the debate, to shift mindsets to incorporate change, as technological adoption needs to be accompanied by cultural adoption.

This article reflects on a more extensive discussion that took place during the webinar Transforming the future of healthcare do cell and gene therapies hold the key?, organised by the European Health Forum Gastein and CSL Behring on 15th June 2021. Ilona Reischl and Diane Kleinermans spoke in a personal capacity during this meeting. To watch the recording of this webinar, please visit https://www.youtube.com/watch?v=QL6OYmoJIzg

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Transforming healthcare: do cell and gene therapies hold the key? - EURACTIV