Drug Development
Despite a disappointing showing in the Phase III MOVE-FA trial, PTC Therapeutics says it will move forward with regulatory talks for the Friedreich’s ataxia treatment. Meanwhile, the company is discontinuing several early-stage gene therapies and reducing its headcount.
FEATURED STORIES
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
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Black/African American hematological cancer patients tend to have the worst outcomes. This is partly driven by a lack of access to allogeneic hematopoietic stem cell transplant and available donors.
The model has been validated against clinically relevant IBD therapies to demonstrate efficacy and mechanism of action, enabling drug developers to gain more accurate therapeutic insights.
In a unanimous vote, a panel of FDA advisors voted to recommend the approval of Moderna’s COVID-19 vaccine for children between the ages of six and 17 years old.
MorphoSys U.S. Inc., Pfizer and Incyte are teaming up to test Pfizer’s TTI-622 in conjunction with Monjuvi plus lenalidomide in patients with lymphoma.
Day One Biopharmaceuticals announced positive early data assessing tovorafenib as a once-a-week treatment in people aged six months to 25 years with relapsed or progressive pediatric low-grade glioma.
The FDA has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults.
Soleno announced promising results of its diazoxide choline extended-release tablets in patients with Prader-Willi syndrome at ENDO 2022.
Eli Lilly’s Olumiant (baricitinib) received approval from the FDA for the treatment of severe alopecia areata, making it the first systemic treatment for adults living with the disease.
Treatments for overactive bladder are not one size fits all, which is why Urovant is advancing URO-902, an injectable gene therapy that is currently being investigated in Phase IIa clinical trials.
Shares of CRISPR Therapeutics fell more than 11% on Monday as investors react negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.