Web Results

www.genengnews.com/gen-edge/gene-therapy-pioneer-regeneron-partner-on-nasal...

The University of Pennsylvania's Gene Therapy Program will partner with Regeneron to explore whether its two-antibody “cocktail” REGEN-COV2 (casirivimab and imdevimab) can prevent COVID-19 ...

www.bizjournals.com/philadelphia/news/2020/11/30/penn-gene-therapy-james...

Gene therapy pioneer Dr. James Wilson and his team at the University of Pennsylvania have linked up with Regeneron Pharmaceuticals to test whether the New York biotechnology company's combination ...

abcnews.go.com/Health/wireStory/fda-blocks-anticipated-biomarin-hemophilia...

The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant to enable the body to produce FVIII on its own.

seekingalpha.com/news/3640000-prevails-gene-therapy-orphan-drug-in-europe-for...

Earlier, the gene therapy received US Orphan Drug and Fast Track designation for the same indication. Click to subscribe to real-time analytics on PRVL. Now read: ...

clarkstonconsulting.com/insights/fda-gene-therapy-guidances

On January 28, 2020, the highly anticipated final FDA gene therapy guidances were released. In total, 7 guidance documents were issued, focusing on gene therapy topics for organ drugs, specific diseases, Chemistry, Manufacturing, and Controls (CMC) for Investigational New Drugs (IND), patient follow-up after drug administration, and testing on retroviral vector-based therapies.

www.zacks.com/stock/news/1107292/selectas-selb-gene-therapy-gets-orphan-drug...

The FDA bestows an orphan drug status on Selecta's (SELB) gene therapy candidate MMA-101 for the treatment of isolated methylmalonic acidemia. The candidate already has a Rare Pediatric Disease tag.

medlineplus.gov/genetics/understanding/therapy/availability

Gene therapy is currently available primarily in a research setting. The U.S. Food and Drug Administration (FDA) has approved only a limited number of gene therapy products for sale in the United States.. Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS.

www.marketscreener.com/news/latest/Neurogene-Announces-EMA-Grants-Orphan-Drug...

“The EMA granting Orphan Drug Designation for a potential gene therapy for AGU represents promise while underscoring the urgent, unmet medical need for patients around the world diagnosed with this condition.” The AGU Orphan Drug Designation represents Neurogene’s third ODD for a rare neurological disease.

www.avexis.com

Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases. COOKIE NOTICE This site uses cookies to store information on your computer.

investingnews.com/.../genetics-investing/4-gene-therapy-stocks-to-watch

The company’s gene therapy product, Zynteglo, has been approved in the European Union, Iceland, Liechtenstein and Norway, and is the first and only one time gene therapy treatment for infusion ...