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Rare‑disease gene therapy heats up: Sarepta files — Genethon moves on
Sarepta Therapeutics reported data showing its experimental gene therapy raised levels of a missing protein in limb girdle muscular dystrophy 2E (LGMD2E) and signaled plans to file for regulatory...
Pharma courts big‑scale genomics: Illumina partners — PacBio wins longevity study
Illumina’s Alliance for Genomic Discovery added Alnylam as a partner, expanding pharma access to a large, clinically annotated genomic dataset sequenced from Vanderbilt’s BioVU biobank, per...
Single‑cell tech race: new RT exposes hidden transcripts — patent ruling shakes market
RNAconnect released preprint data for UltraMarathonRT, a group II intron reverse transcriptase that reportedly captures more full‑length transcripts in single‑cell RNA‑seq than retroviral RTs,...
Partial pig liver transplant supports human metabolism for 38 days
Chinese surgeons reported the first documented case of a genetically modified pig liver segment transplanted as an auxiliary graft into a human patient with cancer; the xenograft supported...
Big pharma bets on AI discovery: AstraZeneca ties to Algen; Shuttle buys Molecule.ai
AstraZeneca struck a multi‑year collaboration worth up to $555 million with Algen Biotechnologies to apply Algen’s CRISPR‑AI platform to identify therapeutic targets, validating functional...
Tempus wins $60.5M ARPA‑H contract for precision cancer platform
Tempus AI secured a $60.5 million, five‑year contract from ARPA‑H to provide testing and contract research organization services for the ADAPT precision cancer therapy program. Under the award,...
Novo buys Akero for $4.7B – bolsters MASH pipeline
Novo Nordisk agreed to acquire Akero Therapeutics for an upfront $4.7 billion to add efruxifermin, an FGF21 analog in late‑stage development for metabolic dysfunction‑associated steatohepatitis...
Bristol Myers shells out $1.5B – doubles down on in vivo CAR‑T
Bristol Myers Squibb agreed to acquire Orbital Therapeutics for $1.5 billion in cash to obtain Orbital’s in vivo CAR‑T platform and lead program OTX‑201 aimed at autoimmune indications. The...
Senate moves on Biosecure Act: new limits tied to defense bill
The U.S. Senate passed a revised Biosecure Act as an amendment to the National Defense Authorization Act, advancing language that would restrict federal funding and contracting with biotechnology...
Genomic infrastructure expands: pharma taps large‑scale long‑read and biobank datasets
Illumina’s Alliance for Genomic Discovery added pharmaceutical partners as the project continues to amass hundreds of thousands of sequenced genomes linked to clinical records, drawing interest...
Big AI bets: AstraZeneca and smaller acquirers back AI‑driven discovery
AstraZeneca struck a multi‑year collaboration with Algen Biotechnologies valued up to $555 million to apply Algen’s CRISPR‑AI functional genomics platform to target discovery in immunology. The...
Mammoth posts primate proof‑of‑concept for triglyceride gene edit
Mammoth Biosciences released nonhuman primate data for its lead program: a gene editing therapy designed to durably lower triglycerides. The preclinical monkey results showed on‑target editing and...
Parse Bio wins court ruling: Scale patent invalidated
A U.S. district court found key claims of a Scale Biosciences patent invalid for failing written‑description and enablement requirements in a lawsuit where Parse Biosciences is the defendant. The...
ARPA‑H awards flow: Tempus and Tessera win major program funding
Tempus AI was awarded a $60.5 million five‑year contract by ARPA‑H to provide testing and CRO services for the ADAPT precision cancer therapy program, supplying whole transcriptome, exome, liquid...
Evommune files for IPO — funding late‑stage autoimmune trials
Evommune filed confidentially to go public to raise capital for two mid‑stage programs: EVO756, an oral MRGPRX2 antagonist for chronic spontaneous urticaria and atopic dermatitis, and EVO301, an...
Gene therapy momentum: Sarepta files for LGMD approval; Chiesi funds Arbor CRISPR pact
Sarepta Therapeutics reported data showing its experimental gene therapy raised levels of the missing protein in limb‑girdle muscular dystrophy (LGMD) 2E and said it will seek regulatory...
Novo buys Akero for $4.7B — bolsters MASH pipeline
Novo Nordisk agreed to acquire Akero Therapeutics for $4.7 billion in cash, taking ownership of Akero’s late‑stage FGF21 analogue efruxifermin and related assets. The deal includes contingent...
Bristol Myers to buy Orbital for $1.5B — deepens in vivo CAR‑T push
Bristol Myers Squibb is acquiring Orbital Therapeutics for $1.5 billion in cash to add Orbital’s in vivo CAR‑T platform and preclinical CD19 autoimmune programs to its pipeline. The deal expands...
ARPA‑H funds Tessera’s in vivo CAR‑T work — up to $41.3M award
Tessera Therapeutics has been awarded up to $41.3 million from ARPA‑H to support its EMBODY program aimed at engineering immune cells directly inside the body. The funding will accelerate...
Chiesi inks Arbor alliance — $115M to back in vivo CRISPR for rare liver disease
Chiesi Global Rare Diseases committed up to $115 million to partner with Arbor Biotechnologies on an in vivo CRISPR gene‑editing candidate for primary hyperoxaluria type 1 (PH1) and other liver...