Remove 2020 Remove Therapies Remove Virus
article thumbnail

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
article thumbnail

AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Nature 578 , 229-236 (2020). References Doudna JA.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

World Antimicrobial Awareness Week 2020

The Pharma Data

HIV drug resistance is caused by changes in the genetic structure of HIV that affects the ability of drugs to block the replication of the virus. Over the past decade, the world has witnessed unprecedented scale-up of antiretroviral therapy (ART), which has saved the lives of tens of millions of people. Department information note.

article thumbnail

Analytical Testing Considerations for Gene Therapy Products

The Premier Consulting Blog

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

article thumbnail

Developing an mRNA therapy for Duchenne muscular dystrophy

Drug Target Review

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Because mRNA is not a virus, it doesn’t have to meet biosafety requirements for manufacturing and administration that an AAV approach would need. 2020 Jul 3;10(1):10967.

article thumbnail

Boehringer Ingelheim sees positive business momentum in 2020 despite impact of COVID-19

The Pharma Data

Strong R&D commitment to developing therapeutics against SARS-CoV-2 virus. billion EUR in 2020 (18.9% Boehringer Ingelheim stepped up its investments in R&D significantly in 2020 in pursuit of innovative medicines and therapies for diseases for which no satisfactory treatments are available. of net sales).

article thumbnail

Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes

The Pharma Data

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.