BioPioneers

CSL Behring B.V. is closing its Pasadena, California, R&D hub for #cell and #genetherapy by January 2025. This decision is part of a strategic shift away from ex vivo #lentiviral #vector-based gene therapies, with operations relocating to Waltham, Massachusetts, to focus on #mRNA and #lipid #nanoparticle technologies. This move reflects a broader industry trend of reassessing ex vivo gene therapy approaches. bluebird bio, a pioneer in lentiviral vector (LVV)-based gene therapies, is facing financial difficulties despite having three approved products: #Zynteglo, #Skysona, and L#yfgenia, prompting a company restructuring. TAKEDA PHARMACEUTICALS AMERICA, INC. has ceased its early-stage research on adeno-associated virus (#AAV)-based gene therapies Similarly, in early 2023, GSK announced its decision to end investments in cell and gene therapy. Positive Developments in the Sector Despite these challenges, there are notable positive developments: - Kyowa Kirin, Inc.- U.S. Kirin's Acquisition of Orchard Therapeutics - U.S.: In the #hematopoietic stem cell gene therapy space, Kyowa Kirin acquired Orchard Therapeutics for $387 million upfront. Orchard's gene therapy for metachromatic leukodystrophy, known as Libmeldy, received FDA approval in March 2024. - AstraZeneca's Expansion into Gene Therapy: AstraZeneca's rare disease unit, Alexion Pharmaceuticals, Inc., acquired Pfizer's portfolio of preclinical gene therapy projects, signaling a strategic expansion into the gene therapy domain. - Roche's Collaboration with Dyno Therapeutics: Roche has expanded its partnership with Dyno Therapeutics to design novel AAV vectors, aiming to enhance the delivery mechanisms for gene therapies. https://lnkd.in/esZCmvCN

Neurogene Inc. has discontinued the high-dose cohort of its NGN-401 (MECP2) #genetherapy trial for #Rettsyndrome following a serious adverse event (#SAE) in a patient who developed systemic hyperinflammatory syndrome after receiving high dose #AAV on November 5, 2024. The U.S. Food and Drug Administration (#FDA) has reviewed the safety data and permitted the continuation of the trial at the low-dose level. The company plans to continue the low-dose cohort and will work closely with regulatory authorities to ensure patient safety and the integrity of the #clinicaltrial. Rett Syndrome Research Trust International Rett Syndrome Foundation Northwest Rett Syndrome Foundation #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation https://lnkd.in/g_tAZizj

#BayArea #LifeSciences #Event #Announcement Biomarkers and Brews: Proteomic Innovations with Akoya Biosciences & Olink Join us for a night of learning about cutting-edge #proteomic #innovations with Akoya Biosciences, Inc. & Olink Proteomics, all while enjoying food & brews! Date and time: Doors at 4:45pm Location: Devil's Canyon Brewing Co., 990 Bayport Avenue San Carlos, CA 94070 About this event Biomarkers and Brews: Proteomic Innovations with Akoya Biosciences & Olink Come join us for an exciting event at Devil's Canyon Brewing Co. where we'll dive into the latest #proteomic #innovations with experts from Akoya Biosciences and Olink. Discover how #biomarkers are revolutionizing the field and enjoy some delicious brews while #networking with fellow enthusiasts. Don't miss out on this unique opportunity to learn and mingle in a relaxed setting! #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation https://lnkd.in/gk_-qc7d

Novartis has acquired Kate Therapeutics, a San Diego-based #genetherapy company, in a deal valued at up to $1.1 billion. Kate Therapeutics specializes in #preclinical programs targeting Duchenne muscular dystrophy (#DMD), facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (#DM1). The company employs its proprietary DELIVER platform, which utilizes directed evolution of adeno-associated virus (#AAV) capsids to create #liver-#detargeted, muscle-tropic #vectors. Acknowledging recent challenges in muscular dystrophy treatments, such as Pfizer's investigational gene therapy for DMD failing to demonstrate efficacy in a Phase 3 trial, Novartis CEO Vas Narasimhan emphasized the company's commitment to learning from past experiences in the #musculardystrophy domain. By acquiring Kate Therapeutics, Novartis aims to leverage innovative #delivery #technologies to overcome previous obstacles in gene therapy for #neuromuscular #diseases. Muscular Dystrophy Association Muscular Dystrophy UK Muscular Dystrophy Canada Facioscapulohumeral Muscular Dystrophy Duchenne UK Duchenne Heroes #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting https://lnkd.in/giRnS_6G

Novartis has partnered with Vyriad to develop next-generation #invivo #CART #celltherapies, utilizing Vyriad's #lentiviral vector platform to #engineer T cells directly within #patients' bodies. This collaboration aims to streamline treatment processes and enhance accessibility for patients with unmet medical needs. Novartis is not the only one on the hunt for in vivo T cell therapies. Umoja Biopharma presented preclinical data in 2023 demonstrating the successful generation of CAR-T cells in #nonhumanprimates using their VivoVec™ #genedelivery #platform - supporting plans to initiate a Phase 1 trial of their lead candidate, UB-VV111, targeting #hematologic #malignancies. Sana Biotechnology, Inc. initially invested in developing in vivo CAR-T therapies but discontinued it in Oct 2023 and reducing its workforce by 29% to optimize resources. #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting #CRISPR  https://lnkd.in/gtBpTqwm

Today we approved an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency. https://lnkd.in/er5tacke

📢 #BayArea #LifeSciences #Event #Announcement AI x Bio: Perspective from those building on the front lines "AI x #Bio: Perspectives from Those Building on the Front Lines" is an event focused on the transformative role of AI within #techbio. Attendees will hear from #innovators who are actively developing AI-driven solutions to advance #biotechnology, from precision #bioengineering to #syntheticbiology and beyond. Brought to you by Bits in Bio and LifeX.vc https://lnkd.in/gtRvzxHX

📢 #BayArea #LifeSciences #Event #Announcement NeuroAge Test Launch Party Date and Time: Thursday, December 5, 5:30 PM - 8:30 PM Location: 135 Mississippi St, San Francisco, California NeuroAge Therapeutics is excited to officially launch the NeuroAge Test! This prognostic test can tell you how old your brain is and your future risk of dementia more than 30 years ahead of time. It has 4 parts: brain MRI, cognitive testing using our software, NeuroGames, RNA-based blood biomarkers, and genetics, which we combine using AI to give you a brain aging score. We will be raffling off a NeuroAge Test during the party. If you are curious about your biological brain age, you can play NeuroGames for free up until the party. These games will let you know how your memory and reaction time compares to the average person your age and will assess your biological brain age. MBC BioLabs https://lnkd.in/ghc4Di37

📢 #BayArea #LifeSciences #TechEvent #Announcement Fireside Chat & Networking: From Harvard Innovation Labs to Startup Success Join us for an engaging fireside chat with Philip Greenwald, a seasoned entrepreneur and ex-Associate Director at Harvard Innovation Labs and Cynthia Luo. Philip has mentored over 70 founders, helping them raise over $250 million, making him a pivotal figure in academic innovation and startups. Cynthia Luo is an AI researcher, investor, and entrepreneur. She is a Research Affiliate at MIT. She holds a PhD in AI and was a Research Fellow at the MIT Media Lab. Key Topics Insights and Lessons from Entrepreneurial Endeavors The Crucial Role of University Innovation Labs in Startup Development Overcoming Startup Hurdles: From Lab to Market Event Schedule 4:30 PM - 5:00 PM: Networking Session 5:00 PM - 6:00 PM: Fireside Chat 6:00 PM - 7:00 PM: Networking Session https://lu.ma/lvx6d9bh

#BayArea #LifeSciences #Event #Announcement Biotech Networks Speed Networking San Francisco December 2nd 2024 Event Information Who: Life science #researchers and biotech #professionals in the #SanFrancisco bay area Date and Time: Monday December 2nd 2024 6:00-9:00 p.m. Location: Harmonic Brewing Thrive City (Chase Center), 7 Warriors Way, Suite 206, San Francisco , CA 94158 Cost: $35 General Admission, $85 for Sales/Consultants. Includes casual dinner, drink ticket, Scientific Speed Networking™ Register/Event Page: sfbn.org/bndec24 also RSVP to LinkedIn Event #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation https://lnkd.in/grnt4PsC