The firm expects its $1.25 billion cash runway to cover operating and capital expenses through 2029, including development plans for key assets.

Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced Dyno-yp2, a novel adeno-associated ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

Thanks to genetic science, gene editing, and techniques like cloning, it’s now possible to move DNA through time, studying ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA ...

Two variants in a single gene are implicated in almost all Alzheimer’s and nearly half of all dementia cases, according to ...

ASPIRO is the first multi-patient, multi-center clinical trial of an autologous cell therapy for Parkinson's disease. It is evaluating safety, tolerability, and preliminary efficacy of ANPD001 in ...

For decades, restoring sight after severe eye damage sat firmly in the realm of science fiction. Now a wave of converging ...

Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, today announced that the final patient has ...

A baby born with a rare and life-threatening genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene editing treatment at Children’s Hospital of Philadelphia. KJ ...

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

Addition Therapeutics has emerged from stealth with $100 million in hand to develop new types of gene therapies that might work against rare as well as chronic diseases. Spun out of research at the ...