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Lipid nanoparticles enable base editing for retinitis pigmentosa
Researchers demonstrated lipid nanoparticle (LNP) delivery of base editors to correct a rhodopsin RHO c.1030C>T (p.Q344X) mutation implicated in retinitis pigmentosa, achieving functional rescue...
Novartis bets on AI: $1.7bn pact with Relation
Novartis signed a multi-program discovery and development collaboration with UK AI-driven biotech Relation to target atopic diseases, committing up to $1.7 billion in milestones and tiered...
FDA clears Waskyra: first nonprofit wins gene‑therapy approval
The FDA approved Waskyra, a gene therapy for Wiskott‑Aldrich syndrome, marking the first time the agency has cleared a genetic medicine developed and submitted by a nonprofit sponsor, Fondazione...
FDA raises bar for CAR‑T approvals: randomized trials preferred
FDA officials signaled a policy shift that will generally require randomized controlled trials (RCTs) to support approvals of CAR‑T cell therapies, except in narrowly defined circumstances such as...
ARPA‑H funds AI‑driven toxicity models: Inductive Bio leads $21M effort
ARPA‑H awarded up to $21 million to an industrial‑academic consortium led by Inductive Bio to build next‑generation, human‑biology‑driven AI models for drug toxicity prediction. Partners include...
Scouter: LLM forecasts transcriptional responses to genetic perturbations
Researchers introduced Scouter, a tool that leverages large language models to predict transcriptional changes arising from genetic perturbations. Published in Nature Computational Science, the...
AlphaFold 3 points to OPG153: new mpox vaccine/antibody target
Researchers used AlphaFold 3 to map antibody‑viral protein interactions and identified OPG153 as a previously unrecognized neutralizing surface antigen on monkeypox virus (mpox). The Science...
Biotech market rally: eight companies raise $3.2bn in one session
Public markets showed strong appetite for biotech as eight companies executed follow‑on offerings raising approximately $3.2 billion in a single overnight session, marking one of the largest...
D3 Bio secures $108M to advance KRAS G12C into phase III
D3 Bio closed a $108 million Series B to finance the global phase III development of its oral KRAS G12C inhibitor elisrasib (D3S‑001). The Shanghai‑based company, led by an AstraZeneca veteran,...
Lilly picks Huntsville for $6bn API hub to secure GLP‑1 supply
Eli Lilly announced Huntsville, Alabama, as the site for a $6 billion active pharmaceutical ingredient (API) manufacturing facility that will produce peptide and small‑molecule APIs, including the...
Congressional action targets HHS leadership amid vaccine policy disputes
A House Democrat introduced articles of impeachment against Health and Human Services Secretary Robert F. Kennedy Jr., alleging abuses of authority and actions that undermine public‑health...
FDA OKs First Nonprofit Gene Therapy: Waskyra Clears Rare Immune Disorder
The FDA approved Waskyra, the first gene therapy authorized to treat Wiskott–Aldrich syndrome, and the first gene therapy cleared from a nonprofit sponsor. The product, developed by Fondazione...
Novartis Pays $55M Upfront — Relation to Drive AI‑Led Atopic Targets
Novartis struck a strategic collaboration with Relation to discover and advance drug targets for atopic diseases, committing $55 million in upfront and R&D funding plus equity and potential...
FDA Raises Bar for CAR‑T Approvals: Randomized Trials Favored
Top officials at the FDA’s Center for Biologics Evaluation and Research signaled a policy shift: new CAR‑T approvals should generally be supported by randomized controlled trials with survival or...
Major ASH Reads Shift Myeloma Playbook — Bispecifics and In‑Vivo CAR‑T Impress
Late‑breaking data at ASH showed striking efficacy for bispecific and in‑vivo CAR‑T approaches in multiple myeloma. The MajesTEC‑3 trial presented an 83.4% three‑year progression‑free survival for...
ARPA‑H Funds Human‑First Toxicity Models: $21M to $32M Awards
Two multi‑partner consortia won ARPA‑H awards to build next‑generation drug safety models that reduce reliance on animal testing. Inductive Bio leads a project (DATAMAP) with up to $21 million to...
Investors Flood Biotech: Record Follow‑On Deals and Holiday‑Era Offerings
Public markets reopened aggressively for biotech as a wave of follow‑on offerings raised record amounts in a compressed window. Reports show $3.2 billion in follow‑on stock offerings executed over...
AI‑Designed Molecules Advance: Insilico PROTAC and PsiThera’s Oral TNF Play
AI‑driven discovery produced two notable developments: Insilico Medicine reported an AI‑designed PROTAC degrader targeting PKMYT1 with preclinical efficacy across biomarker‑selected models, and...
Roche’s Brainshuttle Antibody Clears Amyloid: 91% Below Threshold in Early Trial
Roche presented Phase I/II Brainshuttle data showing its anti‑amyloid antibody trontinemab achieved rapid, deep amyloid plaque clearance below the positivity threshold in 91% of participants with...
AI Finds Monkeypox Weak Spot — While Surveillance Flags New Recombinant Strain
Researchers used AlphaFold‑3 to predict a monkeypox viral surface protein, OPG153, as a neutralizing antibody target; murine immunizations with OPG153 produced neutralizing antibodies, opening a...