Preclinical
Sarepta Therapeutics announced Monday that its next-generation PPMO candidate, SRP-5051, showed promise in a Phase II trial when used as a treatment for Duchenne muscular dystrophy in patients amenable to exon 51 skipping.
The U.S. FDA is expected to give the go-ahead for Pfizer and BioNTech’s COVID-19 vaccine for adolescents ages 12 and older within the week.
The U.S. FDA approved Chiesi Global Rare Diseases’ Ferriprox for treatment of transfusional iron overload caused by sickle cell disease (SCD) or other anemias in adults and children ages three years and older. This approval expands the use of the drug for patients with SCD or other anemias.
The battle against COVID-19 rages on, and other therapeutics are making their way through the clinic, including monoclonal antibody treatments and antivirals. BioSpace takes a look at some of the ongoing efforts to stem the rate of infection from the virus.
Ohio-based Forge Biologics secured $120 million in a Series B financing round to support the clinical development of its gene therapy program for rare diseases, including the typically fatal Krabbe disease.
Researchers are continuing to develop next-generation therapeutics and oral vaccines that could overcome some vaccine hesitancy. Here are some of the top producers.
Top-line results from the Phase III INVIGORATE trial show Aldeyra Therapeutics’, reproxalap, significantly reduced ocular itching in patients with allergic conjunctivitis.
Please check out the biopharma industry’s COVID-19 stories that are trending for April 27, 2021.
There is hope for uniQure’s HOPE-B trial after all. Shares of uniQure NV are climbing this morning after the company announced the clinical hold on its hemophilia B gene therapy has been lifted by the U.S. Food and Drug Administration.
AstraZeneca and Sanofi announced that their MELODY Phase III trial of nirsevimab hit the primary endpoint in medically attended lower respiratory tract infections (LRTI) caused by RSV in healthy late preterm and term infants.
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