Phase 1
Company is now preparing a Series B funding round to finance the Phase 2 clinical program
SMA is a severe neuromuscular disease caused by a mutation in the SMN1 gene, which codes for SMN, a protein necessary for motor neuron function.
The annual meeting of the American Society of Gene & Cell Therapy (ASGCT) was held in Washington, DC this week, with literally hundreds of abstracts and presentations. Here’s a look at some of the highlights.
Celgene Corporation and bluebird bio published interim data from CRB-401, their Phase I trial of bb2121, their CAR-T therapy in patients with relapsed and refractory multiple myeloma.
In its announcement, GSK said the decision to cut the two programs were made following an analysis of available research results, including interim data from an ongoing Phase I study of the flu vaccine.
Companies across Europe and Asia provide information about deal, clinical trials and more, with news from Synaptive Medical, Epic, Inventiva, Velabs, and more.
St. Jude Children’s Research Hospital published data from its Phase I/II clinical trial of its gene therapy for SXCID or SCID-X1, also dubbed “bubble boy disease.” The research was published in the New England Journal of Medicine.
Pharma and biotechs from across the globe share news, pipeline updates, with stories from Vetter, Atomwise, Tessa, GenSight, and more.
The first clinical trials using CRISPR technology are expected to get underway in the United States, even as echoes of a scandal involving the use of CRISPR on embryos in China are still being felt across the globe.
April 17, 2019 is the 29th World Hemophilia Day, a designated day to create awareness of the disease. Hemophilia is one of a number of bleeding disorders. The blood of people with hemophilia does not clot normally as the result of insufficient clotting factor. As a result, they can bleed for a longer time.
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