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AI-enabled drug design partnerships
Alnylam signed a three-year, up-to-$2 billion strategic collaboration with Inceptive Nucleics to apply AI foundation models to RNAi therapeutics discovery. The partnership follows Inceptive...
China genomics and newborn screening with whole-genome sequencing
BGI and Fudan University led a prospective cohort study evaluating whole-genome sequencing–based newborn screening (NBS) in China, reporting that WGS identified single-gene conditions missed by...
FDA diagnostics—breakthrough device designation for invasive mold blood test
Zepto Life Technologies received FDA breakthrough device designation for its FungiFlex Mold Panel, a plasma-based assay designed to detect circulating cell-free DNA from invasive fungal pathogens....
CAR T program design—automation and scale-up for cell therapy manufacturing
Cellares and TScan Therapeutics agreed to evaluate automated clinical manufacturing of TSC-101, TScan’s lead TCR-T cell therapy candidate for AML and MDS patients undergoing allogeneic...
Gene editing licensing — Lilly partners for Ascidian RNA exon editors in inherited kidney disease
Eli Lilly entered a global research and licensing collaboration with Ascidian Therapeutics for RNA exon editing therapeutics targeting monogenic kidney diseases. The deal could reach up to $1.9...
Sickle cell drug setback—Fulcrum drops lead PRC2 program after FDA concerns
Fulcrum Therapeutics ended development of its sickle cell disease candidate pociredir and launched a strategic review after the FDA raised heightened concerns about malignancy risk for PRC2...
Clinical trial therapeutics—automated data expectations and FDA policy direction
The FDA signaled that its real-time clinical trials initiative could deliver tangible benefits beyond speed and cost reduction, including more actionable signals built into trial design. The...
Immune cell therapy—high-level first-in-human in vivo CAR T momentum
Legend Biotech disclosed early findings from an investigator-initiated study of its in vivo CAR-T approach in lymphoma, reporting response activity across the initial cohort. The company said the...
Oncology: pancreatic cancer breakthrough in Phase 3
Revolution Medicines reported Phase 3 RASolute 302 results for daraxonrasib, a pan-RAS inhibitor, showing a median overall survival of 13.2 months versus 6.7 months with chemotherapy in 500...
Cell therapy: in vivo CAR T early efficacy sparks market optimism
Legend Biotech began to validate its in vivo CAR T approach with early data from an investigator-initiated study in lymphoma. Initial results reported by the company showed tumor responses across...
Genetic medicines: Lilly expands RNA exon editing deal for inherited kidney diseases
Eli Lilly and Ascidian Therapeutics signed a global research and licensing collaboration for RNA exon editing therapies targeting inherited monogenic kidney diseases. Lilly will pay up to $1.9...
Diagnostics: FDA breakthrough designation for invasive mold liquid biopsy
FDA granted Zepto Life Technologies breakthrough device designation for the company’s FungiFlex Mold Panel, a cfDNA-based blood test intended to detect invasive mold infections and distinguish...
Drug development with AI: Alnylam and Inceptive Nucleics expand RNAi foundation-model collaboration
Alnylam Pharmaceuticals and Inceptive Nucleics announced a three-year strategic collaboration focused on AI foundation models to support RNAi therapeutic R&D. The deal includes $30 million in cash...
Immunology / translation: microenvironment barriers mapped with spatial pharmacobiology
Researchers from Vanderbilt University Medical Center and Stanford University introduced a single-cell spatial pharmacology (SSP) platform to visualize where antibody drugs distribute and engage...
Clinical policy / regulation: FDA guidance clarifies how sponsors can leverage known gene therapy knowledge
The FDA issued draft guidance in CBER explaining how sponsors can leverage prior knowledge across gene therapy development, rather than restarting evidence generation from scratch for each...
Biotech funding: Ona Therapeutics pushes ADCs into humans with $86.6M Series B
Ona Therapeutics closed an oversubscribed $86.6 million Series B to advance two antibody drug conjugates into human testing. The Barcelona-based biotech plans to move ONA-255, initially targeting...
Cell therapy manufacturing: Cellares and TScan evaluate automated production for TSC-101
Cellares and TScan Therapeutics agreed to evaluate automated manufacturing for TSC-101, TScan’s TCR-T therapy candidate designed for patients with AML and MDS undergoing allogeneic hematopoietic...
Health policy: WHO strengthens clinical trial design and implementation training
The World Health Organization launched an online course aimed at strengthening clinical trial design and implementation, emphasizing ethical principles, trial conduct, oversight, and participant...
RNA exon editing and kidney disease collaboration
Eli Lilly has struck a global research and licensing collaboration with Ascidian Therapeutics focused on RNA exon editing therapeutics for inherited kidney diseases, a deal that could be worth up...
Pan-RAS drug lifts pancreatic cancer survival
Revolution Medicines’ daraxonrasib delivered a substantial survival benefit in a phase 3 trial for advanced pancreatic ductal adenocarcinoma, doubling median overall survival versus chemotherapy....