Archive for the ‘Gene Therapy Research’ Category

Gene therapies have yielded promising results for individuals experiencing rare diseases. However, these groundbreaking therapies come with their own unique set of challenges regarding who will be able to access them, how much they will cost, and how the policymaking and scientific processes will conflict as more and more therapies undergo clinical trials.

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Last week, we convened a panel of experts to address these questions and discuss potential solutions in our latest 5 Slides Were Watching conversation, led by State of Reforms DJ Wilson. The panel featured Danny Seiden, president & CEO of the Arizona Chamber of Commerce and Industry, Dr. Jennifer Hodge, U.S. DMD Gene Therapy Lead at Pfizer, Dr. Rafael Fonseca, chief innovation officer at Mayo Clinic, and Dr. Sharon Hesterlee, chief research officer at the Muscular Dystrophy Association.

Hesterlee brought a slide showing the prevalence of rare diseases in Arizona, noting that 5,500 Arizonans were estimated to be living with rare genetic neuromuscular diseases that were potentially treatable with gene therapy. She highlighted that Charcot-Marie-Tooth disease and Myotonic dystrophy were the most prominent, and that both diseases currently have gene therapy treatments in preclinical development.

She emphasized that ethics need to be an important part of the conversation, and that it will be critical to educate patients and families about the treatments irreversible implications as more and more therapies begin to launch.

Its a permanent change to someone. What we see in particular with parents of a child who has a pediatric disease, they are put in a very difficult position because they have to make a decision without always understanding all of the science and all of the implications.

So I think there is a huge requirement for the physician [who does the informed consent] to be very clear, and then the parents have to decide if it doesnt work, my child cannot be redosed, my child may not be eligible for another trial I think thats been a big challenge and something that weve tried to help our community in the neuro-muscular disease space navigate.

Seiden brought a slide displaying the economic benefits that would come with the increased prevalence of gene therapies. He noted that outdated systems of payment would not be applicable to this kind of treatment, and that these therapies would allow for one-time costs as opposed to a lifetime of treatment for patients with rare diseases.

When you deal with rare diseases, you need to look at it on an annualized basis over the cost of a lifetime, because gene therapy has the potential to save money and a lot of heartache for the patients and the families involved with it Arizona is one of a handful of states that allows for value-based purchasing when it comes to Medicaid contracts With the [Arizona Health Care Cost Containment System (AHCCCS)], which is by far the largest provider, theyve recognized that you have to look at patient outcomes. Its not just about that initial upfront cost.

Hodge presented a slide illustrating the unmet needs of individuals with rare diseases and the potential impacts that gene therapies can have on these individuals. She emphasized the urgent need for innovative treatments for these diseases, as 95% of rare diseases worldwide have limited or no approved treatment options, and 80% of those rare diseases have a genetic cause. She said this makes patients with rare disease collectively one of the most underserved communities in medicine today.

She said educating every organization involved in the process of developing these therapies on the stories of real patients affected by these diseases will be critical as gene therapies move through both scientific and legislative processes.

Its really to address the underlying cause of rare diseases at the root, meaning the genetics, not the symptoms It cant be a line item in a bill, it cant be something on a piece of paper that you hear about, it has to be someone telling their story [and] thinking about the patient and what theyre going through.

You can learn so much by just sitting and talking and just hearing their story, and little things that you didnt even know affected them We need to bring that to more of the audience thats involved in making some of these decisions so they can see it as more than just a line on a piece of paper when theyre deciding something.

Fonseca showed a slide explaining some specific uses of gene therapy that could potentially provide individualized, life-saving treatment to people with red blood cell diseases, as well as preventive genetic interventions for diseases like cancer.

When you think about this approach in looking at the rare disorders, it turns out that by extrapolation, a lot of the diseases that we consider common also become more and more individualized, and therefore, theyre more and more unique. More and more, we see approaches that have to be very, very much [a] tailored design for patients

To have someone who is born with [a red-blood cell disorder] return to normal red blood cell function is just enormous. This is a worldwide problem, its a problem thats associated with pain, serious medical problems, a shorter lifespan, and great expenditures for the health system, and so [Im very excited about where were at with this].

Wilson highlighted that while few gene therapies have been officially launched in the market, many are currently in pre-clinical and clinical trials and are expected to provide promising health solutions for the future.

Continued here:
5 Slides We're Discussing: Gene therapy and the promise for rare disease - State of Reform - State of Reform

Cell lines

Human PC cell lines LNCaP and PC-3 obtained from JCRB Cell Bank (Osaka, Japan), and 22Rv1 purchased from The European Collection of Authenticated Cell Cultures were maintained in RPMI-1640 (FUJIFILM Wako Pure Chemical, Osaka, Japan) containing penicillin, streptomycin and 10% fetal bovine serum (Equitech-Bio, Kerrville, TX). The docetaxel-resistant 22Rv1 cell line, 22Rv1DR, was previously described18. All cell culture experiments were performed using cells within less than 20 passages except for PC-3PRF cells stably transfected with Tet-on tetracycline-inducible perforin expression vector and docetaxel-resistant 22Rv1DR cells.

Docetaxel was purchased from Selleckchem (Houston, TX, USA).

The perforin expression vector for Tet-On system (pT-Rex-DEST30-perforin) was purchased from Thermo Fisher Scientific (Waltham, MA, USA). The pcDNA6/TR regulatory vector (Thermo Fisher Scientific) and pT-Rex-DEST30-perforin vector were transfected to PC-3 cells using Lipofectamine 2000 (Thermo Fisher Scientific). Transfected cells were selected under 500g/ml G418 and 10g/ml Blasticidin (Thermo Fisher Scientific). Perforin was induced by 1g/ml of tetracycline. The human PSA promoter-driven perforin expression vector (pDRIVEperforin-psa-hpsa) was purchased from InvivoGen (San Diego, CA, USA).

Whole cell lysates were harvested and lysed in RIPA buffer containing the protease inhibitor cocktail (Sigma-Aldrich St. Louis, MO, USA). Western blot analysis was performed as described previously19. The anti-PSA and anti--actin antibodies were purchased from Cell Signaling Technology (Danvers, MA, USA). The immunoreactive proteins were detected using horseradish peroxidase-conjugated anti-rabbit antibody (Cell Signaling Technology) and ImmunoStar (FUJIFILM Wako Pure Chemical).

Perforin expression in conditioned medium, mouse serum and harvested xenograft tumors was measured by human perforin ELISA kit according to manufacturers instruction (Abcam, Cambridge, UK).

SS-cleavable and pH-activated lipid-like material (ssPalmM), 1,2-Dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE), cholesterol and 1,2-Dimyristoyl-rac-glycero-3-methoxypolyethylene glycol-2000 (DMG-PEG2000) were purchased from NOF Corporation (Tokyo, Japan). Encapsulation of plasmid vector in lipid nano-particles was conducted according to a previous report20. First, plasmid DNA and protamine solutions (0.3mg/mL and 0.144mg/mL) were prepared in 10mM HEPES buffer (pH5.3). Plasmid DNA/protamine core particle was prepared by the drop-wise addition of 1mL of the protamine solution into the 1mL of the DNA solution with vortexing. Liposome was composed of ssPalmM, DOPE, cholesterol and DMG-PEG2000 in a molar ratio of 3:4:3:0.5. Lipids (3.3mol of total lipids) were dissolved in 2mL of ethanol and the lipid solution (2mL) was rapidly diluted with an equal volume of the plasmid DNA/protamine core particle suspension with vortexing. The solution was further diluted with 36mL of 10mM HEPES (pH 5.3) to obtain 5% ethanol(v/v) concentration. The diluted solutions were concentrated to ten times by Amicon 8400 ultrafiltration stirred cell with a Biomax membrane (Merck Millipore, Allen, TX, USA) following further serial ultrafiltration with 100mM HEPES (pH 7.4) and 10mM HEPES (pH 7.4) using Amicon 8050 ultrafiltration stirred cell with a Biomax membrane. Finally, the liposome solution was filtrated by 0.45m of pore size Millex HV (Merck Millipore).

Liposome concentration was measured as total cholesterol concentration in the presence of sodium dodecyl sulfate using a Cholesterol E test Wako (FUJIFILM Wako Pure Chemical) and the total amount of fatty acids was calculated based of the molar ratio of each lipid. DNA concentration in liposomes was determined using Quant-iT Picogreen dsDNA Assay Kit (Thermo Fisher Scientific) in the presence of Triton X-100. Particle size and -potential were measured at 25C using Zetasizer Nano-S90 (Malvern Panalytical, Worcestershire, UK) after 50 times dilution of samples with distilled water.

This study was approved by the Medical Review Board of Gifu University, Graduate School of Medicine (No. 2018219). A written informed consent was obtained from participants and blood was collected from male volunteers without clinically detectable cancer. All methods were performed in accordance with the relevant guidelines and regulations in compliance with the Declaration of Helsinki. Human PBMCs were isolated by Ficoll-Paque density gradient centrifugation according to the manufacturers instructions (Amersham Biosciences, Piscataway, NJ).

Cells were seeded on 96-well plates. Twenty-four h after seeding, agents with or without PBMCs were added. Cell viability was determined using WST-1 assay kit (Roche Diagnostics, Mannheim, Germany). The mean value obtained from PBMCs alone was deducted from the values obtained from co-culture of prostate cancer cells and PBMCs.

All animal experiments were approved by the Gifu University Animal Experiment Approval Committee (No. 2019116) and carried out in accordance with the approved guidelines. This study is compliant with the ARRIVE guidelines. Six-week-old male athymic nude mice (BALB/cSlc-nu/nu) were purchased from Japan SLC, Inc. (Shizuoka, Japan). A suspension of 22Rv1DR cells (1107) cells in PBS was mixed with Matrigel (1:1) in a final volume of 0.2mL. The mixture was subcutaneously injected to generate tumors. Two weeks after the injection, tumor volume was measured and mice were randomly assigned to 2 groups (n=5). Agents were intravenously administrated via tale vein. The tumor volume and body weight were monitored and measured once a week. Four weeks after treatment, mice were sacrificed and the resected tumors were weighed.

Statistical analysis was performed using Graph Pad Prism 7 version 7.03 (Graph Pad Software, CA, USA). Comparison of 2 groups was made using t-test or MannWhitney U test. Comparison among 4 groups was made using one-way ANOVA with Tukeys post hoc for multiple comparisons. Differences were considered significant if p<0.05.

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Gene therapy of prostate cancer using liposomes containing perforin expression vector driven by the promoter of prostate-specific antigen gene |...

SAN DIEGO--(BUSINESS WIRE)--Excellos, Incorporated, a cell therapy Contract Development and Manufacturing Organization (CDMO), announces its official corporate launch and closing of $15M in growth funding from Telegraph Hill Partners (THP).

Built on the foundation of the San Diego Blood Bank (SDBB), Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. The companys collection network consists of nine SDBB centers in the San Diego area that see an average of over 70,000 diverse donors annually, as well as exclusive access to select, consented material from SDBBs public cord blood bank. Excellos also has access to a nationwide collection network giving it one of the largest cellular material procurement portfolios. Uniquely connecting its broad collection network to state-of-the-art cGMP and R&D facilities in San Diego, Excellos provides a full suite of innovative end-to-end capabilities to facilitate the development and manufacture of autologous and allogeneic cell therapies. Excellos experience includes working with therapeutic companies developing chimeric antibody receptor-engineered T cells (CAR-T), tumor-infiltrating lymphocytes (TILs), and providing isolated immune cells that are integral to the advancement of immunotherapies.

Excellos will create highly characterized, standardized cell products and custom services that are essential for advanced therapeutics, said David Wellis Ph.D., CEO of Excellos. The SDBB had been incubating Excellos for a number of years, but fully capitalizing on the rapidly growing opportunity required significantly more resources than the SDBB was able to provide. We will now be able to focus exclusively on the needs of the expanding cell and gene therapy industry through the development of our data-driven platform to enable the characterization of cellular therapeutics starting at the donor level. The funding from THP will allow a significant facility expansion and overall growth in the capabilities of our organization.

The growth in the cell and gene therapy industry is driving a surge in demand for critical human cells, tissues, and services to support the development and commercialization of new products, said Paul Grossman, Ph.D., J.D., Partner at THP. We look forward to partnering with Excellos and their proven leadership team. Their exclusive access to one of the industrys largest donor bases, coupled with their technology-focused research and development activities, will help to accelerate the advancement of cell and gene therapies for those patients in need. Paul Grossman, Alex Herzick, and Deval Lashkari from THP have joined the Excellos Board concurrent with the financing.

Excellos founding leadership team brings a wealth of commercial and industry experience to bear. Chief Executive Officer David Wellis Ph.D., previously served as CEO of SDBB for nine years where he foresaw the need for products and services to serve the then nascent cell and gene therapy industry. His guidance allowed the organization to meet these needs, a path that ultimately led to the formation of Excellos. David has also held senior leadership roles at both Illumina and GenVault Corporation. Chief Commercial Officer George Eastwood, brings significant experience in the cell and gene therapy space, with a focus on cGMP materials and cell manufacturing. He served as VP, Global Sales and Business Development for HemaCare, and in his over five years there, he saw the company through a period of dramatic expansion that culminated in its acquisition by Charles River in 2020. Chief Scientific Officer Rob Tressler Ph.D., brings vast experience in R&D for advanced cell-based therapies. Most recently, as CSO of SDBB, he led the cell therapy, immunohematology, components manufacturing, and cord blood banking labs. Rob also has extensive drug development experience, both as Head of Preclinical Oncology at Geron Inc. and VP of R&D at Cellerant Therapeutics.

About Excellos: Built on the foundation of the San Diego Blood Bank, Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. Excellos is dedicated to improving human life by providing critical products and services to life science research and the next generation of cell and gene therapies. Learn more at http://www.excellos.com

About Telegraph Hill Partners: Telegraph Hill Partners, founded in 2001 and based in San Francisco, CA, invests in commercial stage life science, medical technology, and healthcare companies. For more information, please see http://www.telegraphhillpartners.com

Excerpt from:
Excellos Launches to Accelerate Innovation in the Cell and Gene Therapy Industry - Business Wire

MaxHggblom Distinguished Professor and ChairDepartment of Biochemistry and MicrobiologySchool of Environmental and Biological SciencesRutgers-New BrunswickHonored for distinguished contributions to understanding both the fundamental and application components of microbialbiotransformationsof pollutants, especially chlorinated aromaticcompoundsand metalloids.

MaxHggblomis a renowned research scientist and educator with a large body of microbial ecology and environmental biotechnology research that has expanded our understanding of how the biodegradation of environmental pollutants, such as dioxins and PCBs,impact our planet.

His research interests revolve around thebioexploration, cultivation and characterization of novel microbes.His research on bacteria has provided a foundation for applications that address the pollution problems facing impacted industrialized and urbanized environments.

Hggblomslab is also actively studying microorganisms that degrade pharmaceutical and personal care products in aquatic environments.

Over the past decadesthediverse chemicalsin pharmaceutical and personal care productshave emerged as a major group of environmental contaminants in numerous watersheds around the world; therefore, it is important to understand how microbes can degrade them.There is much to explore and learn,Hggblomadded.

Hggblomswork also touches climate change, particularly the roles and responses of microbes in rapidly changing environments, such as the Arctic.In his lab at Rutgers, students have the unique opportunity to exploreareas of research such asthe biodegradation and detoxification of anthropogenic pollutant chemicals, including certainpesticides;respiration of rare metalloids; or life in the frozen tundra soils.

For several years,my lab has worked on studying the microbial ecology of Arctic tundra soils to understand how the changing conditions impact microbial activity and turnover of soil organic matter, and consequently enhanced greenhouse gas flux,Hggblomsaid. This is an important area of research as the threat of microbial contribution to positive feedback of greenhouse gas flux is substantial.

His lab recently received funding from the National Science Foundation to studyhowdiverse microbial communitiesare established insoils.Hggblomwill work with an international research team of scientists from the U.S., China, South Africa and Finland to study soils from the three differentregionsacross Arctic, Tibetan Plateau and Antarctic habitats to expand our understanding of how soil ecosystems respond in critical polar regions.

Emily EversonLayden

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Twelve Rutgers Professors Named Fellows of the American Association for the Advancement of Science - Rutgers Today

WASHINGTON, Jan. 25, 2022 (GLOBE NEWSWIRE) -- The GlobalHemophilia Market size is expected to reachUSD 14.2 Billionby 2028, exhibiting a CAGR of5.3%during the forecast period. Hemophilia is a bleeding condition, which leads to the prolonged bleeding after injury or a surgery due to a delay in the blood clotting, The Global Hemophilia Market is anticipated to grow at a substantial rate in coming years because of increasing cases of genetic abnormalities and prevalence of Hemophilia, states Vantage Market Research, in a report, titledHemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028).The market size valued atUSD 12.1 Billionin 2021.

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Market Overview:

Increasing Establishment of Hemophilia Treatment Centers to Drive the Market

The availability of limited treatment therapy options and growing burden on regulatory bodies towards the treatment is resulted to increase R&D efforts. The public as well as private healthcare bodies are heavily investing in development of specialized clinics that are established to meet the targeted needs of patients. In this regard, there is increasing number of Hemophilia treatment centres that also aims at proving treatment to underprivileged patients.It is recommended by authorities that people who are suffering from Hemophilia should visit a treatment center for optimal care and health education to stay healthy. The establishment of healthcare centres is anticipated to fuel theHemophilia Market growth.Additionally, efforts are been undertaken by regulatory bodies for spreading awareness regarding the disease and providing information about the effective treatment.

The COVID-19 outbreak has affected various industries worldwide. TheHemophilia Marketis no exception. Governments across the world took severe actions like border seals, lockdown, and implementing strict social distancing measures, in order to stop swift spread of COVID-19. These actions led to severe impact on the global economy impairing various industries. The impact of COVID-19 on the market demand is considered while estimating the current and forecast market size and growth trends of the market for all the regions and countries based on the following data-points:

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Benefits of Purchasing Hemophilia Market Reports:

The Report on Hemophilia Market Highlights:

Various Supportive Initiatives to Drive Market Growth in Asia Pacific

Asia Pacificis anticipated to witness fastest CAGR over the forecast period. The awareness campaigns and supportive initiatives taken by the government to commence the early screening of neonates, is about to boost the demand for diagnostic tools related to Hemophilia in the region. Some other factors such as advanced healthcare ecosystem and capacity of people to spend on such expensive medical services are defining the regional business growth. Additionally, easy medical reimbursement schemes are promoting the market growth.

List of Prominent Players in the Hemophilia Market:

Read Full Research Report @ https://www.vantagemarketresearch.com/industry-report/hemophilia-market-1216

Recent Developments:

December, 2021:The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for Hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal which could allow patients in Europe to access the therapy sooner,

December, 2021:Global biotherapeutics leader CSL Behring announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.

December, 2021:Patients with severe Hemophilia can develop inhibitors against factor VIII or IX, preventing factor replacement therapy from working, said Dr. Guy Young (University of Southern California, CA, USA). A quarter of patients develop these inhibitors, leading to a worse prognosis. Novel agents are needed to protect these patients from bleeding events and arthropathy and improve their quality of life. In addition, the current IV therapies need to be administered multiple times per week, resulting in venous access issues and poor adherence.

December, 2020:Pfizer Inc. and Sangamo Therapeutics, Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe Hemophilia A.

Browse the Report Hemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028) @ https://www.vantagemarketresearch.com/blog/hemophilia-225379

This market titled Hemophilia Market will cover exclusive information in terms of Regional Analysis, Forecast, and Quantitative Data Units, Key Market Trends, and various others as mentioned below:

Treatment: - On-demand, Prophylaxis

Therapy: - Replacement therapy, ITI therapy, Gene therapy

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Global Hemophilia Market to Reach $14.2 Billion by 2028 - - GlobeNewswire

The switch design works solely for certain subsets of cancers, specifically non-small-cell lung cancer cells with an EGFR gene mutation, where drugs that target mutated proteins in the cancer cell are already on the market.

We are taking a careful approach to design and testing, Pritchard said. We will look specifically for failures within the switch, and analyze what we find, sort of like when civil engineers analyze a building or bridge failure after the fact. Failures help us understand where our ideas about cancer therapy are incomplete, and what we can do to fix them and increase our knowledge.

After initial tests on cancer cell lines, the researchers will test the dual-switch gene drives on human organoids, provided by the University of Massachusetts Medical School, which are patient-derived cancer cells that more closely mimic real tumors.

Co-investigator Shelly Peyton, Armstrong Professional Development Professor at University of Massachusetts Amherst and expert in tissue engineering, will lead the design of microenvironments to determine how the gene therapy functions under different conditions. Peytons team will study how certain switches or parameters fail, or why they function well in some environments but not others.

The research here is trying to take the challenge of cancer treatment and flip it on its head, said Scott Leighow, fifth-year doctoral student in biomedical engineering, who gathered the preliminary data that were key to securing the grant. If we can do that, we'll have a therapy that can handle resistant forms of cancer a lot better than what's currently in our arsenal.

If the study is successful, the researchers will test their treatments on animal models to show proof-of-concept, Leighow said. Far in the future, the technology has the potential to offer a cellular gene therapy that might assist cancer patients who are not candidates for surgery.

The grant is part of a new consortia created by the NCI to promote collaborative approaches to synthetic biology for cancer applications.

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$2.5M grant awarded to flip the switch on lung cancer drug resistance - Penn State News

Patient 1 with Sandhoff disease realized normalization of Hex A enzyme activity by Month 1, achieving 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3

Patient 2 with Tay-Sachs disease achieved Hex A enzyme activity 5-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 1

First-ever data supporting bicistronic vector approach in humans, TSHA-101 is designed to deliver both HEXA and HEXB genes in the endogenous ratio

Conference call and live webcast today at8:00 AM Eastern Time

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported positive initial serum -hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, which represent two forms of GM2 gangliosidosis. Todays data are the first ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.

TSHA-101 is the first bicistronic vector in clinical development, representing an important first for the field of gene therapy, noted RA Session II, President, Founder and CEO of Taysha. TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis. We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.

Based on natural history data, patients with asymptomatic GM2 gangliosidosis have Hex A enzyme levels that are at least 5% of normal activity. Key patient findings for Hex A enzyme activity following treatment with TSHA-101 include:

Patient 1 (Sandhoff disease)

Patient 2 (Tay-Sachs disease)

Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development at Taysha added, GM2 gangliosidosis is a progressive and life-limiting disease with no treatment options. Normalization of patient Hex A enzyme activity levels 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history supports TSHA-101s ability to potentially make a meaningful difference in the lives of patients with Sandhoff and Tay-Sachs diseases after a single intrathecal administration. We look forward to submitting a protocol amendment to expand patient enrollment in the ongoing Phase 1/2 trial and providing additional updates later this year.

Preliminary data suggest that TSHA-101 was well-tolerated with no significant drug-related events.

Patient 1 (Sandhoff) demonstrated signs of clinical improvement at Month 3 and was deemed stable to travel home. Upon returning home, Patient 1 (Sandhoff), who was unvaccinated, was exposed to a family member symptomatic for an upper respiratory infection, possibly Covid-19, and was hospitalized with pneumonia before contracting a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection. On January 14, 2022, the patient succumbed to pneumonia and pleural effusion with a concomitant hospital-acquired MRSA infection. The principal investigator has made the initial assessment that the death was unrelated to study drug. Final determination from the independent data safety monitoring board (DSMB) is anticipated in the near term.

TSHA-101 is an investigational gene therapy that delivers both the HEXA and HEXB genes that comprise the -hexosaminidase A enzyme. The two genes are driven by a single promoter within an AAV9 capsid ensuring that the two sub-units of Hex A are produced in the endogenous ratio within each cell, which is important to ensure efficient production of the enzyme. TSHA-101 is the first and only bicistronic vector currently in clinical development for GM2 gangliosidosis and has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA and Orphan Drug Designation from the European Commission. TSHA-101 is administered intrathecally and is currently being evaluated in a single arm, open-label Phase 1/2 clinical trial for the treatment of infants with GM2 gangliosidosis sponsored by Queens University. Additional clinical safety and efficacy data are expected by the end of 2022.

GM2 gangliosidosis is a rare and devastating monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A (Hex A) enzyme. These genetic defects result in progressive dysfunction of the central nervous system. Residual Hex A enzyme activity determines the severity of the disease. The infantile form of the disease has an onset of symptoms usually before six months of age with residual Hex A enzyme activity of less than 0.1%. Juvenile onset occurs between 1.5 and five years of age with residual Hex A enzyme activity of approximately 0.5%. Early adult onset of the disease has residual Hex A enzyme activity of between 2% to 4%. There are no approved therapies for this disease, and current treatment is limited to supportive care.

Conference Call and Webcast Information

Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to provide an update on the GM2 gangliosidosis program. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13726741. The live webcast and replay may be accessed by visiting Tayshas website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

About Taysha Gene Therapies

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, plans, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, such as TSHA-101 and including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, and our corporate growth plans. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, both of which are available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101 - BioSpace

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that Leber Hereditary Optical Neuropathy (LHON) subjects treated with LUMEVOQ continued to experience significantly improved vision four years after a single injection of the gene therapy. The findings come from RESTORE (CLIN06), the long-term follow-up study to which participants in the RESCUE1 and REVERSE2 Phase III pivotal trials were invited.

When RESTORE subjects enrolled in the study, 2 years after the one-time injection, they had already experienced clinically meaningful improvements relative to the lowest point (the nadir) of their best-corrected visual acuity (BCVA): +18.8 ETDRS letters equivalent* in their LUMEVOQ-treated eyes and +17.3 letters equivalent in their sham-treated eyes. Four years after treatment, the bilateral improvement from nadir was sustained, with LUMEVOQ-treated eyes achieving a mean improvement against nadir of +22.5 letters equivalent and sham-treated eyes demonstrating a mean improvement of +20.5 letters equivalent.

The impact of such results on patients is demonstrated by increases in the self-reported quality of life (QoL) scores at Year 4 vs. baseline. Mean overall QoL increased by a clinically meaningful magnitude relative to baseline, driven by clinically meaningful increases in the sub-scores corresponding to mental health and the ability to carry out activities autonomously (e.g., role difficulties, dependency, near and far activities, general vision).

The 4-year RESTORE long-term extension study provides patients with Leber Hereditary Optic Neuropathy and their families as well as the neuro-ophthalmology community with highly informative data about both the efficacy and safety of intravitreal LUMEVOQ therapy, commented Dr. Robert Sergott, Director, Neuro-Ophthalmology Service, Wills Eye Hospital, and Founding Director and CEO, William H. Annesley EyeBrain Center, Thomas Jefferson University, Philadelphia, PA, USA. Compared to the natural history of LHON, the 4-year data extend and validate the 3-year observations by confirming that objective visual acuity improvement is sustained and is associated with improved functional visual quality of life without any long-term safety concern.

The RESTORE findings underline the therapeutic value of GenSights pioneering one-time treatment for LHON: durable and clinically significant improvement in visual function coupled with impressive safety, noted Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. The body of evidence we have now accumulated is without doubt good news for patients needing an urgent solution for their brutal blinding condition, and consequently we are continuing to work vigorously with the relevant authorities to bring regulatory review process to a successful conclusion.

RESTORE is one of the largest long-term follow-up studies for a rare disease treatment, with 62 subjects accepting the invitation to enroll. All subjects, who were affected by LHON caused by a mutated ND4 mitochondrial gene, were treated with an intravitreal injection of LUMEVOQ in one eye and with sham injection in the other.

Table 1. BCVA Mean Improvement Vs. Nadir* In LUMEVOQ Long-Term Follow-Up (RESTORE)

2 Years Post-Injection

3 Years Post-Injection3

4 Years Post-Injection

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LUMEVOQ-treated eyes

-0.375(0.306)

+18.8

-0.410(0.365)

+20.5

-0.453(0.440)

+22.5

Sham-treated eyes

-0.346(0.291)

+17.3

-0.387(0.369)

+19.4

-0.406(0.361)

+20.5

Note: The RESTORE sample consists of the RESCUE and REVERSE participants who accepted to be followed in the long-term follow-up study. Year 4 values were the LogMAR readings nearest to 1461 days post treatment recorded between 1461 +/- 273 days post- treatment. Missing values were imputed using the Last Observation Carried Forward (LOCF) method. *Nadir = worst best-corrected visual acuity recorded from baseline to Year 4. ** Assessments of best-corrected visual acuity (BCVA) were recorded in LogMAR. The change from nadir in LogMAR was converted to letters equivalent improvement by multiplying the LogMAR by -50 (ref. J.T. Holladay, J Refrac Surgery, 1997;13, 388-391).

Responder analyses at Year 4 indicate that improved BCVA was a benefit for a substantial proportion of the study participants. 71.0% of RESTORE subjects achieved Clinically Relevant Recovery (CRR)4 against nadir four years after treatment, and 80.7% of them had on-chart vision (BCVA 1.6 LogMAR) in one or both eyes.

Viewed against the trend in vision typically seen in untreated patients5, the findings represent a significant departure from the natural progression of LHON.

Safety findings at 4 years post-injection were consistent with previous readouts, which concluded that LUMEVOQ is well-tolerated: no serious adverse events were recorded among LUMEVOQ-treated eyes, and no discontinuations occurred due to ocular events. There were no systemic serious adverse events or discontinuations related to study treatment or study procedure.

The review of the European Marketing Authorisation Application for LUMEVOQ is ongoing, with the decision from the CHMP expected in Q4 2022.

References and notes:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,200-1,500 new subjects who lose their sight every year in the United States and the European Union.

About LUMEVOQ (GS010; lenadogene nolparvovec)

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About RESCUE, REVERSE, and RESTORE

RESCUE and REVERSE were two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measured the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, were used for statistical purposes. Both trials were adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between drug-treated and sham-treated eyes, adjusted to baseline.

The secondary endpoints involved the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was performed, including the proportion of patients who maintained vision (< ETDRS 15L loss), the proportion of patients who gained 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics included automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality-of-life scales, biodissemination and the time course of immune response. Readouts for these endpoints were at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were invited to participate in a long-term follow-up study, RESTORE, for three additional years.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767RESTORE: NCT03406104

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GenSight Biologics Reports Clinically Meaningful Vision Improvement is Maintained 4 Years After One-time Treatment with LUMEVOQ Gene Therapy -...

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced that its Investigational New Drug (IND) application for its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN), was cleared by the U.S. Food and Drug Administration (FDA).

SBT101 is the first AAV-based gene therapy in development specifically designed for people living with AMN, an adult-onset degenerative spinal cord disease caused by mutations in the ABCD1 gene. SwanBio plans to initiate a randomized, placebo-controlled Phase 1/2 clinical trial designed to assess the safety and explore the efficacy of SBT101 in patients with AMN in the second half of 2022.

Todays IND clearance is a formative milestone for SwanBio, enabling us to evolve from a preclinical company to a truly integrated research and development organization, underscoring the expertise of our team and potential of our technology platform, said Tom Anderson, chief executive officer and director of SwanBio Therapeutics. SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients.

The clinical program for SBT101 builds on SwanBios unique understanding of AMN, including new insights being gathered in an ongoing natural history study. SwanBio is deeply committed to the AMN community and has worked closely with patients, family members, and expert physicians including SwanBio Co-Founder Dr. Florian Eichler to ensure that its clinical programs are designed to meet their needs. SwanBio is supported by long-term investment partners Syncona Ltd. and Mass General Brigham Ventures, which both have proven track records in gene therapy, particularly in AAV-focused therapies.

About SBT101SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was shown to be well-tolerated in non-human primates at six months post-treatment.

About AdrenomyeloneuropathyAdrenomyeloneuropathy (AMN) is the adult-onset degenerative spinal cord disease that affects people living with adrenoleukodystrophy (ALD), a category of rare, genetic, and metabolic conditions. AMN is characterized by progressive loss of mobility, incontinence, and debilitating pain. It affects adults with mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Without a functioning version of this protein there is an accumulation of VLCFA to toxic levels that leads to progressive dysfunction of the central nervous system. Between 8,000-10,000 men in the U.S. and E.U. are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom control.

About SwanBio TherapeuticsSwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

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SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy -...

DUBLIN--(BUSINESS WIRE)--The "Protein Expression Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.

The global protein expression market is expected to grow from $2.01 billion in 2020 to $2.13 billion in 2021 at a compound annual growth rate (CAGR) of 6%. The market is expected to reach $3.03 billion in 2025 at a CAGR of 9.2%.

Major players in the protein expression market are Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific Inc., New England Biolabs and Promega Corporation.

The protein expression market consists of sales of protein expression vectors, competent cells, reagents, equipment and related services. Protein expression is a process in which proteins are synthesized, modified, regulated and controlled in living organisms according to the host cell. Protein expression included yeast expression, insect expression, and bacterial expression, algal expression and mammalian cell expression.

The protein expression market covered in this report is segmented by protein expression into yeast expression, mammalian expression, algae expression, insect expression, bacterial expression, cell-free expression. It is also segmented by end use into pharmaceutical and biotechnological companies, academic research, contract research organizations; by product into reagents, competent cells, expression vectors, services, instruments and by application into therapeutic, industrial, research.

Government regulations related to protein therapeutics and production of biologics may hinder the protein expression market growth. Government regulations on biologics to undergo rigorous preclinical and clinical trials prior to regulatory approval, and time consuming process for approval of biologics with regards to health and the safety of any individual are restraining the market growth.

Marketing and distribution of biologics including insulin, hormones, therapeutic antibodies, and vaccines depends upon the successful completion of clinical trials, which is a long, expensive, and uncertain process. According to FDA, for an approval of new biologic, Under the regulations (21 CFR 314.81(b)(2)(vii) and 601.70, a clinical trial approval usually takes 10- 12 months where firms are required to submit a report annually on the status of clinical safety, clinical efficiency, clinical pharmacology, and nonclinical toxicology study.

Companies in the industry are increasingly adopting Microfluidics technology to enhance protein expression tests in order to reduce the time, cost, labor, and increase the accuracy and performance. The microfluidics technology effectively analyzes biological samples than the traditional (macroscale) instruments.

Microfluidics technology is used to measure the expression of proteins on cells and optimizes the output to generate results regarding protein expression. Therapeutics-on-a-chip (TOC) uses microfluidic platform and is able to synthesize proteins in a point of care setting to reduce cost associated with storage and transportation of therapeutic proteins.

For instance, companies such as MissionBio, NanoCellect Biomedical, RainDance Technologies and Sphere fluidics have implemented this technology in protein expression test.

Increase in demand for biologics to counter various genetic disorders and chronic diseases is one the major factors driving the research and sales of protein expression market. Biologics is a medicine produced from living organisms or contains components of living organisms such as protein, tissue, genes, allergens, cells, blood components, blood, and vaccines.

The increasing use of biologics (therapeutic protein and others) to cure chronic diseases such as cancer, cardiovascular conditions and genetic disorders, is increasing the demand for protein expression devices and equipment. According to the World Health Organization, chronic disease prevalence is expected to rise by 57% globally, by the year 2020.

Hence the rising demand for biologics is driving the protein expression market. For instance, according to an article published by Chemistry World, analysts expect the biologics market to hold a market share of more than a quarter of the entire pharmaceutical market by 2020. The global biologics market is expected to grow at 9.9% during 2018-2024.

The Protein Expression market in the U.S. is governed by Food and Drug Administration (FDA) that lays down a series of guidelines for the manufacturers and retailers of this industry. Within FDA, Center for Drug Evaluation and research (CDER) regulates biological products under FDA 101 which includes gene therapy products and vaccines. These regulations ensure quality, safety and efficacy of biological therapeutics products, and speed up innovations that make these products safer, and effective.

The US's FDA announced a fast-track initiative to review its drugs and biologics policy to speed the availability of therapies to patients with serious conditions, orphan drugs for rare disease, while preserving the safety and efficacy standards. FDA also removed a rule (Section 610.21 of the FDA code) which specified minimal potency limits for certain antibodies and antigens.

The European Medicines Agency has also introduced policies which include a provision to waive the scientific advice fee, which encourage more academic groups and small companies to propose candidates for biologics.

Key Topics Covered:

1. Executive Summary

2. Protein Expression Market Characteristics

3. Protein Expression Market Trends and Strategies

4. Impact Of COVID-19 On Protein Expression

5. Protein Expression Market Size and Growth

5.1. Global Protein Expression Historic Market, 2015-2020, $ Billion

5.1.1. Drivers Of the Market

5.1.2. Restraints On the Market

5.2. Global Protein Expression Forecast Market, 2020-2025F, 2030F, $ Billion

5.2.1. Drivers Of the Market

5.2.2. Restraints On the Market

6. Protein Expression Market Segmentation

6.1. Global Protein Expression Market, Segmentation by Protein Expression, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.2. Global Protein Expression Market, Segmentation by End Use, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.3. Global Protein Expression Market, Segmentation by Product, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.4. Global Protein Expression Market, Segmentation by Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7. Protein Expression Market Regional and Country Analysis

7.1. Global Protein Expression Market, Split by Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7.2. Global Protein Expression Market, Split by Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/iix75m

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Global Protein Expression Market Research Report 2021 Featuring Major Players - Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific,...

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, announced a research and development collaboration with Haihe Laboratory of Cell Ecosystem to develop hematopoietic stem cell regenerative therapies and platform technology by combining resources and expertise from both sides.

The Haihe Laboratory of Cell Ecosystem, run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is focused on conducting fundamental research, innovation, and translation in the cell ecosystem.

Under the agreement, both parties will jointly develop hematopoietic stem cell regenerative therapies, including the development of innovative genetically-modified hematopoietic stem cell therapies and the exploration of novel biomarkers to optimize quality control for stem cell production.

With top-notch resources and industry-university-research cooperation, well facilitate the development of cell-based medicine and therapies, said Professor Tao Cheng, Deputy Director of Haihe Laboratory of Cell Ecosystem and President of the Institute of Hematology and Blood Diseases Hospital at the Chinese Academy of Medical Sciences and Peking Union Medical College, a leading hematology researcher who has made a series of discoveries relating to the regulatory and regenerative mechanisms of hematopoietic stem cells. Hematopoietic stem cells (HSCs) have the potential for long-term self-renewal and can differentiate into various types of mature blood cells. These stem cells can be harnessed to provide treatment for a broad range of diseases such as hematological tumors, autoimmune diseases, and hereditary blood disorders. We believe that this collaboration with EdiGene will accelerate the innovation and translation in the field of HSCs, thus enabling healthier patients with new therapies."

Professor Cheng was awarded the second prize of the National Natural Science Award 2020 as the first author of work on basic and translational research that advanced the development of adult hematopoietic stem cells for therapeutic applications.

EdiGene is scaling up clinical translation and development of the first gene-editing hematopoietic stem cell therapy in China following the 2021 approval by the China National Medical Products Administration its IND for its investigational therapy ET-01. Our team has extensive experience in the development and translation of cutting-edge technologies including hematopoietic stem cell and gene editing, said Dong Wei, Ph.D., CEO of EdiGene. "This collaboration with Haihe Laboratory of Cell Ecosystem will further our exploration in the field of hematopoietic stem cells. The partnership with this leading academic institute and our translational know-how enable us to move forward in bringing more innovative treatment options to patients in China and around the world.

In 2021, EdiGene initiated a Phase I multicenter clinical trial of ET-01, its gene-editing hematopoietic stem cell therapy for transfusion-dependent -thalassemia. EdiGene has enrolled the first patient at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College. Currently, the clinical trial is being conducted in Tianjin and Guangdong-Hong Kong-Macao Greater Bay Area (Greater Bay Area). EdiGene also presented its latest research on new surface markers and migration of hematopoietic stem cells at the 63rd Annual Meeting of the American Society of Hematology (ASH) in 2021.

About Haihe Laboratory of Cell Ecosystem

The Haihe Laboratory of Cell Ecosystem ("the Laboratory"), run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is one of the five registered Haihe Laboratories approved by Tianjin Municipal People's Government. With the goal of promoting population health with cell ecosystem, the Laboratory adheres to developing technological frontier, enhancing peoples health, and promoting research, innovation, and development of cell ecosystem in five key areas: cellular ecosystem, cellular ecology and immunity, cellular ecological imbalance and major diseases, cellular ecological reconstruction and frontier technology of cellular ecological research.

About Institute of Hematology and Blood Diseases Hospital (IH), Chinese Academy of Medical Sciences & Peking Union Medical College (CAMS/PUMC)

Founded in 1957, IH is a tertiary specialty hospital under the National Health Commission of China and is the supporting unit of the National Clinical Research Center of Hematologic Diseases and the State Key Laboratory of Experimental Hematology. It is also the main founding unit of Tianjin Base, the core base of the Chinese medical science and technology innovation system with the goal of becoming "the innovation hub of hematology in China." IH mainly engages in basic research, applied research, clinical diagnosis and treatment of hematological diseases, standard-setting, new technology research, new drug evaluation, and translation in hematology and related fields. IH is leading in the diagnosis and treatment of hematological diseases in China and a global scale and has made original achievements. Since 2010, IH has been awarded first place in the Hospital Specialty Reputation Ranking (Hematology) for 12 consecutive years. It has won first place in the Hematology Specialty Ranking for ten consecutive years since 2010 and ranked the first in hematology by the Scientific and Technological Evaluation Metrics (STEM) for Chinese hospitals for eight consecutive years since 2014.

About EdiGene, Inc

EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at http://www.EdiGene.com.

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EdiGene Enters Strategic R&D Collaboration with Haihe Laboratory of Cell Ecosystem to Develop Hematopoietic Stem Cell Regenerative Therapies and...

AMSTERDAM--(BUSINESS WIRE)--VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of muscular and neurodegenerative disorders through vectorized antibodies, today announces a collaboration with Wageningen University to develop novel baculovirus-based technologies for the production of safe and affordable AAV gene therapies.

One of the most important challenges for the gene therapy industry is to develop robust and scalable manufacturing processes that yield safe therapies. VectorY, together with its partner Wageningen University, is making an important strategic investment in these therapies of the future, by developing a production platform capable of successfully manufacturing safe products at a significant lower COGS. The collaboration represents an important step to enable the medical and economical feasibility of gene therapy for diseases that affect larger patient populations.

Under the terms of the collaboration, the Bioprocess Engineering and Virology Groups at Wageningen University and VectorY Therapeutics will work together on two projects. One will utilize the molecular toolbox to generate innovative stable baculovirus genome seeds for AAV production at large scale. The second will focus on the design and evaluation of an intensified and scalable baculovirus production process in bioreactors using state of the art bioprocess technologies.

We are very pleased to be collaborating with VectorY to develop proprietary, next generation AAV expression systems and industry-leading bioprocessing capabilities, said Monique van Oers, Professor of Virology, Wageningen University.

We are honored to partner with Wageningen University, a significant pioneer in the Baculovirology and Bioprocessing field. This partnership will further strengthen VectorYs proprietary AAV production technologies for the development of Next Generation Gene Therapies, added Alexander Vos, CEO VectorY Therapeutics.

ENDS

Notes to Editors

About VectorY

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need. Founded in October 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform andantibody- based targeted degradation technologies, and proprietary manufacturing technology. VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

For more information, see http://www.vectorytx.com

Wageningen University & Research

The mission of Wageningen University and Research is To explore the potential of nature to improve the quality of life. Under the banner Wageningen University & Research, Wageningen University and the specialised research institutes of the Wageningen Research Foundation have joined forces in contributing to finding solutions to important questions in the domain of healthy food and living environment. With its roughly 30 branches, 6.800 employees and 12.900 students, Wageningen University & Research is one of the leading organisations in its domain. An integrated approach to problems and the cooperation between various disciplines are at the heart of Wageningens unique approach. As such, the Laboratory of Virology and The Bioprocess Engineering group work closely together in a number of biotechnological projects aimed at the further development of the baculovirus expression system for vaccine production and gene therapy applications. By combining our research efforts we can optimally profit from knowledge gained in fundamental virology and new technological developments in bioprocess engineering.

For more information, see https://www.wur.nl/en.htm

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VectorY and Wageningen University Sign Strategic Collaboration for the Development of Novel Baculovirus-based AAV Production Technologies - Business...

The human heart, the size of a fist, located just behind and slightly left of the breastbone, tirelessly beats an average of 100,000 times a day. However, conditions that stop the heart from pumping blood efficiently can cause serious problems and ultimately require a heart transplantation.

In a study published in the journal 'Science Translational Medicine', researchers from Osaka University showed that a previously unknown mutation can lead to a condition called dilated cardiomyopathy, which is one of the main causes of heart failure.

Heart failure refers to an incurable condition where the heart is no longer able to meet the body's demands in terms of blood supply. It is one of the most common causes of death and it affects almost 40 million people worldwide, representing a huge public health problem. One of the main factors leading to heart failure is a disease called dilated cardiomyopathy (or DCM). DCM is characterized by dilation of the heart's chambers and a pumping disfunction. Previous research has shown that DCM is often inherited and has a genetic basis. However, for up to 80 per cent of the familial DCM cases, the genetic mutation causing the disease has still not been known.

ALSO READ: Can Omicron cause heart damage? Here's what experts say

The research team identified a gene called BAG5 as a novel causative gene for DCM. First, they studied patients from different families, highlighting a correlation between loss of function mutations in the BAG5 gene and DCM. The researchers found that this mutation has a complete penetrance, meaning that 100 per cent of the individuals presenting it will develop the disease. They then found in a mouse model of dilated cardiomyopathy that mice without BAG5 exhibited the same symptoms of human DCM, such as dilatation of the heart's chambers and irregular heart rhythm. This indicated that mutations that erase the function of BAG5 can cause cardiomyopathy.

"Here we showed that loss of BAG5 perturbs calcium handling in mouse cardiomyocytes," said Dr. Hideyuki Hakui, lead author of the study. BAG5 is important for calcium handling in the heart muscle cells, and calcium is essential for a regular rhythm and overall health of the cardiac muscle, explaining why a loss of BAG5 leads to cardiomyopathy.

"After demonstrating that BAG5 mutations led to loss of functional BAG5 protein," continued Dr. Yoshihiro Asano, senior author of the study, "we also showed that administration of an AAV9-BAG5 vector in a murine model could restore cardiac function. This finding suggests that gene therapy with adeno-associated viruses (AAV) should be further investigated as a possible treatment alternative to heart transplantation for patients who are BAG5 deficient." AAV gene therapy refers to an innovative form of therapy aimed at fixing mutated genes in diseases that have a genetic cause like DCM. Therefore, these findings have paved the way for a potential precision medicine treatment based on gene therapy.

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Not just bones, calcium is vital for heart too: Study - Hindustan Times

New Jersey, United States,-The research study presented in this report offers a complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Gene Therapy Cell Culture Media Market. It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the Gene Therapy Cell Culture Media market. The authors of the report have segmented the Gene Therapy Cell Culture Media market as per product, application, and region. Segments of the Gene Therapy Cell Culture Media market are analyzed on the basis of market share, production, consumption, revenue, CAGR, market size, and more factors. The analysts have profiled leading players of the Gene Therapy Cell Culture Media market, keeping in view their recent developments, market share, sales, revenue, areas covered, product portfolios, and other aspects.

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The report includes company profiling of almost all important players of the Gene Therapy Cell Culture Media market. The company profiling section offers valuable analysis on strengths and weaknesses, business developments, recent advancements, mergers and acquisitions, expansion plans, global footprint, market presence, and product portfolios of leading market players. This information can be used by players and other market participants to maximize their profitability and streamline their business strategies. Our competitive analysis also includes key information to help new entrants to identify market entry barriers and measure the level of competitiveness in the Gene Therapy Cell Culture Media market.

Key Players Mentioned in the Gene Therapy Cell Culture Media Market Research Report:

Fujifilm Holdings Corporation, HiMedia Laboratories Pvt., Ltd, Lonza Group Ltd, Sartorius AG, Thermo Fisher Scientific Inc., Merck KGaA, Danaher Corporation, Takara Holdings Inc., Novartis International AG, Bio-Techne Corporation.

Gene Therapy Cell Culture MediaMarket Segmentation:

Gene Therapy Cell Culture Media Market, By Media Type

Serum-containing Media Serum-free Media Stem Cell Media Specialty Media Chemically Defined Media

Gene Therapy Cell Culture Media Market, By End User

Biotechnology & Pharmaceutical Industry Academic Institute Research Laboratory

The Gene Therapy Cell Culture Media market is segmented as per the type of product, application, and geography. All of the segments of the Gene Therapy Cell Culture Media market are carefully analyzed based on their market share, CAGR, value and volume growth, and other important factors. The report also provides accurate estimations about the CAGR, revenue, production, sales, and other calculations for the Gene Therapy Cell Culture Media market. Each regional market is extensively studied in the report to explain why some regions are progressing at a high rate while others at a low rate. We have also provided Porters Five Forces and PESTLE analysis for a deeper study on the Gene Therapy Cell Culture Media market

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Gene Therapy Cell Culture Media Market Report Scope

Geographic Segment Covered in the Report:

TheGene Therapy Cell Culture Mediareport provides information about the market area, which is further subdivided into sub-regions and countries/regions. In addition to the market share in each country and sub-region, this chapter of this report also contains information on profit opportunities. This chapter of the report mentions the market share and growth rate of each region, country and sub-region during the estimated period.

North America (USA and Canada) Europe (UK, Germany, France and the rest of Europe) Asia Pacific (China, Japan, India, and the rest of the Asia Pacific region) Latin America (Brazil, Mexico, and the rest of Latin America) Middle East and Africa (GCC and rest of the Middle East and Africa)

Key questions answered in the report:

1. Which are the five top players of the Gene Therapy Cell Culture Media market?

2. How will the Gene Therapy Cell Culture Media market change in the next five years?

3. Which product and application will take a lions share of the Gene Therapy Cell Culture Media market?

4. What are the drivers and restraints of the Gene Therapy Cell Culture Media market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Gene Therapy Cell Culture Media market throughout the forecast period?

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Gene Therapy Cell Culture Media Market Size | Global Industry Trends, Segmentation, Business Opportunities And Forecast To 2029 The Oxford Spokesman...

Four years ago, a small Philadelphia biotech company won U.S. approval for the first gene therapy to treat an inherited disease, a landmark after decades of research aimed at finding ways to correct errors in DNA.

Since then, most of the world's largest pharmaceutical companies have invested in gene therapy, as well as cell therapies that rely on genetic modification. Dozens of new biotech companies have launched, while scientists have taken forward breakthroughs in gene editing science to open up new treatment possibilities.

But the confidence brought on by such advances has also been tempered by safety setbacks and clinical trial results that fell short of expectations. In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients. Here are five:

Food and Drug Administration approval of Spark Therapeutics' blindness treatment Luxturna a first in the U.S. came in 2017. A year and a half later, Novartis' spinal muscular atrophy therapy Zolgensma won a landmark OK.

But none have reached market since, with treatments from BioMarin Pharmaceutical and Bluebird bio unexpectedly derailed or delayed.

That could change in 2022. Two of Bluebird's treatments, for the blood disease beta thalassemia and a rare brain disorder, are now under review by the FDA, with target decision dates in May and June. BioMarin, after obtaining more data for its hemophilia A gene therapy, plans to soon approach the FDA about resubmitting an application for approval.

Others, such as CSL Behring and PTC Therapeutics, are also currently planning to file their experimental gene therapies with the FDA in 2022.

Approvals, should they come, could provide important validation for their makers and expand the number of patients for whom genetic medicines are an option. In biotech, though, approvals aren't the end of the road, but rather the mark of a sometimes challenging transition from research to commercial operations. With price tags expected to be high, and still outstanding questions around safety and long-term benefit, new gene therapies may prove difficult to sell.

A record $20 billion flowed into gene and cell therapy developers in 2020, significantly eclipsing the previous high-water mark set in 2018.

Last year, the bar was set higher still, with a total of $23 billion invested in the sector, according to figures compiled by the Alliance for Regenerative Medicine. About half of that funding went toward gene therapy developers specifically, with a similar share going to cell-based immunotherapy makers.

Driving the jump was a sharp increase in the amount of venture funding, which rose 73% to total nearly $10 billion, per ARM. Initial public offerings also helped, with sixteen new startups raising at least $50 million on U.S. markets.

Entering 2022, the question facing the field is whether those record numbers will continue. Biotech as a whole slumped into the end of last year, with shares of many companies falling amid a broader investment pullback. Gene therapy developers, a number of which had notable safety concerns crop up over 2021, were hit particularly hard.

Moreover, many startups that jumped to public markets hadn't yet begun clinical trials roughly half of the 29 gene and cell therapy companies that IPO'd over the past two years were preclinical, according to data compiled by BioPharma Dive. That can set high expectations companies will be hard pressed to meet.

Generation Bio, for example, raised $200 million in June 2020 with a pipeline of preclinical gene therapies for rare diseases of the liver and eye. Unexpected findings in animal studies, however, sank company shares by nearly 60% last December.

Still, the pace of progress in gene and cell therapy is fast. The potential is vast, too, which could continue to support high levels of investment.

"I think fundamentally, investment in this sector is driven by scientific advances, and clinical events and milestones," said Janet Lambert, ARM's CEO, in an interview. "And I think we see those in 2022."

The potential of replacing or editing faulty genes has been clear for decades. How to do so safely has been much less certain, and concerns on that front have set back the field several times.

"Safety, safety and safety are the first three top-of-mind risks," said Luca Issi, an analyst at RBC Capital Markets, in an interview.

Researchers have spent years making the technology that underpins gene therapy safer and now have a much better understanding of the tools at their disposal. But as dozens of companies push into clinical trials, a number of them have run into safety problems that raise crucial questions for investigators.

In trials run by Audentes Therapeutics and by Pfizer (in separate diseases), study volunteers have tragically died for reasons that aren't fully understood. UniQure, Bluebird bio and, most recently, Allogene Therapeutics have reported cases of cancer or worrisome genetic abnormalities that triggered study halts and investigations.

While the treatments being tested were later cleared in the three latter cases, the FDA was sufficiently alarmed to convene a panel of outside experts to review potential safety risks last fall. (Bluebird recently disclosed a new hold in a study of its sickle cell gene therapy due to a patient developing chronic anemia.)

The meeting was welcomed by some in the industry, who hope to work with the FDA to better detail known risks and how to avoid them in testing.

"[There's] nothing better than getting people together and talking about your struggles, and having FDA participate in that," said Ken Mills, CEO of gene therapy developer Regenxbio, in an interview. "The biggest benefit probably is for the new and emerging teams and people and companies that are coming into this space."

Safety scares and setbacks are likely to happen again, as more companies launch additional clinical trials. The FDA, as the recent meeting and clinical holds have shown, appears to be carefully weighing the potential risks to patients.

But, notably, there hasn't been a pullback from pursuing further research, as has happened in the past. Different technologies and diseases present different risks, which regulators, companies and the patient community are recognizing.

"We're by definition pushing the scientific envelope, and patients that we seek to treat often have few or no other treatment options," said ARM's Lambert.

Last June, Intellia Therapeutics disclosed early results from a study that offered the first clinical evidence CRISPR gene editing could be done safely and effectively inside the body.

The data were a major milestone for a technology that's dramatically expanded the possibility for editing DNA to treat disease. But the first glimpse left many important questions unanswered, not least of which are how long the reported effects might last and whether they'll drive the kind of dramatic clinical benefit gene editing promises.

Intellia is set to give an update on the study this quarter, which will start to give a better sense of how patients are faring. Later in the year the company is expecting to have preliminary data from an early study of another "in vivo" gene editing treatment.

In vivo gene editing is seen as a simpler approach that could work in more diseases than treatments that rely on stem cells extracted from each patient. But it's also potentially riskier, with the editing of DNA taking place inside the body rather than in a laboratory.

Areas like the eye, which is protected from some of the body's immune responses, have been a common first in vivo target by companies like Editas Medicine. But Intellia and others are targeting other tissues like the liver, muscle and lungs.

Later this year, Verve Therapeutics, a company that uses a more precise form of gene editing called base editing, plans to treat the first patient with an in vivo treatment for heart disease (which targets a gene expressed in the liver.)

"The future of gene editing is in vivo," said RBC's Issi. His view seems to be shared by Pfizer, which on Monday announced a $300 million research deal with Beam Therapeutics to pursue in vivo gene editing targets in the liver, muscle and central nervous system.

With more and more cell and gene therapy companies launching, the pipeline of would-be therapies has grown rapidly, as has the number of clinical trials being launched.

Yet, many companies are exploring similar approaches for the same diseases, resulting in drug pipelines that mirror each other. A September 2021 report from investment bank Piper Sandler found 21 gene therapy programs aimed at hemophilia A, 19 targeting Duchenne muscular dystrophy and 18 going after sickle cell disease.

In gene editing, Intellia, Editas, Beam and CRISPR Therapeutics are all developing treatments for sickle cell disease, with CRISPR the furthest along.

As programs advance and begin to deliver more clinical data, companies may be forced into making hard choices.

"[W]e think investors will place greater scrutiny as programs enter the clinic and certain rare diseases are disproportionately pursued," analysts at Stifel wrote in a recent note to investors, citing Fabry disease and hemophilia in particular.

This January, for example, Cambridge, Massachusetts-based Avrobio stopped work on a treatment for Fabry that was, until that point, the company's lead candidate. The decision was triggered by unexpected findings that looked different than earlier study results, but Avrobio also cited "multiple challenging regulatory and market dynamics."

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5 questions facing gene therapy in 2022 - BioPharma Dive

DUBLIN, Jan. 14, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Business Outlook" report has been added to ResearchAndMarkets.com's offering.

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The Twice-Monthly Publication Cell and Gene Therapy Business Outlook will offer the following:

Market Sizing and Forecasting of CAGT Markets: Each issue sizes up the market opportunity and projects the future revenues for a given therapeutic segment.

Keeping an Eye on Financing: With billions of investment dollars announced each year, Cell and Gene Therapy Business Outlook tracks who is getting financed (and the companies behind the financing) each issue. On a regular basis we will analyze trends in that financing.

News Briefs and Analysis of the Science That will Shape Tomorrow's Business: Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won't miss an important development in cell and gene therapy.

Deals Between CAGT Companies Tracked: Each issue's "Recent Deals Table" tracks the important deals between stem cell companies as well as the deals they engage in (tech transfers, partnerships, mergers, distribution and other activities) with companies outside the industry.

Cell and Gene Therapy Tools: This newsletter will also report on developments, product launches and deals relating to the makers of cell and gene therapy manufacturing equipment and supplies.

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Cell and Gene Therapy Business Outlook Service - Yahoo Finance

BioMarin Pharmaceutical plans to return to the Food and Drug Administration later this year with clinical trial results it says prove its gene therapy for hemophilia can prevent bleeding for years after treatment.

The data from BioMarin's study, disclosed Sunday ahead of the J.P. Morgan Healthcare Conference, are meant to meet requirements laid out by the agency when it rejected the company's previous approval application a year and a half ago.

They show the gene therapy restored blood clotting protein levels to a range consistent with mild hemophilia and, while those levels waned over time, that trial participants experienced very few, if any, bleeds across the two years most were studied. A handful of volunteers in the Phase 3 trial, the largest to date of a hemophilia gene therapy, were followed for three years and had similar results.

"I believe that these results will answer, quantitatively, quite a lot of the questions that agencies have had," said Hank Fuchs, BioMarin's head of research and development, on a conference call Sunday.

Regulators in Europe have already begun evaluating an application from BioMarin and are expected to make a decision in the first half of this year. In the U.S., BioMarin aims to quickly review the results with the FDA and, should the agency agree, potentially resubmit the therapy in the second quarter.

BioMarin's gene therapy, called Roctavian, is the product of years of research by the California biotech and builds on more than a decade of work by other scientists to develop a treatment for hemophilia's genetic cause. It is designed to deliver into the body a functional copy of the gene that's mutated in people with the "A" form of hemophilia, who are left with little or no clotting protein to stem bleeding.

People with severe hemophilia A, who make up about half of all those with disease, must take regular, preventive infusions of "replacement" clotting protein, also known as Factor VIII. Roctavian, which is meant for these individuals, would in theory allow them to stop, freeing them from chronic treatment while more effectively preventing bleeding.

Results from an earlier, much smaller trial showed such promise and, by mid-2020, BioMarin had come close to replicating those findings in the first group of volunteers enrolled in its Phase 3 study. But the FDA unexpectedly, according to the company sought more information to prove that benefit could last two years.

Last January, BioMarin revealed one-year results from all participants in the trial and, on Sunday, disclosed data from its two-year analysis. Treatment decreased the number of bleeds per year by 85%, from an average of nearly five among the 112 volunteers who were studied for at least six months before infusion to less than one at year two.

Among 17 participants who were given Roctavian three years before the analysis was conducted, the average annual bleeding rate remained below one as well.

"Our clinical outcome here is unassailably great," said Fuchs in a separate interview. "It almost makes the application, honestly, bulletproof."

But levels of Factor VIII activity, which had risen sharply to an average of 43 international units per deciliter of blood at one year, declined to 23 IU/dL by year two and, for those 17 participants, 17 IU/dL by year three. BioMarin reported these values using a lab test known as a chromogenic assay, which it says is more conservative than another one also used.

People with severe hemophilia typically have less than one IU/dL of Factor VIII in their blood, while mild hemophilia is typically considered to be between 5 IU/dL and 40 IU/dL.

The decline has been a source of doubt, causing concerns that Roctavian's ability to prevent bleeds might wane over time as well. At least for the first few years, Sunday's results show that isn't happening yet. BioMarin also points to data from an earlier study, in which annualized bleeding rates remained below one through five years, despite reduced Factor VIII activity.

"A small amount of Factor VIII is going to go a long way towards hemostatic efficacy," said Fuchs on Sunday's call, "and it gives us confidence that what we've seen so far in the Phase 2 study is gonna read through to the Phase 3 study when we get there."

Extrapolating efficacy puts BioMarin on somewhat uncertain ground, however, as it is the first company to advance this far with a gene therapy for hemophilia A. Jean-Jacques Bienaime, BioMarin's CEO, argues the data so far for Roctavian indicate treatment should result in at least five years of bleeding control and perhaps even eight or longer.

"With the Phase 2, we have demonstrated at least five years already. Predicting eight years, I don't think, is a big stretch," he said in an interview.

How the FDA will view BioMarin's data is unclear, although analysts on Wall Street predicted the latest results would be enough to merit an approval. The agency could convene a panel of outside experts to review a resubmitted application from the company, a possibility Fuchs acknowledged on the conference call.

Also uncertain is how Roctavian would be perceived by hemophilia patients and by insurers, should it eventually secure an approval. BioMarin has previously suggested a price as high as between $2 million and $3 million, but that might be viewed as high if Roctavian's benefit isn't lifelong. (ICER, a looked-to drug cost watchdog, previously found Roctavian could be cost effective at a price of even $2.5 million.)

Fuchs said the company plans to present more data at a medical meeting, likely this year, that should help clarify the relationship between Factor VIII activity and expected durability of benefit.

Importantly for Roctavian's future, Sunday's data, while relatively sparse, indicated no new safety issues had emerged in testing. There were no cases of "inhibitors," or antibodies that work against clotting protein, developing following treatment, nor were there any cases of cancer or blood clot blockages.

The former two are both newly of interest following reports of cancers developing in other gene therapy trials, and data showing higher-than-normal levels of clotting factor in a trial of another hemophilia gene therapy being developed by Pfizer and Sangamo Therapeutics.

Note: This story has been updated to include mention of the assay used by BioMarin to measure Factor VIII activity, and of ICER's analysis.

Read more:
BioMarin plans return to FDA with updated data on hemophilia gene therapy - BioPharma Dive

NEW YORK, Jan. 12, 2022 /PRNewswire/ -- Polaris Market Research recently published a research report on "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" in its online research storage.

According to [127+ Pages] research report published by Polaris Market Research, the global Gene Delivery Technologies Market size & share expected to reach to USD 7.86 Billion by 2028 from USD 2.64 Billion in 2020, at a compound annual growth rate (CAGR) of 15.0% during forecast period 2021 to 2028.

What is Gene Delivery Technology? How big is Gene Delivery Technology Industry?

Gene delivery technology is widely used in gene therapies, which involves transferring of genetic and hereditary disorders. These therapies have also performed an important role in shaping the entire pharma landscape. Around 27 gene therapies were revealed in the marketplace and over 990 companies emphasized the research & development, and commercialization of innovative therapies by 2020. The constantly changing market environment for advanced therapies is reportedly driving the market for gene delivery technologies.

The operating market players are building various business strategies to boost the market for gene delivery technologies, while the developing gene delivery technologies are creating openings for several new players in the market. Different research settings offer market applications for various gene delivery technologies. However, due to technical challenges related to each modal type, the clinical settings produce very few applications. Within the clinical settings, physical technologies require a breakthrough in their use.

Request a Sample Report of Gene Delivery Technologies Market: https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market/request-for-sample

Key Aspects Covered By Report:

Top Market Companies Profiles Covered:

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Industrial Gene Delivery Technology Market: Growth Factors

The adoption of gene therapies and subsequent increase in clinical research activities around the globe has fueled the market growth. Also, the growing acceptance of gene therapy products and services has supported the gene delivery technologies market growth prospects. Other key driving factors of the market involve technological advancements in viral vectors, a rising pipeline of advanced therapies, and a growing number of regulatory approvals for advanced therapy products.

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Global Gene Delivery Technology Market: Key Segmentation

Insight by Mode

In 2020, the biological vectors market segment secured the largest revenue share of the gene delivery technologies industry due to the high success rate of Kymriah and Yescarta. Following the acceptance of vectors-based therapy products, the above-mentioned vectors have experienced greater attention.

The chemical delivery method market segment is expected to secure a lucrative growth rate over the study period. Clinical challenges are observed in viral systems which propelled the chemical methods' use. Chemical delivery systems have replaced viral delivery systems because of their capability in combating challenges.

The physical delivery methods market segment has lower transfection efficiency than biological or chemical modes. One other drawback called low cell viability in electroporation-based physical methods enables other market players to gain more share. It helps them to address a high focus on transfection and cell viability issues.

Also Read, Global Gene Therapy Market Report, 2021-2028

Insight by Method

The ex-vivo market segment of the gene delivery technologies market witnessed the largest share in 2020. Its transduction efficiency is the major factor behind the high share achievement, making it an ideal candidate to be used in research settings.

In this market, the in-vivo delivery method market segment is expected to obtain a lucrative growth rate over the estimation period as it features a high preference for highly targeted gene deliveries. Researchers are extending research & development for the market segment. For example, Oregon Health and Science University built the gene-editing tool "Crispr-Cas9" in 2020, which enables genetic code editing for blind people.

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Gene Delivery Technologies Market: Report Scope

Report Attribute

Details

Market Size 2020 Value

USD 2.64 Billion

Market Outlook for 2028

USD 7.86Billion

Expected CAGR Growth

CAGR 150% from 2021 - 2028

Base Year

2020

Forecast Year

2021 - 2028

Top Market Players

Horizon Discovery Group Co., QIAGEN, Oxford Biomedica, SignaGen Laboratories, Hoffmann-La Roche AG, Vectalys, Sirion-Biotech GmbH and Others

Segments Covered

By Mode, By Application, By Method, By Region

Geographies Covered

North America, Europe, Asia Pacific, Latin America and Middle East & Africa

Customization Options

Customized purchase options are available to meet your research needs. Explore customized purchase options

Geographic Overview: Gene Delivery Technology Market

The North American region registered a significant share of the global gene delivery technology market. Various clinical trials are used to access the efficacy of gene therapies to treat hereditary, cancer, genetic mutations, and rare disorders in the U.S. This factor is the key driver of the gene delivery technologies demand growth in North America.

In addition, the availability of better clinical infrastructure also contributes to market growth. Many companies are marketing gene delivery products and accessories, which will boost the gene delivery technologies industry growth prospects. The U.S. has already announced many research projects combined with other leaders under its Horizon 2020 plans. This project will also cover other vector-based gene delivery trials for rare diseases.

Moreover, the Asia Pacific gene delivery technologies industry is anticipated to account for a profitable gene delivery technology market growth rate over the assessment period. The region is well known for the developed pharmaceutical industry even with its large population size, and low labor costs.

Browse the [127+ Pages] Detail Report "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" with in-depth TOC: https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market

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The market is primarily segmented on the basis of mode, application, method, and geographic region.

Gene Delivery Technology Market: By Mode Outlook

Gene Delivery Technology Market: By Application Outlook

Gene Delivery Technology Market: By Method Outlook

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At 15.0% CAGR, Global Gene Delivery Technologies Market Size Will Reach USD 7.86 Billion By 2028: Polaris Market Research - PRNewswire

EXTON, Pa., Jan. 10, 2022 /PRNewswire/ -- Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for rare genetic diseases, today announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek's technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek's development pipeline beyond skin and connective tissue disorders to rare liver diseases.

"This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB)."

With the acquisition of Novavita Thera, formerly aCytotheryx, Inc., company, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (HT1),a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.

Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. The therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.Castle Creek plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.

In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children's Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Dr. Lillegard and Dr. Kaiser will be the company leads for Castle Creek's recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

"It is an exciting time to join Castle Creek, a company that has already established an impressive research and development program in cell and gene therapies with proven clinical development and in-house manufacturing capabilities," said Dr. Lillegard. "I look forward to collaborating with the company's dedicated team on development of novel gene therapies. We believe our work to evaluate the safety of in vivo lentiviral vector administration in HT1 has the potential to be a precedent setting approach that can be applied to a range of new therapeutic areas for underserved patient populations."

About Castle Creek Biosciences, Inc.

Castle Creek Biosciences, Inc. is a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for patients living with rare genetic diseases. Castle Creek's most advanced product candidate, dabocemagene autoficel (FCX-007, D-Fi), an ex vivo, autologous gene therapy, is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also evaluating FCX-013, an ex vivo, autologous gene therapy, in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma.In addition, LV-FAH, an in vivo, investigational gene therapy candidate, is being assessed in preclinical studies for the treatment of hereditary tyrosinemia type 1 (HT1).Castle Creek is pursuing discovery and development of early-stage novel product candidates utilizing its dual platform of ex vivo and in vivo technologies to expand its robust pipeline. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences, LLC

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contacts

Adam DaleyBerry & Company Public Relations212.253.8881[emailprotected]

Karen CaseyCastle Creek Biosciences302.750.4675[emailprotected]

SOURCE Castle Creek Biosciences, Inc.

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Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform - PRNewswire

Risky biotechnology start-ups that soared in recent years can't catch a break in 2022. Beam Therapeutics (NASDAQ:BEAM) recently signed a major research deal with Pfizer (NYSE:PFE) that could be worth up to $1.35 billion, and hardly anyone seemed to notice.

This was clearly great news for Beam Therapeutics, but a stock market scorned for clinical-stage biotech stocks didn't respond the way anyone familiar with the company would have expected. Instead of surging higher in response to the Pfizer deal, Beam Therapeutics stock actually fell nearly 2% on the day of the announcement.

Nearly all biotech stocks are in the doghouse lately and it looks like the market may have missed something here. Let's look closer to see if Beam Therapeutics is a smart buy at the moment.

Image source: Getty Images.

Over the past few years, Pfizer has watched its peers experiment with CRISPR-based gene editing techniques without making any significant investments. Beam Therapeutics' base-editing technology, though, really got the big pharma company's attention.

Instead of removing and replacing entire sections of genetic material like Intellia, andCRISPR Therapeutics (NASDAQ:CRSP), Beam Therapeutics is pioneering a more precise method called base editing. This involves altering just one letter of genetic material at a time, which is a lot more useful than it might seem. Around half of all known genetic variations associated with diseases are caused by single-point mutations.

Pfizer will give Beam Therapeutics a $300 million payment up front to discover new drug candidates aimed at three undisclosed targets that won't compete with Beam's existing programs. Beam's eligible for up to $1.05 billion in milestone payments if all three go on to become a commercial success.

Beam Therapeutics is eligible to receive royalties at an undisclosed percentage of global sales for each future program. Beam Therapeutics even has an option to co-develop and co-commercialize one of the future candidates for a larger cut of sales.

Beam Therapeutics finished September with $934 million in cash after operations burned through $329 million during the first nine months of the year. Pfizer's cash injection should raise the company's cash balance high enough to get through 2023 before it needs to tap investors for more.

There's no telling whether Pfizer will decide to license a candidate from Beam Therapeutics. If Pfizer drags its feet, the gene-editing start-up has some preclinical-stage programs of its own that might have a chance to impress investors before it's time to raise capital again.

The most advanced candidate in Beam's pipeline at the moment, BEAM-101 is an experimental gene therapy for the treatment of sickle cell disease. The company doesn't expect to begin enrolling patients into the first clinical trial with BEAM-101 until the second half of 2022.

With a recent market cap of $4.5 billion, there's already a lot of success for Beam's pipeline priced into the stock. Unfortunately, the road ahead could be a lot more challenging than investors are anticipating. Last year, Vertex Pharmaceuticals (NASDAQ:VRTX) and collaboration partner CRISPR Therapeutics reported highly encouraging results from a clinical trial with CTX001 that started way back in 2018.

CTX001 is an experimental gene therapy for sickle cell disease that's similar to BEAM-101 in that it encourages the production of fetal hemoglobin. If early interim data that Beam Therapeutics posts a couple of years from now doesn't appear competitive with CTX001, the stock could take a tumble. While this is a top gene-editing stock to watch, it's still a little too risky to buy right now.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

See the rest here:
Is Beam Therapeutics a Good Stock to Buy Now? - The Motley Fool

Amicus Therapeutics is moving on from an early-phase Batten disease program after follow-up data showed the therapy didn't stop the fatal nervous system disease from progressing long-term.

The company disclosed the pipeline trim in a preliminary revenue guidance announcement ahead of its Wednesday presentation at the J.P. Morgan Healthcare Conference.

Amicus was advancing a gene therapy program for CLN6 Batten disease, a type of the rare genetic disorder that causes development regression and typically begins in childhood. Back in 2019, Amicus shared data on AAV-CLN6 that showed the gene therapy stabilized children's motor and language functions.

But the CLN6 program will now be discontinued after the company got a look at long-term extension data from a phase 1/2 trial.

RELATED:Amicus shares early look at Batten disease gene therapy

The company found the disease stabilization that had occurred during the earlier portion of the trial was not sustained at the two-year mark. Amicus plans to review the data with the CLN6 Batten disease community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed, according to the guidance announcement.

During its last earnings report in November 2021, Amicus had said it would be ramping up manufacturing activities and regulatory discussions for the program.

Meanwhile, the company will advance its CLN3 Batten disease program, which is currently in a phase 1/2 trial. A readout from the trial and additional preclinical data are expected in 2022, Amicus said. Once the data are released, the company can begin work on a pivotal trial.

Continued here:
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another - FierceBiotech

I said, I am an RNA scientist. I can do anything with RNA, Dr. Karik recalled telling Dr. Weissman. He asked her: Could you make an H.I.V. vaccine?

Oh yeah, oh yeah, I can do it, Dr. Karik said.

Up to that point, commercial vaccines had carried modified viruses or pieces of them into the body to train the immune system to attack invading microbes. An mRNA vaccine would instead carry instructions encoded in mRNA that would allow the bodys cells to pump out their own viral proteins. This approach, Dr. Weissman thought, would better mimic a real infection and prompt a more robust immune response than traditional vaccines did.

It was a fringe idea that few scientists thought would work. A molecule as fragile as mRNA seemed an unlikely vaccine candidate. Grant reviewers were not impressed, either. His lab had to run on seed money that the university gives new faculty members to get started.

By that time, it was easy to synthesize mRNA in the lab to encode any protein. Drs. Weissman and Karik inserted mRNA molecules into human cells growing in petri dishes and, as expected, the mRNA instructed the cells to make specific proteins. But when they injected mRNA into mice, the animals got sick.

Their fur got ruffled, they hunched up, they stopped eating, they stopped running, Dr. Weissman said. Nobody knew why.

For seven years, the pair studied the workings of mRNA. Countless experiments failed. They wandered down one blind alley after another. Their problem was that the immune system sees mRNA as a piece of an invading pathogen and attacks it, making the animals sick while destroying the mRNA.

Eventually, they solved the mystery. The researchers discovered that cells protect their own mRNA with a specific chemical modification. So the scientists tried making the same change to mRNA made in the lab before injecting it into cells. It worked: The mRNA was taken up by cells without provoking an immune response.

More:
How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents - The New York Times

Leptin is a hormone that comes from fat cells. It helps control food intake by sending signals about hunger to the hypothalamus in the brain. This process regulates appetite.

Leptin regulates energy levels by maintaining a balance between hunger and appetite. The hormone triggers the body to respond by eating more when energy levels are low and eating less when energy levels are stable or high.

People who have high levels of body fat have high circulating levels of leptin.

Research shows that having elevated leptin levels can lead to leptin resistance, making weight loss difficult.

This article looks at what the leptin hormone is, what the leptin diet involves, and the advantages and disadvantages of following the leptin diet.

Scientists discovered leptin, a protein that functions as a hormone, in 1994.

Leptin is one of the main hormones responsible for maintaining body weight. Leptin helps people balance how much food they consume by regulating hunger levels. The hormone also controls how much energy a person uses throughout each day.

Leptin comes from fat cells within the body. It enters the blood supply and travels up to the brain. The hormone must cross the blood-brain barrier, a membrane that protects the brain from harmful toxins, to get to the hypothalamus. The hypothalamus is the area in the middle of the brain that controls hormone regulation, among other important functions.

At the hypothalamus, leptin can function by signaling that the body does not need any more food. This response causes the person to feel full. If leptin levels are low, or leptin does not reach the hypothalamus, a person will continue to feel hungry.

Leptin regulates body weight and is an important marker for energy storage. This means if the body has excess energy stored as fat, leptin signals the hypothalamus to reduce appetite and burn excess body fat for fuel. This response helps a person maintain a moderate body weight.

However, when a person has high amounts of body fat, they can develop a resistance to leptin, which leads to abnormally high leptin levels.

Having leptin levels that are too low is less common. Low leptin levels can occur in severe childhood obesity and delayed puberty.

When leptin levels are below average, the brain thinks no body fat is present. This reduced level can cause symptoms of uncontrollable hunger, resulting in excessive food intake. Leptin injections are a way of reducing this problem.

After scientists discovered the hormone in 1994, Byron J. Richards created a diet named after it: the leptin diet.

The goal of the leptin diet is to return leptin levels to normal and create balance within the body. The leptin diet has five main principles:

The leptin diet permits most types of food, but guidelines suggest avoiding chemical additives and processed sugars and sticking to fresh and organic produce.

The leptin diet encourages other lifestyle changes, such as getting plenty of sleep and participating in regular physical activity.

A 2021 study suggests that diets high in fat, carbohydrates, fructose, and sucrose and low in protein are drivers of leptin resistance. The researchers concluded that leptin resistance might be reversible by reducing calories.

However, this research has some limitations, such as small sample sizes, so further evidence is required to verify these claims.

The leptin diet includes limiting snacking and shortening your daily eating window. If a person reduces how much they snack, this could create a calorie deficit necessary for weight loss.

A leptin diet is a sensible approach to weight loss for some people, as the diet promotes eating healthily without harsh restrictions but encourages a routine.

However, at present, no studies are investigating the effects of the leptin diet on weight loss and leptin levels.

It is important to remember that all bodies are different, and a diet that meets the nutritional demands of one person will not always work for someone else.

For example, limiting the number of meals to three per day and cutting out snacking may be effective for a person with a low activity level. However, it is unlikely to meet the energy demands of a person who leads an active lifestyle, exercises intensely, or has a physically demanding job.

Many factors can impact energy needs, including age, pregnancy, breastfeeding, and certain medical conditions.

A person should consider consulting a healthcare professional like a registered dietitian if they are interested in improving health through dietary changes.

Increasing research suggests that obesity causes people to develop leptin resistance.

When someone carries an excessive amount of body fat, they will have too much leptin circulating in the blood. This excess results in that person becoming leptin-resistant. This resistance means their brain stops responding to the leptin signals traveling up to it. It also means their body continues to produce leptin, contributing to elevated leptin and leptin resistance.

Research suggests that weight loss and energy-restricted diets may help reverse leptin resistance.

Leptin is the hormone that controls appetite. Leptin informs the brain when a person has eaten enough, reducing appetite, and produces hunger signals when a person requires energy.

As with any weight loss plan, a person should approach the leptin diet with caution. The diet may be effective for some people, but it may not meet the nutritional demands of every person. Check with a doctor before starting any significant weight loss diet.

Link:
Leptin: Benefits and risks of the leptin diet - Medical News Today

CAR-T cell therapy has been a boon for treating blood cancers. Since the technology was first brought to the clinic, CAR-T has offered patients months or years of life after they had exhausted all other treatment options and would have died within weeks.

Its been incredible, said Marcela Maus, an immunologist and cell therapist at Mass General Cancer Center. Weve seen patients who had multiple lines of therapies and progressed after all of those, [then] get CAR-T and go into long-term remission.

But CAR-T does have hefty limitations, and scientists like Maus are researching ways to overcome some of its major shortcomings. These issues have prevented CAR-T from being used safely and effectively outside of leukemia and myeloma, and even patients who have responded spectacularly to CAR-T usually see their cancers return. The therapies are also still incredibly costly and carry risks, including a reaction known as a cytokine storm that can be life-threatening.

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Potential solutions to these problems are still in the early stages, but scientists are beginning to get a vision of what the future of CAR-T cell therapy might look like. It could involve synthetic biology to engineer a more advanced cell, or engineering other parts of the T cell to make it work better in the challenging environment around a tumor.

The field is growing tremendously, Maus said. Different people are working on different issues then, ideally, the data kind of decides whats going to be the next big thing.

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Heres a look at what experts see as some of the most promising approaches.

Current CAR-T cells use their CAR, or chimeric antigen receptor, to identify and kill cancer cells. These are synthetic proteins that bind to a specific target, like a protein on a cell surface membrane, and then activate the T cell to kill any cell carrying this target.

Armed with a CAR, T cells become pros at killing cancer cells that have their target, but theyll also kill normal cells that happen to carry the protein, too. Once a CAR-T cell is in the body, there isnt much a clinician can do to rein it in if it starts causing a lot of toxicity.

Once we let the CAR out, theyre like teenage kids, Maus said. You can maybe watch, but you cant really control them. So, theres some desire to be able to turn them on or off at will.

So, researchers are also trying to create CAR-T cells that they can manually activate or deactivate. As a group, these are known as controllable CARs, and most work by engineering an additional genetic circuit in the CAR-T cell. In theory, clinicians should be able to activate a switch on the genetic circuit that induces the CAR-T cell to activate their CAR and express it on the T cells surface membrane, thereby activating the receptor. Then, after a while, the T cell will disarm.

The goal is really getting our hands back on the steering wheel for a bit, Maus said.

There are several ways that synthetic biologists are doing this. In one example, researchers engineered a CAR with a protein switch that activates the receptor in the presence of blue light. In another example, researchers added a gene to CAR-T cells that force it to create its CAR and express it on the cell surface, thereby activating it, only in the presence of ultrasound radiation.

That way, it can be focused into a specific location, said Peter Yingxiao Wang, a synthetic biologist at University of California, San Diego, who works on controllable CARs. When the light or ultrasound is on the tumor locally, they can activate the CAR gene to trigger killing. Anywhere else, the CAR T-cells will be benign.

The idea is that the clinician can focus the light or ultrasound onto the tumor to get CAR-T cells to begin killing there. Once that signal is turned off, the CARs should disarm or slowly degrade and deactivate the CAR-T cells killing function. This way, even if the CAR does kill healthy tissue, the damage will theoretically be limited to the area around the tumor.

But this is an infant field right now, Wang added. A lot of these studies are just proof of concepts to show that theyre technically achievable. If you want to move to clinical trials, all of the components must be optimized.

Scientists also must show that theyre truly safe in humans, and that keeping the damage to a smaller surface area will be enough to outweigh the risks in treating tumors located near vital organs like the heart.

Other researchers are working on developing new CARs that can function like a biomolecular computer, able to make simple logical decisions to target cancer cells. Conventional CARs can cause dangerous toxicity because they only use one protein to identify cancer cells, and it may be impossible to discover the perfect target that exists only on cancer cells and not at all on healthy cells.

You can never uniquely define cancer or any other healthy tissue just by one marker, explained Wilson Wong, a synthetic biologist at Boston University. It just doesnt work. Its like trying to find a person and saying, he has black hair. Its like, oh, my God, youll never find him.

But it might be possible to distinguish cancer cells from healthy ones by looking at multiple proteins on a single cell. So, researchers like Wong have begun building more advanced CAR T-cells that use genetic circuits that only activate a CAR under more complex conditions, like the presence of several specific proteins that arent often seen in combination on healthy cells.

In this sense, the CAR is making a logical decision like basic Boolean computing, and synthetic biologists call this technique logic-gating.

Theres a lot of cool genetic circuits you can build, said Yvonne Chen, a synthetic biologist at UCLA. One can think of conditional systems to obliterate cancer cells. One can build OR-gates, AND-gates, and NOT-gates, and layer them on top of one another.

Although, Chen added, a drawback of logic-gating is that by increasing the complexity of the system, you might also be increasing the chance something goes wrong. Its important not to overcomplicate the design. Sophisticated circuits are exciting, but sometimes the solution itself causes problems. For example, for an AND-gate, you also make it easier for the tumor to escape. If the tumor loses either target A or B, it escapes from therapy, she said.

Another issue with conventional CAR-T therapy is that after a while, T cells can simply stop working. Solid tumors, like lung or pancreatic cancer, often have strategies to defend themselves from immune system attacks, including those from CAR-T cells. That makes it harder for CAR-T cells to treat solid tumors and can provide an opening for the tumor to return or progress.

So, researchers like Chen are working on armoring the CAR T-cell against the hostile signals in the microenvironment around a solid tumor. One of these signals is called TGF-beta, a protein which can help shut down T cell activity and help cancer cells avoid death and detection from the immune system. Chen was able to create a CAR cell that is not only resistant to TGF-beta, but can actually subvert the signal and become more deadly when it encounters TGF-beta.

Instead of being dysfunctional, they become activated, Chen said. That actually converts a tumor defense mechanism into a stimulatory signal for our T cells and tells them, youre in an environment where youre likely to encounter a tumor cell. Get ready.

Other scientists are working to keep CAR-T cells which can lose power over time functional for longer. Even with a good antigen, the T cells rapidly lose function, said Shivani Srivastava, an immunologist at the Fred Hutchinson Cancer Research Center who works on this problem. If you trigger a T cell or CAR over and over again, that causes the cell to become exhausted rather than turning into a memory cell or something else.

In one case, Stanford immunologist Crystal Mackall engineered a CAR-T cell that takes breaks before returning to work. She did this by creating a transient CAR that can be turned on or off. It can enhance [the T cells] function and limit how exhausted they are by giving them periodic rest, Srivastava said. Thats a really interesting strategy in principle.

But most of the tactics that scientists have tried so far in the realm of armored CAR-T cells havent worked in the long term, Srivastava said. You need a strategy that can help the CAR T-cells persist long enough to eradicate the cancer and prevent its return, which might be a lifelong project for the immune system.

Well have to find the right combination that will be durable, she said. Often we can find strategies that enhance function for only a short period of time.

Some future approaches might see T cells abandoned altogether. Scientists are slapping synthetic receptors on new or different cell types, such as natural killer cells. One company, called CoImmune, is putting CARs on a synthetic cell called a CIK cell, or cytokine-induced killer cell.

This is a novel cell type. They dont occur in nature, explained Charles Nicolette, the biotechs chief executive.

Theyre made by taking white blood cells and growing them while exposing them to certain immune molecules called cytokines. The advantage of creating new cell types is that biologists can combine certain useful traits from other immune cells, Nicolette said. For example, CIK cells could have the NK cells natural ability to distinguish normal cells from malignant ones and the CAR T-cells enhanced ability to kill.

One day, UCLAs Chen hopes to take this concept even further. To her, the ideal cancer-killing cell would not be derived from anything biological, but a completely artificial cell.

Instead of taking a cell from a patient, but rather build a completely defined, minimal cell that can do what we want and nothing else. It cannot evolve. Cannot mutate. Then, self-destruct when you dont want it there, she said. But, she added, creating synthetic cells like that would be unimaginably challenging, and it might not be possible to create a cell thats both persistent but also unchangeable.

Still, a scientist can dream.

Read more:
STAT's guide to the next generation of CAR-T therapies - STAT

TheCancer Gene Therapy market report is a useful foundation for people looking out for a comprehensive study and analysis of the Cancer Gene Therapy . This report contains a diverse study and information that will help you understand your niche and concentrate on key market channels in the regional and global space. To understand competition and take actions based on your key strengths you will be presented with the size of the, demand in the current and future years, supply chain information, trading concerns, competitive analysis and the prices along with vendor information. The report also has insights about key players, applications of Cancer Gene Therapy , its type, trends and overall market share.

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The research covers the current Cancer Gene Therapy market size and its growth rates based on records with company outline of Key players/manufacturers: GlaxoSmithKline plc, Adaptimmune Therapeutics plc, Merck & Co., Inc., bluebird bio, Inc., Shanghai Sunway Biotech Co., Ltd, Celgene, SIBIONO, Anchiano Therapeutic, Achieve Life Sciences, Inc., and Synergene Active Ingredients Pvt. Ltd.

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COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets. Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Regional Analysis

All the regional segmentation has been studied based on recent and future trends, and thedomainis forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Cancer Gene Therapy market report are U.S., Canada, and Mexico in North America, Germany, France, U.K., Russia, Italy, Spain, Turkey, Netherlands, Switzerland, Belgium, and Rest of Europe in Europe, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, China, Japan, India, South Korea, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), and Argentina, Brazil, and Rest of South America as part of South America.

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Table of Contents

Chapter 1: Cancer Gene Therapy Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3: Global Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis

Chapter 12: Global Cancer Gene Therapy Market Forecast

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Cancer Gene Therapy Market 2021 Industry Outlook, Comprehensive Insights, Growth and Forecast 2031 | Celgene, SIBIONO, Anchiano Therapeutic, Achieve...