Breaking Gene Therapy News

Nearly 1 million Belgians have donated to get lifesaving gene therapy for a baby - New York Daily News

Sep 18, 2019 | 23:42 pm

Nearly 1 million Belgians have donated to get lifesaving gene therapy for a baby New York Daily NewsNearly a million Belgians have donated money to get a baby lifesaving gene therapy for an otherwise fatal condition.

Why an RTP gene therapy company just acquired a competing delivery technology - Triangle Business Journal

Sep 18, 2019 | 20:45 pm

Why an RTP gene therapy company just acquired a competing delivery technology Triangle Business JournalSince raking in its first large venture capital funding earlier this year, a Triangle gene therapy company has been making a number of strategic acquisitions.

Ocugen gene therapy receives orphan drug designation - Healio

Sep 18, 2019 | 20:02 pm

Ocugen gene therapy receives orphan drug designation HealioOCU400, a gene therapy candidate, has received a second orphan drug designation from the FDA for the treatment of CEP290 mutation associated retinal ...

First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 - Fabry Disease News

Sep 18, 2019 | 19:30 pm

First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 Fabry Disease NewsThe first patient in a Phase 1/2 clinical trial of Fabry disease gene therapy candidate FLT190 has been dosed. Enrollment is ongoing at the Royal Free Hospital, ...

The rise, fall and resurgence of gene therapy - Phys.Org

Sep 18, 2019 | 18:09 pm

The rise, fall and resurgence of gene therapy Phys.OrgTwenty years ago, scientists and investors were giddy over the promise of gene therapy to cure a host of deadly diseases. But the tragic death of a young man ...

Ocugen Granted FDA Orphan Drug Designation for OCU400 (NR2E3) Gene Therapy for the Treatment of CEP290 Mutation Associated Retinal Disease - GlobeNewswire

Sep 18, 2019 | 13:00 pm

Ocugen Granted FDA Orphan Drug Designation for OCU400 (NR2E3) Gene Therapy for the Treatment of CEP290 Mutation Associated Retinal Disease GlobeNewswireMALVERN, Pa., Sept. 18, 2019 (GLOBE NEWSWIRE) -- Ocugen, Inc., a clinical stage biopharmaceutical company focused on discovering, developing and ...

Five benefits of gene therapies - Echo Live

Sep 18, 2019 | 12:05 pm

Five benefits of gene therapies Echo LiveAs an increasing numbers of gene therapies are available as health treatments, Lisa Salmon finds out some of the ways it's used.

Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz

Sep 18, 2019 | 01:35 am

Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy BioBuzzCell and gene therapy companies are on the verge of transforming how we treat unmet medical needs such as cancer, rare diseases, genetic disorders and ...

Alabama Man Free of Sickle Cell After Gene Therapy - WebMD

Sep 17, 2019 | 16:50 pm

Alabama Man Free of Sickle Cell After Gene Therapy WebMDAn Alabama man is free of sickle cell disease after receiving gene therapy for two years as part of a clinical trial.

Dr Ahmar Zaidi: How Social Determinants Could Limit Access to Gene Therapy for Sickle Cell - AJMC.com Managed Markets Network

Sep 17, 2019 | 15:21 pm

Dr Ahmar Zaidi: How Social Determinants Could Limit Access to Gene Therapy for Sickle Cell AJMC.com Managed Markets NetworkThe only thing that should matter in these patients is their genetic code, but often it's your zip code that really restrains your ability to access the healthcare you ...

FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire

Sep 17, 2019 | 11:49 am

FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial WRAL Tech WireDURHAM - Precision BioSciences, a genome-editing company based in Durham, has received authorization from the U.S. Food and Drug Administration to ...

7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News

Sep 16, 2019 | 16:00 pm

7-Year-Old Receives New FDA-Approved Retina Gene Therapy University of Michigan Health System NewsGene therapy may prevent blindness from rare eye disease Leber congenital amaurosis.

Gene Therapy Drugs That Cost Millions Have Employers and Health Plans Worried - Insurance Journal

Sep 13, 2019 | 07:03 am

Gene Therapy Drugs That Cost Millions Have Employers and Health Plans Worried Insurance JournalEmployers and health plans fear they could be swamped financially by a coming wave of expensive new genetic therapies for rare diseases. Regulators have.

The redemption of James Wilson, gene therapy pioneer - Chemical & Engineering News

Sep 12, 2019 | 22:16 pm

The redemption of James Wilson, gene therapy pioneer Chemical & Engineering NewsOn Sept. 17, 1999, Jesse Gelsinger died after receiving an experimental gene therapy from James Wilson's lab at the University of Pennsylvania. That tragedy ...

Gene Therapy Repairs Stroke Damage and Improves Motor Function - Genetic Engineering & Biotechnology News

Sep 12, 2019 | 17:00 pm

Gene Therapy Repairs Stroke Damage and Improves Motor Function Genetic Engineering & Biotechnology NewsA new gene therapy turns glial cells into neurons, repairing damage that results from stroke and significantly improving motor function in mice.

Gene therapy helps functional recovery after stroke - Science Daily

Sep 12, 2019 | 00:49 am

Gene therapy helps functional recovery after stroke Science DailyA new gene therapy turns glial cells -- abundant support cells in the brain -- into neurons, repairing damage that results from stroke and significantly improving ...

Is treatment forever? Success of gene therapy for inherited blindness depends on timing - Penn: Office of University Communications

Sep 10, 2019 | 09:00 am

Is treatment forever? Success of gene therapy for inherited blindness depends on timing Penn: Office of University CommunicationsAn FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved ...

Success of gene therapy for a form of inherited blindness depends on timing - Science Daily

Sep 9, 2019 | 09:00 am

Success of gene therapy for a form of inherited blindness depends on timing Science DailyAn FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved ...

Pharmacquired: Vertex has an appetite for deals. Gene therapy is on the menu - BioPharma Dive

Sep 5, 2019 | 09:00 am

Pharmacquired: Vertex has an appetite for deals. Gene therapy is on the menu BioPharma DiveCreating a toolbox — that's how Vertex CEO Jeffrey Leiden sees business development. While best known for small-molecule drugs, the Boston biotech has ...

Meeting the Challenge of Gene Therapy Manufacturing - Genetic Engineering & Biotechnology News

Sep 3, 2019 | 09:00 am

Meeting the Challenge of Gene Therapy Manufacturing Genetic Engineering & Biotechnology NewsSponsored *content* brought to you by. Pall Biotech logo. Gene therapy has come a long way over a rocky road in the 30 years since the first patients received ...

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