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FDA oversight and regulatory process: CRL release pause and back on track
The FDA moved to address litigation risk while reworking its handling of pharma rejection letters, with agency postings indicating a pause followed by a partial restart. Separately, the FDA also...
Late-stage oncology win in China: B7-H3 ADC posts Phase 3 overall survival results
Hansoh Pharmaceutical’s B7-H3 antibody-drug conjugate risvutatug rezetecan (ris-rez), partnered with GSK, cleared its Phase 3 primary endpoint in China by improving overall survival in advanced or...
Cardiovascular gene silencing setback: AstraZeneca/Ionis ATTR-CM failure
AstraZeneca’s and Ionis’ gene-silencing therapy eplontersen (Wainua) failed in a Phase 3 trial in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The 1,432-patient Cardio-TTRransform...
Renal inflammation immunotherapy: Vera’s accelerated approval for IgA nephropathy
Vera Therapeutics received U.S. FDA accelerated approval for TRUTAKNA (atacicept-vymj) to reduce proteinuria in adults with primary IgA nephropathy at risk for disease progression. The ORIGIN 3...
CRISPR and next-gen base editing delivery: VLP cytosine base editing with glycosylase inhibition
Researchers reported an approach to improve in vivo cytosine base editing efficiency by combining virus-like particle (VLP) delivery with uracil DNA glycosylase inhibition. The strategy targets a...
Biotech product performance in colorectal screening: Freenome SimpleScreen CRC validation results
Freenome announced validation data for an updated version of its SimpleScreen CRC blood test in the PREEMPT CRC registrational study. The assay met its primary and secondary endpoints, with...
CAR-T platform progress: Fate’s FT-839 gets FDA IND clearance for autoimmune disease
Fate Therapeutics secured FDA IND clearance for FT-839, its next-generation off-the-shelf CAR T-cell candidate targeting CD19 and CD38 for autoimmune diseases. The company positioned FT-839 as...
Biopharma M&A and asset reshaping: Chemolab and Scipher to merge as Scipher Medicine
Chemolab Therapeutics and Scipher Medicine announced a planned merger and rebrand into Scipher Medicine Corporation, with Scipher CEO Reginald Seeto becoming CEO of the combined entity. The...
Clinical diagnostics expansion: Myriad broadens Precise MRD test to more cancer types
Myriad Genetics is expanding use of its ultrasensitive tumor-informed Precise MRD ctDNA test beyond an early access program. The company said it is rolling the assay out across colorectal and...
Genomics and oncology execution: En Carta raises funding to pursue FDA and EU clearances for point-of-care Lyme test
En Carta Diagnostics raised €5 million in private financing to advance regulatory clearances for its molecular point-of-care Lyme disease test and other pipeline products in Europe and the U.S....
GSK and Hansoh score a Phase 3 survival win for B7-H3 ADC in China
GSK reported that its Hansoh-partnered antibody-drug conjugate risvutatug rezetecan (ris-rez) met the primary endpoint in a Phase 3 study in China in advanced or relapsed small-cell lung cancer....
Roche walks away from Huntington’s antisense programs
Roche ended development on two Huntington’s disease gene-silencing programs after disappointing efficacy and a separate safety signal. The company said it is discontinuing the Phase 2 trial of...
AstraZeneca and Ionis take a Phase 3 cardiomyopathy setback
AstraZeneca said its partner Ionis’ antisense therapy Wainua (eplontersen) failed a pivotal Phase 3 trial in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The drug did not beat placebo...
ARPA-H puts $160M behind bespoke gene editing for rare pediatric diseases
ARPA-H announced THRIVE, a program committing up to $160 million over five years to develop custom gene editing therapies for rare genetic pediatric diseases. The agency described THRIVE as...
FDA-approved gene therapy Otarmeni to be provided free by Regeneron
Regeneron said it will provide its FDA-accelerated gene therapy Otarmeni (lunsotogene parvec) for genetic hearing loss free of charge to eligible U.S. patients. The company said the decision was...
CARB-X and CGD design a 10-year $60M market-shaping fund for neonatal sepsis diagnostics
CARB-X is backing a market-shaping effort to accelerate the development of point-of-care diagnostics for neonatal sepsis. A Center for Global Development (CGD) program called NeoTest will use a...
Vera Therapeutics wins FDA accelerated approval for TRUTAKNA in primary IgA nephropathy
Vera Therapeutics said the FDA granted accelerated approval to TRUTAKNA (atacicept-vymj) for adult patients with primary IgA nephropathy (IgAN) who are at risk for disease progression. The company...
Atsena selects ATSN-401 gene therapy candidate for Stargardt IND-enabling studies
Atsena Therapeutics selected ATSN-401 as its lead clinical candidate for Stargardt disease, and the program is now moving through IND-enabling studies. The gene therapy candidate is intended to...
Fate Therapeutics clears FDA IND for FT-839 off-the-shelf CAR T in autoimmune disease
Fate Therapeutics obtained FDA IND clearance for FT-839, a next-generation off-the-shelf CAR T-cell therapy targeting both CD19 and CD38 for autoimmune diseases. The clearance advances Fate’s...
Stem cell pipeline expands: STEM-PD posts first human feasibility and safety results in Nature Medicine
A Phase 1/2 open-label study in Parkinson’s disease reported first human outcomes for STEM-PD, transplanting cryopreserved, off-the-shelf dopamine progenitor cells derived from human pluripotent...