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FDA issues complete response letter to Regenxbio gene therapy — regulator flags data gaps
Regenxbio received a complete response letter for RGX‑121, its AAV‑based gene therapy for mucopolysaccharidosis II (Hunter syndrome), after the agency raised concerns about the surrogate endpoint...
Capsida confirms cerebral edema in fatal trial case — investigations continue
Capsida Biotherapeutics reported an autopsy‑confirmed cause of death as cerebral edema for the patient who died after infusion in its suspended SYNRGY trial of CAP‑002, an AAV gene therapy for...
Madrigal expands MASH arsenal: $60M upfront, up to $4.4B in siRNA deal with Ribo
Madrigal Pharmaceuticals struck a major licensing and acquisition push to bolster its MASH pipeline, agreeing to license six preclinical siRNA programs from China’s Ribo Life Science and paying...
Medicare covers Personalis’ NeXT Personal MRD test for lung‑cancer surveillance
The Centers for Medicare & Medicaid Services granted coverage for Personalis’ NeXT Personal molecular residual disease assay for surveillance of stage I–III non‑small cell lung cancer. CMS based...
Thermo Fisher, Datavant partner to link clinical research with large real‑world data sets
Thermo Fisher Scientific’s PPD clinical research business announced a strategic partnership with Datavant to enable privacy‑preserving tokenization and linkage of de‑identified patient data across...
Lilly buys Orna in multimodal deal — in vivo CAR‑T and circular RNA technologies in focus
Eli Lilly agreed to acquire Orna Therapeutics to add circular RNA (oRNA) and LNP delivery capabilities aimed at enabling in vivo CAR‑T therapies, in a transaction with up to $2.4 billion in...
FDA refuses to review Moderna mRNA flu vaccine — CBER flags trial design
The FDA declined to start a review of Moderna’s experimental mRNA influenza vaccine, citing deficiencies in the trial’s control arm and overall study design in a letter signed by Vinay Prasad,...
FDA issues complete response letter to Regenxbio — Hunter syndrome approval paused
The FDA issued a complete response letter to Regenxbio for RGX‑121, a proposed gene therapy for mucopolysaccharidosis type II (Hunter syndrome), saying the submission did not adequately support...
Capsida confirms cerebral edema in AAV trial fatality — probe continues
Capsida Biotherapeutics reported that an autopsy confirmed cerebral edema as the cause of death in a patient treated in its SYNRGY AAV gene‑therapy trial for STXBP1, but the company said the...
ILiAD raises $115M Series B to launch pivotal pertussis program
ILiAD Biotechnologies closed an oversubscribed $115 million Series B led by RA Capital Management, with participation from Janus Henderson and BNP Paribas Asset Management Alts, to advance its...
Madrigal snaps up six China‑sourced siRNAs: $60M now, $4.4B possible
Madrigal Pharmaceuticals struck a series of deals with Suzhou Ribo Life Science and Ribocure Pharmaceuticals to license six preclinical siRNA programs aimed at metabolic dysfunction‑associated...
Atopic dermatitis shakeup: Evommune posts Dupixent‑competitive Phase II... Nektar shows year‑long durability
Two mid‑stage readouts shifted expectations in atopic dermatitis. Evommune reported EVO301 achieved a statistically significant placebo‑adjusted improvement in EASI scores at Week 12 that the...
Lilly acquires Orna for up to $2.4B — accelerates in vivo CAR‑T strategy
Eli Lilly agreed to acquire Orna Therapeutics in a deal worth up to $2.4 billion to add Orna’s circular RNA and LNP delivery platform and a clinic‑ready in vivo CAR‑T candidate targeting CD19....
Medicare covers Personalis NeXT Personal MRD test for lung cancer surveillance
The Centers for Medicare & Medicaid Services granted Medicare coverage for Personalis’s NeXT Personal molecular residual disease (MRD) assay for surveillance of stages I–III non‑small cell lung...
Precision Biosciences cleared to start PBGENE‑DMD trial — IND accepted
Precision Biosciences received FDA clearance of an Investigational New Drug application for PBGENE‑DMD, enabling site activation for a Phase I/II trial (FUNCTION‑DMD) in ambulatory patients with...
OncoDNA rolls out fragmentomics liquid biopsy OncoXplore for therapy guidance
OncoDNA will begin early‑access roll‑out of OncoXplore, a fragmentomics‑based cell‑free DNA sequencing assay, to research and early access customers while pursuing CE‑IVDR certification for...
FDA refuses review of Moderna's mRNA flu shot: regulator flags trial design
The FDA has refused to start a review of Moderna’s application for its standalone mRNA influenza vaccine, citing flaws in the pivotal trial’s control-arm design rather than explicit safety or...
FDA issues complete response to Regenxbio’s Hunter syndrome gene therapy — approval paused
The FDA issued a complete response letter for Regenxbio’s RGX-121 gene therapy for Hunter syndrome, concluding that the submission did not adequately demonstrate clinical benefit using the trial’s...
Lilly buys Orna... in vivo CAR‑T bet expands pharma playbook
Eli Lilly agreed to acquire Orna Therapeutics in a deal worth up to $2.4 billion to obtain Orna’s circular RNA platform, LNP delivery expertise, and an in vivo CAR‑T therapy poised for Phase I...
Madrigal pays $60M upfront — $4.4B deal for siRNA programs to bolster MASH strategy
Madrigal Pharmaceuticals agreed to acquire six preclinical siRNA programs from Ribo Life Science in a deal with $60 million upfront and as much as $4.4 billion in contingent payments. The programs...