The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it ...

In a Phase 1, first-in-human trial, a one-time infusion of an investigational CRISPR-Cas9 therapy targeting angiopoietin-like protein 3 (ANGPTL3) was safe and reduced LDL cholesterol by nearly 50% and ...

A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...

In biotech, success often begins in the lab — and for CRISPR pioneers, every clinical trial feels like a moonshot.

Only one in every six million people have the Rh null blood type. Now researchers are trying to grow it in the laboratory in ...

One of the rarest – the Rh null blood type – is found in just 50 known people in the world. Should they ever be in an ...

GATC brought together a broad coalition of researchers and executives in cell and gene therapy, gene editing, drug discovery and AI as well as patients and advocates.

The Sickle Cell Disease Treatment Market offers significant opportunities with advancements in gene editing, stem cell therapies, and precision medicine driving innovation. Increased awareness and ...

At AAPS PharmSci 360 2025, Elly Zhou says digital twins helped forecast the effects of drugs on human drugs via a digital control arm.

Investigators who designed a base-editing treatment are planning an "umbrella of umbrellas" trial, while others are developing roadmaps to guide personalized treatments.

UBS Global Healthcare Conference 2025 November 10, 2025 8:00 AM ESTCompany ParticipantsMaxwell Krakowiak - Senior VP ...

The Global Usher Syndrome Market is estimated to be valued at USD 2.29 Bn in 2025 and is expected to reach USD 3.20 Bn by 2032, exhibiting a compound annual growth rate (CAGR) of 4.9% from 2025 to ...