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Sarepta to file for gene therapy approval in ultra‑rare LGMD2E
Sarepta Therapeutics reported data showing its experimental gene therapy increased expression of the missing gene in limb‑girdle muscular dystrophy type 2E (LGMD2E) and said it plans to file for...
FDA orphan‑drug office leadership change: director reassigned
The FDA reassigned the director of the office responsible for orphan drug designations, an internal realignment confirmed to industry press. The official, Sandra Retzky, was removed from the...
FDA clears dordaviprone for H3(K27M) DMG: first systemic therapy
The FDA granted accelerated approval to dordaviprone for diffuse midline glioma (DMG) harboring the H3(K27M) mutation in patients aged one year and older with progressive disease after prior...
Chinese team implants genetically modified pig liver segment into patient
Surgeons in China reported transplanting a genetically modified pig liver segment into a patient with a liver tumor, marking what researchers describe as the first case of a xenogeneic liver graft...
Long Life Family Study taps PacBio HiFi for large‑scale longevity sequencing
The Long Life Family Study selected PacBio’s Revio HiFi long‑read sequencing platform to generate genomic and epigenomic data from up to 7,800 participants to uncover genetic contributors to...
Chiesi partners with Arbor on in vivo CRISPR for rare liver disease
Chiesi Global Rare Diseases and Arbor Biotechnologies launched a collaboration to develop an in vivo CRISPR‑based therapy targeting primary hyperoxaluria type 1 (PH1), with Chiesi committing up to...
Evommune files for IPO — public debut to fund mid‑stage immune programs
Evommune filed to go public to raise capital for development of two mid‑stage assets targeting chronic inflammatory and atopic diseases. The company seeks proceeds to advance EVO756, an oral...
Novo doubles down on MASH: $4.7B Akero takeover
Novo Nordisk agreed to acquire Akero Therapeutics in a cash deal valuing the company at $4.7 billion, plus contingent value rights that could push the total to about $5.2 billion. The deal secures...
BMS pays $1.5B for Orbital – in vivo CAR‑T enters big‑pharma fold
Bristol Myers Squibb agreed to acquire Orbital Therapeutics for $1.5 billion in cash, buying access to Orbital’s in vivo CAR‑T technology and lead autoimmune program OTX‑201. BMS said the purchase...
Novo shutters cell‑therapy unit – 250 roles cut amid reorg
Novo Nordisk announced it will discontinue its cell therapy R&D activities and lay off nearly all 250 employees in that division as part of a larger corporate reorganization led by CEO Maziar Mike...
FDA clears first DMG systemic therapy: dordaviprone approved
The FDA granted accelerated approval to dordaviprone for diffuse midline glioma (DMG) harboring the H3(K27M) mutation in patients aged one year and older with progressive disease after prior...
Sarepta files for LGMD approval – gene therapy momentum in rare muscle disease
Sarepta Therapeutics presented data showing its experimental gene therapy increased expression of the missing gene in limb girdle muscular dystrophy (LGMD) 2E and said it plans to file for...
Genetically modified pig liver supports human metabolism for 38 days
Chinese surgeons transplanted a genetically modified pig liver segment into a patient with a liver tumor at risk of rupture; the xenograft operated as an auxiliary graft and supported the...
Pan‑disease blood proteome maps 9,000 samples across 59 conditions
An international team led by SciLifeLab and KTH published a large proteomic survey profiling blood from more than 9,000 individuals spanning 59 diseases using high‑throughput Olink assays. The...
Shuttle Pharma buys Molecule.ai – $10M leap into AI drug discovery
Shuttle Pharmaceuticals submitted a letter of intent to acquire Molecule.ai for $10 million in cash and stock to incorporate the company’s AI models into Shuttle’s drug discovery workflows....
Senate tacks Biosecure Act onto NDAA – industry‑level access limits move forward
The U.S. Senate passed a revised Biosecure Act as an amendment to the National Defense Authorization Act, advancing legislation that would restrict federal funding and contracting with certain...
Novo buys Akero for $4.7B — blockbuster push into MASH
Novo Nordisk reached an agreement to acquire Akero Therapeutics for approximately $4.7 billion in cash, adding the FGF21 analogue efruxifermin to its metabolic pipeline. The deal includes...
BMS buys Orbital for $1.5B — in vivo CAR-T race heats up
Bristol Myers Squibb offered $1.5 billion in cash to acquire Orbital Therapeutics, a start‑up developing in vivo CAR‑T approaches, according to reports. The acquisition brings Orbital’s lead...
Partial pig liver transplant supports human patient…then removed
A Chinese surgical team reported transplanting a genetically modified pig liver segment into a human patient with a hepatic tumor, documenting 38 days of graft support before removal due to...
Sarepta to seek LGMD gene therapy approval — first in class possible
Sarepta Therapeutics presented data showing its experimental gene therapy raised expression of the missing gene in limb‑girdle muscular dystrophy type 2E (LGMD2E) and announced plans to file for...