FDA grants priority review to uniQure's Huntington's gene therapy AMT-130, decision due May 1, 2027
The FDA accepted uniQure's BLA for its Huntington's disease gene therapy and set a May 1, 2027 PDUFA date, with an advisory committee also planned, marking a po
Yahoo Finance · +14 outlets
Washington state resident dies from bird flu after exposure to infected cattle, first US death in a year
The death underscores ongoing H5N1 transmission risk from dairy cattle to humans in the US.
STAT · +13 outlets
Karyopharm, deep in debt, to sell itself in bankruptcy; Gamida Cell parent is lead bidder
The court-supervised auction follows a forbearance deal to delay debt payments, adding to a wave of biotech restructurings.
Biopharmadive · +2 outlets
FDA finalizes cell and gene therapy potency guidance
The finalized guidance and related draft rules aim to standardize potency testing and streamline development of cell and gene therapies.
RAPS · +13 outlets
FDA approves Inovio's Voceryq, sending its stock surging
The speedy approval covers a novel therapy for a wart-like airway disease, a rare respiratory papillomatosis condition.
The Pharma Letter · +14 outlets
Merck's Keytruda biosimilars approach, but face an uphill battle for market share
As the patent cliff nears for Merck's flagship blockbuster, pricing and pharmacy benefit dynamics may limit biosimilar uptake.
Biopharmadive · +7 outlets
STAT+: FDA approves generic HIV prevention shot, widening access for lower-income countries
The generic lenacapavir approval is a key step toward expanding prevention access in dozens of lower-income countries.
STAT · +4 outlets
Moderna stock falls on $2B convertible notes deal as cancer vaccine rally is questioned
The financing comes amid investor focus on Moderna's personalized cancer vaccine program and competition with Merck's Keytruda.
TradingKey · +3 outlets
Hyderabad RNA foundry builds personalized cancer vaccines, with PopVax CEO calling them experimental
The Indian biotech's personalized mRNA cancer vaccine work reflects growing global activity, though executives caution the approach is not yet an established cu
Firstpost · +4 outlets
BioNTech's October: a lung-cancer signal, a modest breast-cancer win, and a missed mRNA endpoint
The mixed clinical results highlight the uneven progress of BioNTech's oncology pipeline beyond COVID vaccines.
ad-hoc-news.de · +1 outlets
STAT+: Psilocybin's lasting benefits prove elusive in mice across five Bay Area labs
The multi-lab comparison found limited lasting effects on anxiety and depression-like behavior, tempering enthusiasm for psychedelic therapeutics.
STAT · +1 outlets
Kymera's mid-stage eczema readout looms as a high-stakes test against Dupixent
Investors and doctors await results of the experimental therapy positioned to rival Sanofi and Regeneron's blockbuster.
STAT
Cell therapy investment grows as safety events complicate Big Pharma's CAR T autoimmune push
Rising funding for cell therapy contrasts with safety concerns that are slowing CAR T expansion into autoimmune disease.
BioSpace · +4 outlets
BridgeBio prepares rare muscular dystrophy launch with FDA approval in sight
The company is gearing up for commercialization of its therapy for a rare form of muscular dystrophy.
BioSpace · +1 outlets
Drugmakers rush to pair with Summit's ivonescimab ahead of a key verdict
Partnering interest is building around the bispecific antibody as a pivotal trial readout approaches.
BioSpace · +1 outlets
FDA places partial hold on Bright Minds' seizure trial over toxicity in mice
The clinical hold dims the company's outlook for its experimental epilepsy therapy.
Fierce Biotech · +9 outlets
Study identifies translation regulation mechanisms behind cancer development and treatment resistance
The findings on protein translation pathways could inform new targets to overcome therapy resistance.
EurekAlert! · +1 outlets
Merck stock rises as its cancer strategy gets personal
Investor sentiment improves on Merck's personalized cancer vaccine efforts alongside its Keytruda franchise.
TheStreet · +1 outlets