Editas Medicine

The U.S. Court of Appeals for the Federal Circuit has partially vacated the Patent Trial and Appeal Board's previous decision in the CRISPR/Cas9 patent interference case, remanding it for further review.

CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.

The global CRISPR-Cas9 therapy market is rapidly expanding with over 25 therapies currently in clinical trials, many of which are expected to receive regulatory approval and enter the market within the next 5-7 years.

Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer.

Editas Medicine achieved in vivo preclinical proof of concept by editing hematopoietic stem cells in non-human primates, a crucial step for sickle cell disease and beta thalassemia treatment.

• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen. • AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment. • YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.

Editas Medicine's reni-cel (EDIT-301) demonstrates a 100% vaso-occlusive crises-free rate in SCD patients during follow-up, with sustained hemoglobin levels.

• CRISPR-Cas9 gene editing has achieved a significant milestone with FDA approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • Clinical trials are underway for CRISPR-based therapies targeting urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, and cardiovascular diseases. • CRISPR technology is being explored for chronic conditions like type 1 diabetes, systemic lupus erythematosus, and HIV, with early trials showing potential for disease management. • Challenges remain in addressing the high costs of CRISPR therapies and establishing regulatory standards to manage potential off-target effects and ethical implications.

A CRISPR-based gene editing therapy, EDIT-101, has shown promising results in treating Leber Congenital Amaurosis (LCA) Type 10, a rare genetic form of blindness.

CRISPR-based therapies are entering a new phase with the first approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia.