Get the Daily Brief
Latest Biotech News
Cell therapy Phase 3 readout in Duchenne muscular dystrophy
A Phase 3 trial in The Lancet reports that deramiocel, a heart-derived allogeneic cell therapy, slowed functional decline in boys and young men with advanced Duchenne muscular dystrophy (DMD). The...
FDA adcomm fallout over deramiocel efficacy evidence
An FDA advisory committee vote rejected Capricor’s evidence for deramiocel in Duchenne muscular dystrophy after a meeting dominated by dispute over statistical analysis and how the Phase 3...
Alzheimer’s: interim safety signal in amyloid-directed trial
ProMIS Neurosciences reported an interim safety analysis in its amyloid-targeting Alzheimer’s trial, describing low rates of brain bleeding and no brain swelling in a blinded safety assessment...
CAR-T platform licensing push: J&J options Sail in vivo CAR-T
Johnson & Johnson lined up a $785 million upfront option deal to work with Sail Biomedicines, part of Flagship Pioneering, on in vivo CAR-T approaches. The transaction structure calls for J&J to...
AI drug discovery partnership between Elix and University of Vienna
Elix and the University of Vienna entered a joint research agreement to accelerate drug discovery using AI. The collaboration centers on Elix’s integrated AI discovery platform and the Orts Lab’s...
Gene therapy targeting Huntington’s: in vivo base editing reverses disease in mice
Researchers at the University of Illinois Urbana-Champaign reported that in vivo CRISPR base editing can reduce toxic protein burden and neurologic symptoms in a mouse model of Huntington’s...
Regulatory economics: FTC sues Hims over privacy and deceptive billing
The U.S. Federal Trade Commission, joined by Utah and California officials, sued Hims & Hers alleging the telehealth provider shared sensitive health information with third-party advertising...
Biotech M&A and financing: Caldera reverse merger plus PIPE funding
Caldera Therapeutics agreed to a reverse merger with Synlogic and raised a $278 million private financing to support a mid-stage inflammatory bowel disease asset. The funding is intended to back...
Diagnostics and infectious disease: CRISPR-TB distribution expands to Taiwan and South Africa
IntelliGenome expanded commercial reach for its CRISPR-TB molecular tuberculosis test by naming distributors in Taiwan and South Africa. The assay detects Mycobacterium tuberculosis complex...
Deal-making in immunology: AbCellera and Vertex for multispecific T-cell engagers
AbCellera and Vertex Pharmaceuticals agreed to collaborate on discovery, early development, manufacturing and commercialization of multispecific T-cell engagers for autoimmune diseases and other...
FDA adcom takes aim at deramiocel’s statistical support
The FDA advisory committee meeting on Capricor Therapeutics’ Duchenne muscular dystrophy therapy deramiocel ended with experts voting against the company, underscoring a high bar for statistical...
Phase 3 win for deramiocel in The Lancet—offsets regulatory fight
A phase 3 clinical trial published in The Lancet reports that deramiocel slowed muscle decline in boys and young men with advanced Duchenne muscular dystrophy, with signals also reported for...
Gene therapy—base editing strategy shows Huntington reversal in mice
Researchers from the University of Illinois Urbana-Champaign report that in vivo CRISPR base editing reversed Huntington’s disease–associated toxic protein burden and neurological symptoms in...
Regulatory exposure—FDA and advisory scrutiny surround Replimune’s RP1
A running FDA review of Replimune Group’s melanoma therapy RP1 continued to churn as regulators reiterated concerns about the evidence base. FDA briefing materials challenged whether the...
Cell therapy mechanism readout—CD19 transfers between immune cells
A Nature Communications study reports that the B-cell marker CD19 can be transferred between immune cells in both mice and humans, adding a new mechanism for how surface proteins move across the...
Biotech partnership to expand cancer single-cell biomarker discovery
10x Genomics announced a multi-year collaboration with Lausanne University Hospital to apply single-cell and spatial biology tools to biomarker discovery across advanced solid cancers. The program...
Corporate deal—Argenx acquires Forte Biosciences to add FB102
Argenx struck an acquisition deal for Forte Biosciences valued at about $2.2 billion, aiming to expand its immunology portfolio with Forte’s anti-CD122 antibody FB102. Under the agreement, Argenx...
Funding + pipeline—Claris moves CSB-001 into pivotal trials for LSCD
Claris Biotherapeutics raised $118 million in a Series B financing to advance CSB-001, its eye-drop candidate for limbal stem cell deficiency (LSCD), toward pivotal clinical studies. The company...
Rare disease expansion—Papillon and Cellares scale CRISPR-based FA therapy manufacturing
Cellares partnered with Papillon Therapeutics to scale manufacturing for PPL-001, a CRISPR-based cell therapy candidate for Friedreich’s ataxia. The collaboration will focus on transferring the...
AI-enabled diagnostics—En Carta prepares Lyme at-home molecular test
En Carta Diagnostics is preparing to launch an at-home Lyme disease molecular test using an aptamer sensor coupled with isothermal amplification and a cartridge-based workflow. The company says it...