uniQure wins FDA priority review for Huntington's gene therapy, decision due May 2027
An FDA accelerated decision on ifezuntirgene inilparvovec would give the Huntington's field one of its first gene therapies after a difficult year for uniQure.
Yahoo Finance · +14 outlets
Karyopharm files Chapter 11, names Ayrmid lead bidder in planned merger
The bankruptcy filing marks a rare Chapter 11 sale process for a publicly traded oncology company.
Fierce Pharma · +2 outlets
FDA approves GSK's functional cure for hepatitis B
A functional cure would shift chronic hepatitis B treatment from lifelong suppression toward finite regimens.
Drug Discovery News · +14 outlets
Keytruda biosimilars approach, but pricing and PBM dynamics may blunt uptake
Merck's flagship faces a patent cliff, yet analysts see structural barriers limiting how much share biosimilars win.
Biopharmadive · +2 outlets
Kymera eczema readout looms as high-stakes test of a Dupixent rival
Mid-stage results for the oral program will signal whether an alternative mechanism can challenge Dupixent in atopic dermatitis.
STAT
FDA places partial hold on Bright Minds' BMB-101 seizure trial over mouse toxicity
The clinical hold stalls a psychedelic-adjacent epilepsy program and dents the company's near-term outlook.
Mugglehead Investment Magazine · +7 outlets
Drugmakers rush to partner with Summit's ivonescimab ahead of key verdict
Interest in the PD-1/VEGF bispecific reflects its potential to reshape first-line lung cancer treatment if pivotal data hold.
BioSpace
ABC News: FDA approves breakthrough drug for advanced pancreatic cancer
Pancreatic cancer has among the lowest survival rates, making any new approved therapy notable.
Good Morning America
Indian startup PopVax builds mRNA foundry for personalised cancer vaccines
The Hyderabad facility aims to manufacture more than 100 personalised mRNA cancer vaccines in 12 months, a test of low-cost individualized manufacturing.
NDTV · +6 outlets
FDA delays PDUFA action on Agios' mitapivat sNDA for sickle cell disease
The delay pushes back a potential new oral option in sickle cell disease and adds regulatory uncertainty for Agios.
Genetic Engineering and Biotechnology Ne · +5 outlets
BridgeBio preps rare muscular dystrophy launch as FDA approval nears
A near-term approval would expand BridgeBio's commercial portfolio beyond its existing rare disease franchise.
BioSpace
Cell therapy investment rises as CAR T safety events complicate autoimmune push
Big Pharma interest in CAR T for autoimmune disease is growing even as safety signals give pause.
BioSpace · +2 outlets
BioNTech's October: Gilead trial pact, Melbourne plant, and two poor readouts
Mixed clinical results and continued infrastructure investment frame BioNTech's push beyond COVID vaccines.
ad-hoc-news.de
FDA executes on long-planned platform approach to gene therapy oversight
A platform-based regulatory framework could speed development of gene therapies built on shared vectors.
BioSpace · +1 outlets
Family-funded research helps bring Sanfilippo gene therapy to patients
The case highlights the growing role of patient-family philanthropy in financing gene therapy development.
SuaraGarut.ID · +3 outlets
Pharmalittle: drugmaker payments to Russian middlemen, a possible Dupixent rival
The report alleges four large drugmakers paid tens of millions to intermediaries supplying Russian government entities.
STAT
Gene editing shares jump on ESGCT data and FDA draft guidance
Positive conference data plus a new draft guidance lifted sentiment across gene editing names.
Genetic Engineering and Biotechnology Ne
Merck stock rises as its cancer strategy gets more personal
Merck's oncology narrative and raised guidance are steering investor attention past the looming Keytruda patent cliff.
TheStreet