Gene Therapy
A Areas medical research research area page covering state of the field, key trials running, recent readouts and what is close to approval, alongside the
Merck KGaA acquires Silence Therapeutics for $1.3 billion
Merck KGaA is buying Silence Therapeutics for $1.3 billion to gain control of divesiran, an RNAi therapy showing positive Phase 2 results in...
Estuary Biotherapeutics Launches with $93M for ADPKD Gene Therapies
Estuary Biotherapeutics launched with $93 million in Series A funding to develop one-time gene therapies for autosomal dominant polycystic kidney...
BioNTech's BNT113 Fails Key Goal in Head and Neck Cancer Trial
BioNTech's mRNA cancer therapy BNT113 combined with Keytruda did not improve progression-free survival in a Phase 3 trial for HPV-driven head and...
Shionogi Acquires IntraBio for $2 Billion to Expand Rare Disease Portfolio
Shionogi has agreed to acquire biotech firm IntraBio for $2 billion in cash, gaining the marketed rare disease therapy Aqneursa.
Novartis licenses preclinical radioligand in $900M BoomRay deal
Novartis has licensed a preclinical radioligand therapy from China's BoomRay Therapeutics in a deal worth up to $900 million.
FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A
The FDA has approved rebisufligene etisparvovec (Fayuvi) as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA.
AI startup Superluminal raises $60M for first drug candidate
AI drug discovery firm Superluminal Medicines has raised $60 million to advance its first candidate, an oral therapy targeting the MC4R receptor.
UniQure Seeks FDA Approval for Huntington's Gene Therapy
UniQure has submitted AMT-130, a gene therapy for Huntington's disease, to the FDA for accelerated approval based on three-year data showing it can...
Genentech pays $45M for DualityBio ADC pact
Roche's Genentech will pay DualityBio $45 million upfront for rights to antibody-drug conjugates designed for patients who progress after existing...
T Cell Therapy Treats Atopic Dermatitis
A Lancet comment published August 21, 2026, discusses rezpegaldesleukin, a novel therapy designed to expand regulatory T cells.
Revolution's pancreatic cancer drug Rasonque cleared by FDA
Revolution Medicines' pancreatic cancer therapy Rasonque cleared FDA approval via priority review voucher, marking a major advance for second-line...
Daraxonrasib Approved for Metastatic Pancreatic Cancer
The FDA has granted approval to the RAS inhibitor daraxonrasib for adults with metastatic pancreatic ductal adenocarcinoma who have progressed on...
TALENTOP trial: conversion surgery in advanced HCC
The TALENTOP study, a multicentre, open-label, randomised phase 3 trial, compares liver resection after atezolizumab and bevacizumab with maintenance...
Focal Therapy for Prostate Cancer Divides Physicians
Focal therapy for prostate cancer has divided physicians, with some considering it a viable alternative to traditional treatments and others viewing...
UK feasibility trial finds psilocybin therapy safe for resistant depression
A 2026 feasibility study delivering psilocybin-assisted therapy within the NHS reported high adherence and no safety concerns, raising questions...
FDA Clears Bristol Myers Squibb's Oral Multiple Myeloma Therapy, Introducing First-in-Class Agent
The U.S. FDA has approved Bristol Myers Squibb’s oral drug iberdomide, marketed as Zenbexus, for use with daratumumab and dexamethasone in patients...