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FDA and uniQure clash: agency demands randomized sham control for Huntington’s gene therapy
A senior FDA official publicly rejected uniQure’s characterization of agency actions and defended a request for a randomized, sham‑controlled trial of AMT‑130, the company’s intracranial gene...
FDA converts Tecvayli accelerated approval and greenlights Tec‑Dara combo under priority voucher
The FDA granted full approval to Johnson & Johnson’s Tecvayli as a monotherapy and cleared the Tecvayli plus Darzalex Faspro combination for previously treated relapsed or refractory multiple...
Servier to buy Day One for $2.5B: acquires pediatric brain‑tumor drug Ojemda
Servier agreed to acquire Day One Biopharmaceuticals for roughly $2.5 billion in cash, securing global rights to the marketed pediatric low‑grade glioma drug Ojemda and Day One’s rare oncology...
Tenaya and Alnylam sign $1B‑plus cardio research pact: $10M upfront, big milestones
Tenaya Therapeutics struck a research collaboration with Alnylam Pharmaceuticals to identify up to 15 genetic targets for cardiovascular disease discovery, with $10 million upfront and potential...
Moderna to pay up to $2.25B to settle lipid nanoparticle patent fight
Moderna agreed to pay up to $2.25 billion to Arbutus Biopharma and Genevant Sciences to resolve a longstanding dispute over lipid nanoparticle (LNP) delivery technology used in mRNA vaccines. The...
FDA places partial hold on PepGen’s DM1 trial after reviewers flag preclinical signals
The FDA placed a partial clinical hold on PepGen’s Freedom2‑DM1 Phase II multiple ascending‑dose trial of PGN‑EDODM1 for myotonic dystrophy type 1, citing concerns from previously submitted...
Kestrel clears IND for oral pan‑KRAS KST‑6051, moves toward first‑in‑human trial
Kestrel Therapeutics received FDA clearance of an IND for KST‑6051, an oral small‑molecule pan‑KRAS inhibitor targeting KRAS‑driven cancers, and plans to start a first‑in‑human Phase I FALCON...
Illumina expands pharma research alliance with 50,000 genomes paired to proteomics
Illumina announced it will add 50,000 whole genomes with matched proteomic data to the Alliance for Genomic Discovery (AGD), with DeCode Genetics generating protein data using Illumina’s Protein...
Liquid biopsy moves beyond cancer: cfDNA fragmentomics detects liver fibrosis and chronic disease indicators
Two independent reports detailed advances in fragmentomics‑based liquid biopsy: Johns Hopkins published a prototype cfDNA fragmentomics test that identifies liver fibrosis, cirrhosis, and chronic...
UCB licenses Antengene’s ATG‑201 in $1.1B autoimmune deal: bispecific T‑cell engager pact
UCB struck a global licensing agreement with Antengene for ATG‑201, a CD19/CD3 bispecific T‑cell engager designed to deplete pathogenic B cells in autoimmune diseases, with $60 million upfront...
Moderna to pay up to $2.25B: patent settlement ends LNP fight
Moderna agreed to a multi-hundred-million to multibillion-dollar settlement to resolve a long‑running dispute over lipid nanoparticle (LNP) delivery technology used in its COVID‑19 vaccine. The...
UCB inks $1.1B deal... Antengene’s CD19/CD3 T‑cell engager
UCB secured global rights to ATG‑201, a CD19/CD3 bispecific T‑cell engager (TCE) from Antengene in a deal that could total more than $1.1 billion in milestones plus tiered royalties. Antengene...
Zealand’s amylin analog posts Phase 2 gains – Roche backs tolerability
Zealand Pharma released Phase 2 data for petrelintide, an amylin analog partnered with Roche, showing up to ~10.7% mean weight loss at 42 weeks with what Zealand characterized as "placebo‑like"...
Lilly pushes employers: new programs aim to broaden GLP‑1 access
Eli Lilly launched programs to expand employer coverage and alternative access pathways for its GLP‑1 obesity drugs, including an employer‑focused service and a platform to ease workplace...
Liquid biopsy beyond cancer: cfDNA fragmentomics flags liver disease
Johns Hopkins researchers reported an AI‑driven fragmentomics liquid biopsy that identifies liver fibrosis and cirrhosis from genome‑wide cell‑free DNA (cfDNA) fragmentation patterns. The team...
FDA places partial hold on PepGen’s DM1 trial – shares tumble
The U.S. Food and Drug Administration placed a partial clinical hold on PepGen’s Freedom2 Phase II program testing PGN‑EDODM1 in myotonic dystrophy type 1 (DM1), citing questions about preclinical...
Evo 2: DNA foundation model scales genome design across life
Researchers published Evo 2, a generative DNA foundation model trained on genomes from more than 100,000 species, and demonstrated its ability to identify disease‑causing human variants and design...
UCLA builds next‑gen CAR‑T to breach solid tumor resistance
UCLA scientists reported a next‑generation CAR‑T cell engineered to overcome immunosuppressive barriers in solid tumors, addressing a principal limitation that has constrained CAR‑T success...
Zorevunersen reduces seizures dramatically: first Dravet gene therapy data
Clinical trial results published in NEJM showed zorevunersen—an investigational gene regulation therapy for Dravet syndrome—cut seizure frequency by large margins and improved quality‑of‑life...
Illumina adds proteomics to pharma research alliance—50k paired datasets
Illumina announced an expansion of its Alliance for Genomic Discovery, committing to add 50,000 genomes paired with proteomic profiles to the consortium’s dataset, generated by DeCode Genetics...