BioSpace

Gene therapy leaders aim to help more Baby KJs with novel regulatory models

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...

The breakthrough: High Point native honored for her work in gene therapy

When Santa Claus brought 10-year-old Kathy High a junior chemistry set for Christmas more than 60 years ago, he never could’ve imagined what an incredible gift he was giving her. And ...

Gene therapy targets untreatable cystic fibrosis mutation affecting about 10% of patients

Cystic fibrosis is among the most common, known and studied genetic diseases. It affects over 100,000 people worldwide and ...

Measles spreading in the shadows; plus a cancer-fighting vaccine benefit you may not know and a gene therapy breakthrough

Measles is spreading in the shadows and could the HPV vaccine be beneficial to men too? Here are your top health stories for ...

Regeneron wins gene therapy nod, signs MFN pact

The final major drugmaker President Donald Trump had pressed to accept a most-favored-nation deal has signed an agreement ...
Managed Healthcare Executive

Facing the practical problems of gene therapy | AMCP Annual 2026

Providers and payers certainly face some formidable challenges when it comes to gene therapy, but it pales in comparison to ...
Pharmaceutical Technology

Regeneron cuts White House pricing deal, offers new gene therapy for free

This White House deal will see Regeneron offer all its new medicines at “MFN prices”, while providing Otarmeni for free to eligible patients.

A Gene Therapy That Can Reverse Blindness Just Won a $3 Million Prize

Molecular biologist Jean Bennett and ophthalmologist Albert Maguire were awarded the $3 million Breakthrough Prize in Life ...