Biocom California’s Post

𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐚𝐧𝐝 𝐏𝐡𝐚𝐫𝐦𝐚 𝐔𝐩𝐝𝐚𝐭𝐞𝐬: December 15 - 16, 2024 Novo Holdings & Catalent deal full steam ahead after FTC ok, Promise Bio launches with AI-driven precision medicine for autoimmune diseases ambitions, Candid Therapeutics signs on trio of bispecific R&D deals, Roche discontinues Ph3 gene therapy asset development + 30 more stories Get the full newsletter on the TLDR Biotech website: https://lnkd.in/g8W7zgbd

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Sangamo Therapeutics, Inc. Therapeutics’ stock has plummeted by 50% in premarket trading after Pfizer ended the companies’ partnership to co-develop a gene therapy for haemophilia A. Click here for the full article - https://lnkd.in/dU-_dxat #pharma #genetherapy #haemophilia

Cell and gene therapies (#CGTs) are vital scientific breakthroughs for many patients with complex and rare diseases. More are on the way, with 66 new approvals anticipated by 2032. Our analysis offers a clear visualization of the current obstacles faced by patients and their caregivers that need to be minimized to help patients reach their full potential through these transformative therapies.    I’m pleased to work with Tyler Wagner and Brian Sils to publish this new white paper from the National Pharmaceutical Council: https://lnkd.in/ew5SdUQf

New Regulatory Intelligence from Ben Kaspar, April Nguyen, PharmD, RAC, MPH, and team! Earlier in November, the FDA Center for Biologics and Research (CBER) released a new FAQ for developers of cellular and gene therapy (CGT) products. Sponsors of CGT products planning INTERACT, Type-D, Pre-IND or pre-BLA meetings with the Agency will benefit from starting their question development with a detailed review of this guidance. Gain a quick overview here: https://hubs.li/Q02-H2xF0 #OneMMS #RegulatoryStrategy #CBER #IND #BLA #CGT #CellandGeneTherapy

#Cardiometabolic risk factors are still among the leading drivers of both global mortality and morbidity. Over the past 5 years, the number of clinical-stage cardiometabolic assets has increased by 13%, and today, it accounts for 7% of the overall industry R&D pipeline. This includes cell, gene and RNA therapeutics. Explore the latest trends in cardiometabolic breakthroughs, the market opportunity, and critical success factors for innovators. Download the white paper to learn more: https://bit.ly/3ZeOCWf #obesity #clinicaltrials

In the buildup to the JPM 2025 global healthcare conference, Lori Ellis, Head of Insights, BioSpace is speaking with Ali Pashazadeh, Treehill Partners CEO, and Miguel Forte, President of the International Society for Cell & Gene Therapy, a board member of ARM, and CEO of Kiji Therapeutics about the current malaise within investment for the life science and biopharmaceutical sectors, and what companies can do to survive. This is episode 1 of the discussion: https://lnkd.in/e9pXZaud

Asuragen, a Bio-Techne brand has established the market-leading technology for the reliable amplification and analysis of CGG triplet repeats in the FMR1 gene. The AmplideX PCR/CE C9orf72 Kit (RUO) is an expansion of this technology into the neurogenetics space. The kit provides clinical and pharmaceutical researchers with a reliable and reproducible method for high-resolution genotyping of hexanucleotide repeat expansions in the C9orf72 gene, which is of increasing interest in frontotemporal dementia (FTD) – the second most common form of early onset dementia after #Alzheimers Disease – and amyotrophic lateral sclerosis (ALS). Learn more: https://lnkd.in/ebKsEAHB

Great insight for connections working in Cell and Gene Therapy pipelines! Message me to meet with our Regulatory team to discuss the strategy behind this as you plan to interact with the FDA in 2025! #OneMMS #RegulatoryStrategy #CBER #IND #BLA #CGT #CellandGeneTherapy