Mines D`or Orbec Inc

2024 was a challenging year for the biopharma industry, with companies like Bayer, Bristol Myers Squibb, and Johnson & Johnson cutting hundreds to thousands of jobs. Cassava Sciences announced layoffs of 10 employees, 33% of its workforce, following a failed Phase III trial for its Alzheimer's drug. CytomX Therapeutics cut 40% of its staff to focus on clinical programs. Velia, a San Diego biotech, is shutting down, affecting 47 employees. Regeneron's acquisition of Oxular led to layoffs, with no Oxular employees joining Regeneron. Javara, Ring Therapeutics, Outlook Therapeutics, Editas, Bavarian Nordic, BenevolentAI, Chroma Medicine and Nvelop Therapeutics, Cellectar Biosciences, Carisma Therapeutics, Belharra Therapeutics, National Resilience, AmplifyBio, Agenus, Alligator Bioscience, Idorsia Pharmaceuticals, Kronos Bio, Novartis, Recursion Therapeutics, Medigene, Alector, Bristol Myers Squibb, Sonata Therapeutics, 23andMe, Johnson & Johnson, Merck, Gilead Sciences, Adaptimmune, Sensei Biotherapeutics, Marinus Pharmaceuticals, Orna Therapeutics, Thermo Fisher Scientific, Charles River Laboratories, Aurinia Pharmaceuticals, Viracta Therapeutics, Astellas Gene Therapies, Sana Biotechnology, Sage Therapeutics, Compass Pathways, Spero Therapeutics, ICON, Pfizer, Takeda, SalioGen Therapeutics, Evonik, Medtronic, CareFusion Resources, Turnstone Biologics, Leo Pharma, Astellas Pharma, Prime Medicine, Kaléo, Stryker, Relay Therapeutics, ImmunityBio, Shattuck Labs, Inventprise, bluebird bio, Athira Pharma, AGC Biologics, Oncternal Therapeutics, Biosense Webster, Vesigen Therapeutics, Connect Biopharma, BioMarin, IN8bio, Edwards Lifesciences, DermTech, Repare Therapeutics, Genentech, Tome Biosciences, Aadi Bioscience, Lykos Therapeutics, Evotec, Galera Therapeutics, Grail, Ovid Therapeutics, Lexicon Pharmaceuticals, Acelyrin, Boundless Bio, FibroGen, Ajinomoto Bio-Pharma Services, AN2 Therapeutics, Entero Therapeutics, Precigen, Sumitomo Pharma America, uniQure, Vir Biotechnology, Arbutus Biopharma, HilleVax, and Bayer also announced significant layoffs, reflecting a tough year for the industry.

Vertex Pharmaceuticals and Orna Therapeutics collaborate to develop gene therapies for sickle cell disease and transfusion-dependent beta-thalassemia, using Orna's lipid nanoparticle technology. Vertex pays Orna $65m upfront, with potential for up to $635m in milestone payments and royalties. Vertex, already a leader in gene therapy, aims to improve its CRISPR-based therapy Casgevy and transition to in vivo therapies, leveraging Orna's technology for better delivery to stem cells.

The global Sickle Cell Disease (SCD) treatment market is expected to grow to US$ 17.7 billion by 2034, driven by genetic therapy advancements and increased prevalence in regions like sub-Saharan Africa and Asia. Key players like Bluebird Bio and Novartis are innovating treatments, with a focus on gene-editing and therapies targeting the disease's root causes. Regulatory support and rising public awareness are also fueling market growth.

The global Sickle Cell Disease (SCD) treatment market is projected to grow significantly, reaching US$ 17.7 billion by 2034, driven by genetic-based therapies and increased prevalence in regions like sub-Saharan Africa and Asia. Breakthroughs in gene-editing and medical research are fueling demand for innovative treatments, with key players like Bluebird Bio and Novartis leading advancements. Regulatory support and rising public awareness are also contributing to market expansion.

Johnson & Johnson's CAR-T therapy, cilta-cel, gains a six-month FDA review, potentially narrowing Bristol-Myers Squibb and bluebird bio's Abecma lead. Both target BCMA for multiple myeloma, with cilta-cel showing strong clinical results. Competition and data on earlier-line therapy use are ongoing, with Abecma ahead in Europe.

December 2024 saw significant updates in cell and gene therapies: Cilta-cel showed higher MRD negativity in multiple myeloma; Descartes-08 produced durable responses in myasthenia gravis; Bendamustine was effective with fewer AEs in LBCL; Beti-cel demonstrated long-term curative potential in β-thalassemia; AURN001 showed efficacy and safety in corneal edema treatment.

The CAR-T cell therapy market is rapidly growing, with 11 products commercialized globally, including 7 FDA-approved in the U.S. CAR-T therapy modifies patient T-cells to target and kill cancer cells, showing promise in treating blood cancers. Challenges include treating solid tumors, reducing 'vein-to-vein' time, and reimbursement issues. The industry is advancing through gene-transfer techniques, partnerships, and exploring allogeneic therapies.

The CAR-T cell therapy market is rapidly growing, with 11 products commercialized globally, including 7 FDA-approved therapies. CAR-T therapy modifies T-cells to target and kill cancer cells, showing high effectiveness in blood cancers. Challenges include treating solid tumors, reducing 'vein-to-vein' time, and improving reimbursement. The industry is advancing through gene-transfer techniques and partnerships to enhance CAR-T cell modification and efficacy.

In 2024, significant progress in gene and cell therapies for hematological disorders was noted, including Canada's approval of Casgevy for SCD and TDT, and Beqvez for hemophilia B. Despite FDA approvals, uptake of SCD gene therapies remained slow. Allo-HSCT showed positive outcomes for TDT, and a SCD patient died from busulfan-conditioning complications in a BEAM-101 trial.