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Inaxaplin: A Breakthrough APOL1 Channel Inhibitor for a Genetically Defined Kidney Disease | https://lnkd.in/g5j7MdSV Inaxaplin (VX-147), developed by Vertex Pharmaceuticals is an inhibitor of APOL1 (apolipoprotein L1) channel activity currently in a Ph. II/III pivotal study for the treatment of chronic kidney disease caused by specific variants of the APOL1 gene. It was recently granted Breakthrough Therapy designation by the FDA and PRIME designation by the EMA. Notably, inaxaplin has been called “the most important genomic-driven drug discovery for [chronic kidney disease] this century”, acting on a target with a fascinating human genetic validation story. Check out our full case study on inaxaplin discussing how a thallium flux assay was used to identify a submicromolar hit, how MetID studies informed discovery strategies, the most recent clinical updates, its synthesis, and more. Article | https://lnkd.in/g5j7MdSV

INAXAPLIN: "THE MOST IMPORTANT GENOMIC-DRIVEN DISCOVERY OF THIS CENTURY (for chronic kidney disease) Persons with toxic gain-of-function variants in the gene encoding apolipoprotein L1 (APOL1) are at greater risk for the development of rapidly progressive, proteinuric nephropathy. Despite the known genetic cause, therapies targeting proteinuric kidney disease in persons with two APOL1 variants (G1 or G2) are lacking. INAXAPLIN is an inhibitor of the function of the APOL1 channel. Tested in preclinical models, in vitro, in vivo in an APOL1 G2 homologous transgenic mouse model, and in a Phase 2a clinical study on 13 patients, administering the drug for 13 weeks (15 mg for 2 weeks and 45 mg for 11 weeks) , together with standard therapy, inaxaplin reached the adherence threshold, the mean change from baseline urinary protein/creatinine ratio at week 13 was -47.6%. Adverse events were mild or moderate in severity; none led to the interruption of the study. [N Engl J Med 2023; 388:969-979; https://lnkd.in/eKRMnJD3] In conclusion, targeted inhibition of APOL1 channel function with inaxaplin reduced proteinuria in participants with two APOL1 variants and focal segmental glomerulosclerosis. #inaxaplin #APOL1 #proteinuric #nephropathy

There are just over 2 weeks until we open our doors at the 4th Gene Therapy Comparability Summit in Boston (February 13-15). Comparability can turn into a significant bottleneck in filing for approval with regulators if drug developers aren’t prepared for it. If your company is at any stage from filing for IND through to running a pivotal clinical trial, this program needs to be a priority. So far industry leaders from the likes of Spark Therapeutics, CSL, Bayer, Genethon, AbbVie and Regeneron have secured their place to join 60+ experts dedicated to implementing an effective comparability study, significantly de-risk costly delays to clinical programs and ultimately increase the chances of approval. With only 8 tickets remaining and limited time, secure your place today: https://ter.li/bgu6et

Gene&Cell Therapy >> EnGene’s $60M PIPE; AbbVie to develop neuropsychiatric drugs with Gedeon Richter: Plus, news about Tectonic Therapeutic, Molecular Partners and Arcus Biosciences: EnGene snags $60M: The Boston and Montreal gene therapy biotech raised the private placement from new and current investors a month after giving an early look at bladder cancer data. It’s the second big private placement for the biotech since it went public via a SPAC last fall. — Kyle LaHucik AbbVie partners again with Gedeon Richter: The companies have worked together for nearly two decades, developing CNS medicines like the antipsychotic Vraylar, and now plan to collaborate on more neuropsychiatric drugs. The Budapest-based pharma company gets $25 million upfront in the new pact. — Kyle LaHucik Tim Springer buys more shares of Tectonic Therapeutic: A repeat biotech builder and co-founder of Tectonic, Springer bought $10 million worth of shares from Atlas Ventures this week, according to an SEC filing. He owns about 31% of the company, which went public via a reverse merger this summer. — Kyle LaHucik Molecular Partners prices $20M offering: The Swiss biotech said the raise from HBM Healthcare Investments and other investors will close next week, and the process will support its radiopharma pipeline. — Kyle LaHucik Arcus Biosciences touts data from HIF-2a inhibitor in kidney cancer: The company shared Phase 1/1b data for casdatifan showing 81% of patients experienced disease control with either a partial response or stable disease. Other data demonstrated that 34% of patients experienced an objective response. It’s planning to start a Phase 3 trial of casdatifan in the first half of next year. The study is also enrolling another arm of patients to try out a combination of Arcus’ drug and Exelixis’ Cabometyx. — Katherine Lewin #lucidquest #genetherapy #celltherapy

#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

Want to hear more about negotiating cell and gene therapy deals? We’ve got a great panel line up on 5 June so do sign up to hear more about their experiences when CGT licensing and collaboration agreements, investment rounds and more. I’m really looking forward to chairing this discussion! 😀🧬🏥 #bristowsllp, #onenucleus, #cellandgene, #intellectualproperty, #lifesciences

Check out this webinar on June 11th to learn about analysis of AAV Genome Integrity using a quadplex dPCR assay!

The next session of MoFo’s and Bayer Co.Lab’s “Biologics in Focus” series on November 12 will focus on the specific regulatory and collaboration considerations for cell and gene therapies. This session will provide an overview of the regulatory framework for biological products and essential considerations for collaborations involving cell and gene therapies, including resulting IP, supply chain management, and common financial frameworks. Register to join MoFo’s Brigid Bondoc and Matt Ferry: https://bit.ly/3BWuize

This is the right path for rare disease treatments. Fix the breaks before the crash! so powerful that stakeholders got together yesterday to talk about this, figure it out. #GoingBeyondEveryDay