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Huntington’s drug failures end clinical development
Roche moved to shut down its Huntington’s disease development programs, ending clinical work on tominersen after a Phase 2 failure and stopping a separate early-stage asset following preclinical...
Cardiovascular gene-silencing setback reshapes ATTR-CM strategy
AstraZeneca’s partnered gene-silencing therapy Wainua (eplontersen) missed its primary goal in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), adding pressure to reshape the company’s...
UK pricing pressure increases for Amgen’s lung cancer therapy
The UK’s drug pricing watchdog recommended restricting access to Amgen’s Lumakras (sotorasib) for new patients, calling for reimbursement to be cut off. The National Institute for Health and Care...
Stem-cell Parkinson’s trial reports first-in-human safety and feasibility
Lund University and Skåne University Hospital published first human results showing that transplanting cryopreserved stem-cell-derived dopamine progenitors into people with Parkinson’s disease is...
AI biomedical co-scientist aims to automate research workflows
Stanford researchers unveiled Biomni, an AI “co-scientist” designed to autonomously run biomedical research workflows rather than only answer questions. The system is built to interpret natural...
Gene-editing push: ARPA-H funds THRIVE custom therapies
ARPA-H announced it will spend up to $160 million to accelerate custom gene editing treatments for rare diseases under the THRIVE program. The agency will back seven different teams, with a goal...
FDA accelerated approval lands first-in-class BAFF/APRIL dual inhibitor in IgA nephropathy
Vera Therapeutics received FDA accelerated approval for TRUTAKNA (atacicept-vymj) to treat adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. The decision...
Commercial diagnostics: Myriad expands MRD assay beyond breast
Myriad Genetics expanded the availability of its ultrasensitive Precise MRD test beyond a breast-cancer early access program. The company is broadening indications to colorectal and renal cancers...
Precision microbiome therapy for Crohn’s moves with selective AIEC phage strategy
McMaster researchers reported preclinical evidence for a phage therapy that targets Crohn’s disease–associated inflammatory E. coli strains while aiming to preserve broader gut microbiome balance....
Cell and gene therapy lab infrastructure scales: OrganaBio PBMC processing to San Diego
OrganaBio expanded cell and gene therapy sample processing capacity by launching time-sensitive PBMC isolation and cryopreservation in San Diego at Excellos Labs. The local workflow is designed to...
Cardiovascular setbacks for antisense Huntington’s and transthyretin amyloidosis
Roche and Ionis announced the end of two Huntington’s disease programs, including the Ionis-partnered antisense therapy tominersen, after trials missed efficacy goals. Roche said the Phase 2...
FDA policy shift on CRLs and legal risk for rejection letter disclosures
The FDA paused a controversial practice of publicly releasing complete response letters (CRLs), citing litigation risk as it works toward a formal policy for future disclosures. The change follows...
Stem cell and organ replacement progress across Parkinson’s and diabetes modeling
A new cell therapy data package strengthened the feasibility case for Parkinson’s replacement strategies as a stem cell-derived dopamine progenitor program advanced to first-in-human clinical...
Precision oncology diagnostics and MRD expansion across cancers
Myriad Genetics expanded the commercial footprint of its ultrasensitive minimal residual disease assay, Precise MRD, moving beyond an early-access breast cancer program. The company broadened...
Gene editing funding push for custom therapies via ARPA-H THRIVE
ARPA-H launched THRIVE, an up to $160 million effort aimed at developing bespoke gene editing treatments for rare diseases. The program will fund seven different teams, with a requirement that...
New Parkinson’s and neuroscience therapy platforms in early human testing and mechanistic targets
A new off-the-shelf stem cell product reached early clinical checkpoints for Parkinson’s in the STEM-PD program, reinforcing the feasibility of transplanting dopamine progenitors derived from...
Microbiome-targeted phage therapy shows Crohn’s synergy and precision inflammation control
McMaster University researchers reported a precision bacteriophage therapy that neutralizes adherent-invasive E. coli (AIEC) linked to Crohn’s disease inflammation in a mouse model. The team used...
Biopharma deals and platform build-out: AstraZeneca-Sino COPD and Novartis Myricx ADC payload
AstraZeneca expanded its respiratory pipeline through a licensing deal with Sino Biopharmaceutical’s unit Chia Tai Tianqing Pharmaceutical Group for an ex-China PDE3/4 inhibitor, TQC3721. The...
Regenerative medicine momentum: off-the-shelf diabetes islet biology and cell therapy enabling technologies
Diabetes research advanced through new mechanistic insights into how human stem cells commit to pancreatic alpha versus beta cell fates—an important step toward generating functional islets for...
Regulatory and clinical proof for kidney disease: Vera Therapeutics’ TRUTAKNA speeds approval
Vera Therapeutics won accelerated FDA approval for TRUTAKNA (atacicept-vymj) to reduce proteinuria in adults with primary IgA nephropathy at risk for disease progression. The approval is based on...