CRISPR

R&D

Deep Dive: Research and Development 2024

In this edition of Deep Dive, delve into the revolutionary world of genetic editing as we trace the development of CRISPR, discover why Alexion’s Soraya Bekkali believes now it the time to champion

R&D

The opportunities and challenges facing rare disease therapi...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.

R&D

The innovative future of CRISPR tech

CRISPR-based therapies have moved from theory to reality, after the recent landmark approval of the first such treatment by the FDA.

R&D

JP Morgan 2024 – Jonah Comstock talks with Trevor Martin

JP Morgan 2024 – Trevor Martin

Editor-in-chief Jonah Comstock caught up with Trevor Martin, co-founder and CEO of Mammoth Biosciences

News

Intellia’s president and chief executive John Leonard

Intellia’s CRISPR drug could offer one-shot HAE treatment

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, p

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A giemsa stained blood smear from a person with beta thalassaemia

First CRISPR drug Casgevy gets swift second approval

The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-