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DMD cell therapy — Phase 3 results and FDA advisory risk
Capricor Therapeutics’ deramiocel delivered a measurable slowing of muscle decline in advanced Duchenne muscular dystrophy in the Phase 3 HOPE-3 trial, with results published in The Lancet. In the...
Schizophrenia trial — MapLight’s mid-stage signal reshapes investor expectations
MapLight’s schizophrenia program delivered a mixed Phase 2 readout tied to dosing frequency, sending the stock sharply lower after investors expected a clearer path to differentiation. The...
Transatlantic acquisitions — Caldera and Vidya pursue reverse-merger paths to Nasdaq
Caldera Therapeutics agreed to merge with Synlogic in a deal aimed at accelerating advancement of its China-licensed anti-inflammatory asset CLD-423, while also raising $278 million in a...
Immunology partnership — AbCellera teams with Vertex for autoimmune T-cell engagers
AbCellera expanded its T-cell engager footprint by entering a collaboration with Vertex Pharmaceuticals focused on discovering, developing, and commercializing multispecific T-cell engagers for...
Ophthalmology — Claris Biotherapeutics funds limbal stem cell deficiency pivots
Claris Biotherapeutics closed a $118 million Series B to advance CSB-001 (oremepermin alfa ophthalmic solution) toward pivotal trials in limbal stem cell deficiency. The company’s proof-of-concept...
TB diagnostics — IntelliGenome expands distribution for CRISPR blood test
IntelliGenome expanded global commercialization for its CRISPR-TB blood assay with new distribution appointments in Taiwan and South Africa. The company’s test is designed to detect Mycobacterium...
Immunology dealmaking — AbCellera-Vertex and Argenx’s Forte acquisition both show portfolio-in-a-product momentum
Argenx struck a $2.2 billion acquisition of Forte Biosciences to fold the Phase Ib-ready anti-CD122 antibody FB102 into its immunology portfolio. Argenx said the decision is driven by clinical...
Alzheimer’s safety signal — ProMIS interim ARIA readout improves tolerability profile
ProMIS Neurosciences reported interim safety results from an amyloid-targeting Alzheimer’s trial suggesting a low rate of brain bleeding and no brain swelling, with results communicated ahead of...
Regulatory scrutiny — Replimune faces renewed FDA advisory concerns on RP1 data
FDA reviewers reiterated concerns that Replimune’s melanoma therapy RP1 does not have adequate data to support approval in briefing documents ahead of a third FDA advisory committee push....
Gene editing — In vivo CRISPR base editing reverses Huntington’s disease phenotypes in mice
University of Illinois Urbana-Champaign researchers reported in vivo CRISPR base editing that reduced toxic protein fragments and neurologic symptoms in a mouse model of Huntington’s disease. The...
DMD cell therapy: HOPE-3 phase 3 signals on function and cardiac preservation
Capricor Therapeutics’ donor-cell bloodstream therapy deramiocel produced statistically significant slowing of upper-limb functional decline in boys and young men with advanced Duchenne muscular...
Immunotherapy delivery in bladder cancer: trial supports potential to avoid cystectomy
An international phase 3 study in The Lancet Oncology reported that cretostimogene grenadenorepvec, an intravesical oncolytic immunotherapy, may enable some patients with high-risk,...
Autoimmune T-cell engagers: Vertex doubles down on AbCellera’s TCE platform
AbCellera and Vertex Pharmaceuticals expanded their T-cell engager strategy with a new collaboration to discover, develop, manufacture, and commercialize multispecific T-cell engagers for...
Regulatory scrutiny in melanoma gene therapy: Replimune faces continued FDA skepticism
Regulatory and investor attention stayed focused on Replimune’s RP-1 as FDA reviewers reiterated concerns about the company’s statistical approach and the adequacy of evidence in a single-arm...
Precision microbiology and diagnostics: 10x Genomics and Lausanne Hospital target tumor biomarkers
10x Genomics announced a multi-year collaboration with Lausanne University Hospital to use single-cell and spatial technologies to discover tumor biomarkers tied to treatment response and...
Biotech takeout: Argenx agrees $2.2B acquisition of Forte for FB102 autoimmunity expansion
Argenx agreed to acquire Forte Biosciences for about $2.2 billion, adding FB102, an anti-CD122 monoclonal antibody, as a potential pipeline-in-a-product. The proposed deal follows Forte’s reported...
Oncology ADC signal: GSK/Hansoh B7-H3 therapy posts another phase 3 win in China
Hansoh’s B7-H3-directed antibody-drug conjugate risvutatug rezetecan (ris-rez), partnered with GSK, logged another phase 3 progression-free survival win in China for relapsed osteosarcoma. The...
Alzheimer’s pipeline safety signal: interim ProMIS study suggests lower ARIA rates
An interim, blinded safety analysis from ProMIS Neurosciences’ amyloid-targeting Alzheimer’s candidate was reported with low brain bleeding and limited swelling signals. The company said the...
New funding for ophthalmology: Claris $118M Series B to move eye drop into pivotal LSCD testing
Claris Biotherapeutics secured a $118 million Series B to advance CSB-001 (oremepermin alfa ophthalmic solution) toward pivotal testing for limbal stem cell deficiency. The late-stage company said...
Gene therapy and PO C manufacturing: Cellares partners with Papillon to scale CRISPR cell therapy for Friedreich’s ataxia
Cellares and Papillon Therapeutics partnered to scale manufacturing for Papillon’s CRISPR-based hematopoietic stem and progenitor cell therapy PPL-001 for Friedreich’s ataxia. The arrangement...