VantAI and Blueprint Medicines Expand Collaboration to Tackle "Undruggable" Targets with AI-Powered Proximity Therapeutics
• VantAI and Blueprint Medicines have expanded their strategic collaboration for the second time since 2022, incorporating additional drug programs focused on targets previously considered undruggable.
• The expanded agreement makes VantAI eligible for up to $1.67 billion in milestone payments plus royalties, highlighting significant industry validation for their AI-driven approach to induced proximity therapeutics.
• The collaboration will leverage VantAI's recently announced Neo-1 foundation model, the first AI system capable of generating molecular glues and other protein-protein interaction modifiers to address complex therapeutic challenges.
Tourmaline Bio's Heart Disease Antibody Shows Promise in Phase 2, But Shares Fall Amid Competition Concerns
• Tourmaline Bio's antibody drug pacibekitug demonstrated significant reduction in cardiovascular risk biomarkers compared to placebo in Phase 2 trials, but shares fell 18% as investors question its competitive edge against Novo Nordisk's offering.
• Schrödinger announced CFO Geoffrey Porges will leave the company amid near-record low share prices, coinciding with plans to lay off 7% of staff as part of cost-cutting measures.
• CRISPR Therapeutics is expanding its technological capabilities through a $95 million deal with Sirius Therapeutics to develop SRSD107, a novel siRNA blood thinner designed to minimize bleeding risk.
CRISPR Therapeutics Expands into siRNA Therapies with $95M Sirius Partnership for Thromboembolic Disorders
• CRISPR Therapeutics is diversifying beyond gene editing through a strategic $95 million partnership with Sirius Therapeutics to develop SRSD107, a long-acting siRNA therapy targeting Factor XI for thromboembolic disorders.
• In Phase 1 trials, SRSD107 demonstrated impressive efficacy with over 93% reduction in Factor XI levels and doubled blood clotting time, with effects lasting up to six months from a single dose.
• The collaboration includes a 50-50 cost and profit-sharing structure for SRSD107 development, with CRISPR leading U.S. commercialization and retaining rights to license two additional siRNA targets.
FDA Clears Capsida's Revolutionary IV Gene Therapy for STXBP1-DEE, First to Cross Blood-Brain Barrier
• Capsida Biotherapeutics has received FDA IND clearance for CAP-002, the first intravenously administered gene therapy that crosses the blood-brain barrier while avoiding off-target tissues for STXBP1-DEE.
• In non-human primate studies, CAP-002 achieved transduction of over 70% of neurons across critical brain areas, showing potential to correct seizures, developmental disabilities, and motor abnormalities after a single IV infusion.
• The SYNRGY Phase 1/2a clinical trial is expected to begin dosing patients in Q3 2025, offering the first potential disease-modifying treatment for STXBP1-DEE, a rare disorder affecting approximately 5,000 pediatric patients in the US and Europe.
CAR-T Cell Therapy Funding Surges to $141.2 Billion as Industry Expands Globally
• The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with estimates suggesting total industry funding could reach $281.7 billion when including undisclosed deals.
• More than 170 companies worldwide are developing CAR-T products with 1,944 therapies in development, while 13 CAR-T cell therapies have received regulatory approval globally since 2017.
• Despite a slowdown in IPOs and M&A activity in 2024, venture capital funding remains strong with 89 CAR-T companies securing $7.7 billion since 2014, supporting advancement in both blood cancer and solid tumor applications.
NKure and CRISPR Therapeutics Partner to Develop Off-the-Shelf CAR-T Therapy for Cancer in India
• Bengaluru-based NKure Therapeutics and CRISPR Therapeutics have formed a strategic partnership to co-develop CTX112, a next-generation allogeneic CAR-T therapy for B-cell malignancies in India.
• CTX112 utilizes CRISPR/Cas9 gene-editing technology on healthy donor cells to create an off-the-shelf product that could potentially reduce treatment costs to one-third of current autologous CAR-T therapies in India.
• The companies plan to seek CDSCO approval in June to conduct Phase II clinical trials in India, enrolling approximately 25 patients, with a marketable product expected within two years.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Eli Lilly Commits $250 Million to Expand Purdue University Research Alliance Through 2032
• Eli Lilly has extended its research collaboration with Purdue University through 2032, investing up to $250 million over the next eight years in what could become the largest industry-academic partnership in the United States.
• The expanded alliance will facilitate researcher exchanges between Purdue's campus and Lilly's Indianapolis facilities, while also utilizing Indiana's LEAP Research and Innovation District to accelerate drug discovery and development.
• This partnership represents a significant investment in pharmaceutical innovation infrastructure, focusing on improving both drug discovery processes and manufacturing capabilities in the U.S.
Capsida Biotherapeutics Advances Novel Gene Therapies for CNS Disorders Ahead of ASGCT 2025
• Capsida Biotherapeutics will present seven scientific presentations at ASGCT 2025, showcasing progress on their engineered AAV gene therapies targeting neurological disorders.
• The company's lead candidate CAP-002 for STXBP1-DEE demonstrated widespread brain expression in non-human primate studies, with potential to correct seizures, motor abnormalities, and developmental disabilities.
• Capsida is on track to initiate clinical trials for both CAP-002 (STXBP1-DEE) and CAP-003 (Parkinson's disease with GBA mutations) programs in the first half of 2025.
Pfizer Withdraws Sickle Cell Drug Oxbryta Globally Following Safety Concerns
• Pfizer has voluntarily withdrawn its sickle cell disease therapy Oxbryta (voxelotor) from all global markets after discovering an imbalance in deaths during clinical trials.
• The withdrawal marks a significant setback for the sickle cell community as Oxbryta was the first approved medication that directly prevented sickle hemoglobin polymerization, the molecular basis of the disease.
• The decision follows Pfizer's $5.4 billion acquisition of Global Blood Therapeutics in 2022, with Oxbryta having generated $328 million in sales last year and previously projected to reach $2 billion in peak annual revenue.
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
Bayer and CRISPR Therapeutics Form $300 Million Joint Venture to Advance Gene-Editing Therapies
• Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer.
• The collaboration will focus on developing potential curative treatments for hemophilia, congenital heart disease, and Stargardt disease using CRISPR-Cas9 gene-editing technology.
• This partnership represents Bayer's first investment through its newly established Bayer LifeScience Centre and joins other major pharmaceutical companies including Vertex, Novartis, and Celgene in backing CRISPR-Cas9 technology.
Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs
• Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025.
• Krystal Biotech's KB407 for cystic fibrosis received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025.
• CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally.
• CRISPR Therapeutics expects key clinical data updates in 2025 for CTX112 in oncology and autoimmune diseases, and for CTX310 and CTX320 in cardiovascular programs.
Intellia Therapeutics Prioritizes Late-Stage CRISPR Therapies, Announces Workforce Reduction
• Intellia Therapeutics is prioritizing NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis, focusing on late-stage development.
• The company plans to complete enrollment for the Phase 3 HAELO study of NTLA-2002 in HAE in the second half of 2025, with a BLA submission expected in 2026.
• Intellia anticipates enrolling over 550 patients in the MAGNITUDE study for nex-z in ATTR amyloidosis with cardiomyopathy (ATTR-CM) by the end of 2025.
• A strategic reorganization, including a 27% workforce reduction, will extend the company's cash runway into the first half of 2027, supporting commercial readiness.
Vir Biotechnology's Dual-Masked T-Cell Engagers Show Promise in Early Cancer Trials
• Vir Biotechnology's VIR-5818 demonstrated tumor shrinkage in 50% of HER2-expressing cancer patients at doses ≥400 µg/kg.
• VIR-5500 showed PSA reductions in 100% of mCRPC patients at initial doses ≥120 µg/kg, with a 58% confirmed PSA50 response.
• Both VIR-5818 and VIR-5500 exhibited promising safety profiles with minimal cytokine release syndrome and manageable adverse events.
• The PRO-XTEN™ masking technology may enable tumor-specific activation and a wider therapeutic index for T-cell engagers.
Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments
• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.
• Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways.
• These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets.
• The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.
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