Los Angeles Biotech Networks’ Post

Capsida Announces AbbVie Opt-in for First Genetic Medicine Program from Neurodegenerative Disease Collaboration https://lnkd.in/gKNBbHPr Capsida to receive a $40 million license payment and is eligible for additional milestones and royalties THOUSAND OAKS, Calif., Jan. 7, 2025 /PRNewswire/ — Capsida Biotherapeutics (“Capsida”) today announced that AbbVie has exercised an option [...]

🏆 #recognition So proud to be on GEN's first A-List of 10 Companies to Watch in #AI Drug Discovery. It's clear that we made the list because of our robust pipeline, which the article did a good job highlighting. - Phase 2 #Oncology Trials: BPGbio’s BPM31510, a novel lipid nanodispersion, showed significant improvements in survival in Phase 2 trials for #pancreatic cancer. An on-going Phase 2b trial for #GBM is also progressing well. - Rare Pediatric Disease Phase 3 Trials: BPGbio is planning a #Phase3 trial for BPM31510, targeting primary CoQ10 deficiency to address unmet medical needs for patients. BPM3150IV for Primary CoQ10 Deficiency and BPM31510T for Epidermolysis Bullosa were respectively granted Rare Pediatric Disease Designations by the FDA recently, marking two Rare Pediatric Disease Designations for the BPM31510 franchise. #primarycoq10deficiency #rarepediatricdiseasedesignation #PRV - Protein Degradation Breakthrough: BPGbio pioneered its #E2-based Targeted Protein Degradation (#TPD) program, drugging the long considered undruggable and unlocking new treatments in oncology, #cardiology, and #neurology with improved efficacy and reduced resistance risks.

There is a lot of advancement happening globally using the tools of AI. In drug discovery, using AI in an appropriate way to build a pipeline of effective solutions for patient health - therapeutics and diagnostics - that is where the rubber meets the road. Pipeline is key.

MEET US AT - Our CSO Dr Thierry Bordet and Head Pharmacology & Toxicology Dr Karine BIGOT will be in Valencia, Spain next month attending the European Association for Vision and Eye Research 27th EVER Congress. They will be presenting data in an oral presentation (ID# T.129) and a poster (ID# T.063) on our lead program PST-611, a plasmid expressing human transferrin, in dry age-related macular degeneration (AMD)/geographic atrophy (GA). PST-611, which has a very good safety profile, is a first in class non-viral vectorized gene therapy encoding human transferrin, a highly potent iron chelator. By restoring normal iron homeostasis, transferrin mitigates the toxic effects related to excess iron and protects retinal cells, offering the potential to slow GA lesions growth and improve patient’s visual function. Get in touch to meet up and find out more – email [email protected] Read about our approach and therapies in development here – www.pulsesight.com #AMD #GA #genetherapy #opthalmology #drugdevelopment #blindness #retinaldisease

Sanofi, Alloy Therapeutics, Inc. partner for AntiClastic Antisense Platform Under the collaboration, Sanofi will use its neuroscience expertise and Alloy’s novel AntiClastic Antisense Platform to develop a novel class of genetic medicine capable of crossing the blood-brain barrier. Alloy Therapeutics genetic medicines chief scientific officer Vinod Vathipadiekal said: “At Alloy Therapeutics, we’re transforming RNA therapeutics by bridging foundational insights with modern innovation. Read more: https://lnkd.in/eJEidpTR #pharmanews #pharmaceuticalindustry #drugdiscovery #drugdevelopment #genetictherapeutics #worldpharma

Our Chief Scientific Officer, Alice Brown, recently presented at The Chartered Institute of Patent Attorneys annual Life Sciences Conference, the UK’s premier educational and #networking event for patent and intellectual property (IP) professionals in the #pharma, medtech and #biotech sectors. Alice’s presentation, ‘Purespring: Gene Therapy to Cure Kidney Disease,’ provided senior leaders in the IP field with a comprehensive overview of Purespring’s scientific and corporate journey, from our founding in 2020 to recent milestones such as our first presentations of preclinical data on our novel #GeneTherapy programme at leading nephrology congresses. #KidneyDisease #Conference #People

Interesting- reclassifying APOE4 homozygotes as a genetic form of AD. We still don't know why APOE4 carriers have more side effects from the new anti-amyloid medications, but it's clear that we need to segment AD by genetics and biomarkers.

Don't miss our poster presentations at Discovery on Target in Boston, MA! 📃 "PCKD, the Most Relevant Model for ADPKD" 🏷 Session A, #A40 📆 October 1st to 2nd a.m. 👨🏼🔬 Laurent Saniere, PhD, SVP Pharmacology & DMPK Abstract: Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder, affecting 12 million people globally. It is characterized by the development of fluid-filled kidney cysts, which often leads to kidney failure. The vasopressin receptor 2 antagonist, Tolvaptan, is the only approved treatment that slows the progression of ADPKD. Nevertheless, there is still a high unmet need for treatment options for patients with ADPKD because Tolvaptan has limited efficacy and non-negligible side effects. For more questions and details, come and meet our team at our booth 508: https://lnkd.in/edM4ecJj Visiopharm #DrugDiscovery #Pharmacology #ADPKD #PCKD #model #KidneyDisease #DiscoveryOnTarget #BostonDOT24

Exciting Breakthrough in Hemophilia B Research! 🌠 The F9 knockout mouse model offers a promising avenue for advancing hemophilia B research. By mimicking the genetic deficiency seen in patients, this model provides invaluable insights into the disorder's underlying mechanisms. ✴ Additionally, it serves as a robust platform for testing new therapeutic treatments, accelerating the development of interventions for hemophilia B. Cogen's commitment to advancing hematology research through customized models further solidifies its pivotal role in driving transformative discoveries in this field. Join the conversation by sharing your thoughts on the potential impact of F9 knockout mice in hemophilia B research! 💪 #HemophiliaB #MedicalResearch #preclinicalresearch #scienceandtechnology #research #researchanddevelopment #biotech #biotechnology #sciencecommunication #preclinical #preclinicalresearch

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