CRISPR Therapeutics
NEWS
2022 featured pivotal data that could change the landscape in non-alcoholic steatohepatitis, Alzheimer’s disease, sickle cell disease and many more indications.
The FDA lifted its partial clinical hold on bluebird’s experimental sickle cell disease gene therapy for patients younger than 18.
PACT Pharma shared results of a first-of-its-kind trial using CRISPR technology to swap a gene in a patient’s immune cell to treat solid tumors.
While worthy advances have recently been made in sickle cell disease, companies such as Graphite Bio, bluebird bio, Vertex Pharmaceuticals and Editas Medicine have loftier ambitions.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
The FDA has granted a rolling review to CRISPR Therapeutics and Vertex’s exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
CRISPR Therapeutics, Clovis Oncology and VistaGen all released Q2 results this week, while GreenLight Biosciences has some extra cash to work with.
Vertex Pharmaceuticals plans to acquire ViaCyte for $320 million in cash. ViaCyte focuses on stem cell-derived cell replacement therapies that they hope will cure type 1 diabetes (T1D).
Precision BioSciences and Novartis are teaming up to develop a potential cure for hemoglobinopathies such as sickle cell disease and beta-thalassemia.
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