The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
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Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.
Vertex Pharmaceuticals is amending its collaboration with CRISPR Therapeutics, paying $900 million now for chance to grab a bigger share of a gene-editing therapy’s profits later---if the treatment is approved. Clinical trials evaluating the CRISPR-based therapy are underway in sickle cell disease and beta thalassemia.
Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
The Alliance for Regenerative Medicine and 13 biotech companies released a statement outlining bioethical principles on genome editing, including opposition to modifications that can be passed to future generations.
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The Phase I/II trial is in beta-thalassemia, a rare blood disorder. Another Phase I/II study of the same drug, in sickle cell disease, is expected to infuse its first patient in mid-2019.
Shares of gene-editing companies were down Tuesday morning following the news, but analysts sought to reassure investors about Editas.
The agency also accepted the two companies' IND to begin the Phase I/II study of CTX001 in patients with sickle cell disease, sending CRISPR shares up more than 11 percent Thursday morning.
In a deal worth up to $25 million for ViaCyte, the companies will work together to create allogeneic stem cell therapies to treat the disease.
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It follows similar moves by Editas Medicine and Intellia Therapeutics earlier this year.
CRISPR gene editing is heralded one of the most promising technologies today - and it's ramping up fast. Here's a commercial timeline - from just the past year.
The companies will develop new therapeutics targeted at blood disorders, blindness and congenital heart disease.
Vertex Pharmaceuticals, known for its line of cystic fibrosis drugs, has entered the gene editing space with a new $105 million deal with CRISPR Therapeutics.
The market potential of CRISPR technology has been legitimized by Editas Medicine's impressive, $120 million fundraise. Here are six more biotechs shaping the CRISPR landscape look like now? And what other players are in this game?