2024 is already shaping up to be a landmark year with groundbreaking FDA approvals. With the first half of the year aimed at cell & gene therapy, the second half of 2024 is equally thrilling with key FDA decisions on the horizon: 🧠 Eli Lilly and Company's donanemab for Alzheimer's - promising better cognitive outcomes. 💪 Adaptimmune’s afami-cel for synovial sarcoma - a potential game-changer in T cell therapy. 💊 Lykos Therapeutics’ MDMA for PTSD - could be the first psychedelic drug approved for a neuropsychiatric disease. 🧬 Bristol Myers Squibb's KarXT for schizophrenia - set to be the first new class in decades. 🎯 Daiichi Sankyo US & AstraZeneca's Dato-DXd for non-small cell lung cancer - aiming to join the elite ADC club. Stay tuned for these exciting developments! 🌟 #Biotech #FDA #Innovation #Healthcare https://lnkd.in/eR998NUX
Jennifer Iantosca’s Post
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Managing Consultant @ PA Consulting | Biochemical Engineer | Cell & Gene Therapies Enthusiast
Day 4 of celebrating good news in cell and gene therapies: Promising study using HER2 targeting CAR-T to treat glioblastoma (the most common and aggressive malignant primary brain tumor in adults) conducted by a Chinese research group at Second Hospital of Dalian Medical University. With positive results in both in vitro and in vivo mouse models, while still far from the clinic, this can be a potentially curative treatment for glioblastoma which currently has a five-year survival rate of roughly 10%, with a median of 15- to 18-month survival after diagnosis. CAR-T may be able change that for patients in a few years' time. Very exciting piece of research! 🎉 https://lnkd.in/dKbrf738 #CGT #celltherapies #genetherapies #celebrate #monthofCGTcelebrations
HER2-targeting CAR-T cells show highly efficient anti-tumor activity against glioblastoma both in vitro and in vivo - Genes & Immunity
nature.com
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Associate Director, Digital Experiences and Marketing Technology at Charles River Laboratories
We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/IgAp97 #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
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We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/qmAofK #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
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We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/Q86wAf #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
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Business Development Manager Central/South Spain Charles River Laboratories
We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/E4uxk3 #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
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Passionate about cell therapy and bringing treatments from clinical initialization to commercialization and beyond through process development, validation, and implementation.
It is important to put risks in context
Founder at Rx Group 🧬 Helping biotech’s bring safer medicines to patients, by finding them the best quality assurance talent. 🌎
Rollercoaster couple of weeks in biotech. FDA approved two gene therapies last week - huge news for the industry and for patients and families affected by sickle cell disease. Congrats to all involved at bluebird bio and CRISPR Therapeutics and Vertex Pharmaceuticals. The week before, the FDA announce a cancer safety review for autologous CAR-T cell therapies, citing a 'serious risk' of secondary cancer following treatment...queue the subsequent tumble in share price for many of those in the autologous car-t space. The incidence rate of T-cell malignancies following on from treatment of these autologous car-t cell therapies is less than 0.1%. In numbers, that is 12 reported T-cell lymphoma cases out of nearly 18,000 patients. The industry needs confidence and momentum to keep the flow of investment into area's like CGT, and in particular CAR-T cell therapies that are potentially vulnerable right now. As the FDA said "The benefits of these products continue to outweigh their potential risks for their approved uses". Just read the stories of Emily Whitehead, Laurie Adami and thousands more, if you want to hear how these therapies have saved lives. #biotech #cgt #cartcelltherapy #genetherapy
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We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/lwAQ2h #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
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I help biotechs successfully conduct FDA-compliant trials, saving them 70% on costs 💊 ▸ High-Quality Research ▸ Up to 43.5% Aus R&D Grant ▸ No IND needed ▸ 8 Weeks Avg Startup ▸ FDA Expertise
Immusoft Administers the First Engineered B Cell in a Human Clinical Trial Immusoft received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS I Sean Ainsworth, CEO, Immusoft. “This is the first patient in the world to receive an engineered B cell therapy – a major accomplishment for Immusoft and a major advancement in cell and gene therapy.” MPS I is a rare, lethal genetic disease that in its severe form is lethal in the first decade of life. It affects the body’s ability to produce IDUA (alpha-L-iduronidase), which is an essential enzyme that helps to break down long-chain sugars inside cells. When the sugar chains cannot be broken down and disposed of, they accumulate in the cells and cause progressive damage. This accumulation can happen in the tissues, including the brain. Severe MPS I occurs in about 1 in 100,000 births, and symptoms appear within a child’s first year of life. In what is referred to as attenuated MPS I, symptoms appear later in childhood. It occurs in about 1 in 500,000 births.
Immusoft Administers the First Engineered B Cell in a Human Clinical Trial
businesswire.com
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Advanced Therapeutics | Plasmid, Vector, Cell Therapy Manufacturing | GMP Analytic Testing
We are proud to continue our collaboration with Rznomics Inc. who recently received both Orphan Drug and Fast Track designations from the FDA for RZ-001, underscoring the potential clinical impact of this pioneering RNA-based gene therapy on both liver and brain cancers. "This signifies an important milestone in developing a safe and effective treatment for patients with GBM who require new therapeutic options." Dr. Seong-wook Lee (CEO) Glioblastoma multiforme (GBM), the most common malignant brain and central nervous system (CNS) tumors, accounts for around half of all cases, while hepatocellular carcinoma (HCC), the most prevalent type of primary liver cancer, accounts for 80% of cases globally. Read the story in full: https://okt.to/MrUgSi #genetherapy #RNAtherapy #anticancer #orphandrug #livercancer #HCC #braincancer #GBM James Cody
Promising Anticancer Gene Therapy Makes Headway
criver.com
