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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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FDA Approves Emergency Use of Pfizer’s COVID Vaccine

The Pharma Data

“More than 100,000 in long-term care have died from this virus in the U.S. and our nursing homes are now experiencing the worst outbreak of new cases since last spring, with more than 2,000 residents succumbing to this virus each week.” When you don’t have that capacity, that means people will die.”

Vaccine 52
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FDA Approves First Rapid COVID Test for Home Use

The Pharma Data

The simple nasal swab test, developed by Lucira Health, requires a prescription and people under the age of 14 can’t perform the test on themselves, the FDA said in a statement. The California company said that clinical trials showed 100% of patients were able to perform the Lucira test in about two minutes.

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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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FDA Set to Approve Pfizer’s COVID Vaccine

The Pharma Data

Operation Warp Speed, the White House-led initiative to develop and distribute vaccines, has already said it plans to begin shipping the vaccine to all 50 states within 24 hours of an FDA approval. Instead, a modified version of the vaccine will be tested in a smaller trial set to begin in February, the newspaper said.

Vaccine 52
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New England Journal of Medicine Publishes 24-Week Results from Phase 3 Study Evaluating RINVOQ™ (upadacitinib) in Psoriatic Arthritis

The Pharma Data

” Recently, the European Commission (EC) approved RINVOQ (15 mg) for use in adults with active psoriatic arthritis. . ” The safety profile of upadacitinib was generally similar to results reported previously in rheumatoid arthritis trials. The long-term extension of the trial is ongoing. About RINVOQ (upadacitinib).

Doctors 52
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Topical Gene Therapy FDA-Approved for Severe Skin Disease, Dystrophic Epidermolysis Bullosa

PLOS: DNA Science

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.