Phase I
A year after treatment, Vertex’s VX-880 has eliminated the need for insulin injections in two Type 1 diabetes patients in a Phase I/II clinical trial of its investigational stem cell-based therapy.
The Huntington’s disease space saw a flurry of activity Wednesday as PTC and uniQure released data from their respective mid-phase trials.
The Bespoke Gene Therapy Consortium intends to bring AAV-based gene therapies to patients whose diseases are often ignored by commercial interests.
Eli Lilly has secured the rights to Verve Therapeutics’ gene editing approach, a “one-and-done” method that the companies hope will lower the cardiovascular risk factor lipoprotein(a).
Following cases of serious bleeding in patients, five of which were fatal, the regulator has put Mersana’s investigational antibody-drug conjugate UpRi on partial clinical hold in two ovarian cancer trials.
The company announced Monday with its second-quarter earnings that the regulator has officially halted the Phase I study after a child with acute myeloid leukemia died following treatment.
The initial public offering on the Nasdaq is a last-ditch effort by the biotech as there is “substantial doubt” as to the company’s “ability to continue as a going concern” without the IPO, according to its SEC filing.
The company is paying $3.2 billion upfront in cash for Chinook’s two immunoglobulin A nephropathy candidates, atrasentan and zigakibart, which will complement its own IgAN hopeful iptacopan.
Data show the potential of Editas’ sickle cell disease and transfusion-dependent beta thalassemia gene therapy candidate, but it might not be enough to overtake Vertex and CRISPR Therapeutics.
The FDA has launched a new super office to prepare for myriad decisions on cell and gene therapies, including the potential first CRISPR therapy and the first gene therapy for Duchenne muscular dystrophy.
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