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FDA clears first-in-class gene therapy for ultra-rare glycogen storage disease
The FDA granted accelerated approval to Ultragenyx’s one-time gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa), marking the company’s first...
European regulators pull marketing authorization for Amgen’s Tavneos
National drug regulators in Europe have ordered a recall of all batches of Amgen’s Tavneos (avacopan) after the European Commission formally canceled the drug’s marketing authorization earlier...
China’s NMPA accepts NDA for PD-L1/4-1BB bispecific opamtistomig
China’s National Medical Products Administration accepted the new drug application for opamtistomig (LBL-024), a PD-L1/4-1BB bispecific antibody, as monotherapy for previously treated advanced...
DeepCyte launches DeeTox Atlas for single-cell toxicity mechanism modeling
DeepCyte launched DeeTox Atlas, a single-cell metabolomic reference atlas designed to map drug toxicity mechanisms at cellular resolution. The company said it plans enterprise pilot programs with...
Silence Therapeutics reports positive Phase II results for divesiran in polycythemia vera
Silence Therapeutics reported positive Phase II trial results for divesiran, an siRNA therapy targeting TMPRSS6 in polycythemia vera (PV). In the SANRECO study (NCT05499013), the company said...
Ongoing clinical trial updates: Ambros reverse merger to advance phase 3 pain therapy
Ambros Therapeutics agreed to merge with Werewolf in an all-stock transaction and raised $150 million, positioning the combined company to advance a Phase 3 rare disease pain program toward an...
Amid questions, NGS testing adoption remains uneven even after Medicare coverage
A new analysis using seven years of Medicare claims data found that adoption of next-generation sequencing (NGS) in cancer remained slower than expected after CMS began covering testing nationally...
FDA clears first standalone robotic blood draw device in the US
The FDA approved Vitestro’s Aletta, described as the first standalone robotic device in the U.S. designed to draw blood with limited hands-on assistance. The device performs key steps—tourniquet...
Silicon and AI meet cell line development: Deepcell and Gilead co-develop morphology foundation model
Deepcell and Gilead Sciences entered a partnership to codevelop a custom AI foundation model for Chinese hamster ovary (CHO) cell line development using cell morphology data. Under the...
CAR T engineering approach: localized secretion of EpCAM bispecific engagers
Researchers proposed a CAR T strategy designed to overcome tumor heterogeneity and reduce systemic toxicity by localizing EpCAM-targeting bispecific T cell engagers at the tumor site. The approach...
Regulatory approval – rare genetic disease gene therapy
FDA has cleared Ultragenyx’s one-time AAV8 gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa) in patients aged 8 and older. The approval...
Oncology – FDA approval for bone disease drug
The FDA approved Regeneron’s Pasatru (garetosmab) to treat fibrodysplasia ossificans progressiva (FOP), clearing a new option for patients with the rare degenerative condition that causes muscle...
Company/market – chronic pain biopharma advances via reverse merger
Ambros Therapeutics is moving its pain drug into later-stage development via a reverse merger with Werewolf Therapeutics. Alongside the all-stock transaction, the combined company will raise $150...
Clinical trial – home-based breast cancer injections supported by telemedicine
A study in JAMA Network Open found that a telemedicine-supported home injection model can reduce the practical burden for breast cancer patients receiving injectable gonadotropin-releasing hormone...
Immunotherapy discovery – engineered drug “molecular glue” flips cancer drivers
Researchers at Stanford Medicine reported in mice that a two-headed experimental “molecular glue” strategy can eradicate aggressive human lymphoma tumors by converting a cancer growth driver into...
Rare disease pipeline – siRNA Phase II shows strong PV response
Silence Therapeutics reported positive Phase II results for divesiran, an siRNA targeting TMPRSS6 in polycythemia vera (PV). In the SANRECO trial (NCT05499013), the company said the program met...
Cell and gene therapy platform – targeted lipid nanoparticles for in vivo editing
A Nature Biomedical Engineering study described a strategy to deliver gene-editing technology directly to human hematopoietic stem cells in vivo using targeted lipid nanoparticles, avoiding viral...
Diagnostics/AI governance – pathology models need uncertainty signals and human review
A framework called TRUECAM, developed by researchers at Hong Kong Polytechnic University, is designed to show when pathology AI predictions are trustworthy, when they are uncertain, and when...
Oncology – NMPA accepts NDA for 4-1BB bispecific in extrapulmonary NEC
China’s NMPA accepted the new drug application for opamtistomig (LBL-024), a PD-L1/4-1BB–directed bispecific antibody, as monotherapy for previously treated advanced extrapulmonary neuroendocrine...
Biotech innovation – Open-access human cell surface atlas for drug delivery
The Hertz Foundation Entrepreneurship Award is backing an open-access human cell surface map intended to support drug discovery and delivery decisions. The open atlas concept targets a bottleneck...