Breaking Gene Therapy News

Once a 'refugee,' a gene therapy pioneer finds a renewed calling - STAT

Feb 15, 2019 | 19:50 pm

Once a 'refugee,' a gene therapy pioneer finds a renewed calling STATWilson among Co-Founders of Gene Therapy Developer Launched with $115.5M Genetic Engineering & Biotechnology NewsJames Wilson turns to old mentor for gene therapy startup's star-studded $115M launch round Endpoints NewsWith 5 Gene Therapies Licensed From Penn, Passage Bio Launches With $115.5 Million Series A BioSpaceView full coverage on Google News

FDA finalizes guidance for expedited review of cell and gene therapy applications - Seeking Alpha

Feb 15, 2019 | 17:17 pm

FDA finalizes guidance for expedited review of cell and gene therapy applications Seeking AlphaIn (yet another) statement, FDA Commission Scott Gottlieb, M.D. announces the issuance of final updated guidance for gene therapy and cell therapy develope.

uniQure (QURE) Announces Publications on AAV Gene Therapy Approach to Treating ALS and FTD in Journal Molecular Therapy Nucleic Acids - StreetInsider.com

Feb 15, 2019 | 13:25 pm

uniQure (QURE) Announces Publications on AAV Gene Therapy Approach to Treating ALS and FTD in Journal Molecular Therapy Nucleic Acids StreetInsider.comGet instant alerts when news breaks on your stocks. Claim your 2-week free trial to StreetInsider Premium here. uniQure N.V. (NASDAQ: QURE), a leading gene ...

Dosing underway in study of Sarepta's gene therapy for MPS IIIA - Seeking Alpha

Feb 14, 2019 | 22:14 pm

Dosing underway in study of Sarepta's gene therapy for MPS IIIA Seeking AlphaLysogene and licensee Sarepta Therapeutics (NASDAQ:SRPT) announce that the first patient has been dosed in a Phase 2/3 clinical trial, AAVance, evaluating.

Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again - Xconomy

Feb 14, 2019 | 20:50 pm

Passage Bio Gets $116M As Wilson, Yamada Team Up On Gene Therapy Again XconomyLongtime biopharmaceutical executive Tachi Yamada helped keep the work of gene therapy pioneer James Wilson alive 20 years ago when Wilson was mired ...

Ocugen gene therapy receives orphan drug designation - Healio

Feb 14, 2019 | 20:40 pm

Ocugen gene therapy receives orphan drug designation HealioThe FDA has granted orphan drug designation to OCU400, Ocugen's gene therapy for the treatment of NR2E3 mutation-associated retinal degenerative disease ...

Phase 2 Data of Gene Therapy Show Increased Factor IX Activity in Hemophilia B - Specialty Pharmacy Times

Feb 14, 2019 | 16:03 pm

Phase 2 Data of Gene Therapy Show Increased Factor IX Activity in Hemophilia B Specialty Pharmacy TimesA small mid-stage study demonstrated promising results for gene therapy candidate AMT-061 in patients with severe hemophilia B.

Ocugen wins orphan drug status for ocular gene therapy - Mass Device

Feb 14, 2019 | 15:32 pm

Ocugen wins orphan drug status for ocular gene therapy Mass DeviceThe FDA has given Ocugen orphan drug status for a gene therapy designed to treat NR2E3 mutation-associated retinal degenerative disease. The Malvern ...

At 33.3% Soaring CAGR, Gene Therapy Market to Exhibit $4,402 Million by 2023 - PRNewswire

Feb 13, 2019 | 11:30 am

At 33.3% Soaring CAGR, Gene Therapy Market to Exhibit $4,402 Million by 2023 PRNewswireThe research study provides an in-depth analysis of the Global Gene Therapy Market, covering aspects such as industry size and share, market classification,...

Boomerang Effect Of Gene Therapy on Oncology Contracting - Specialty Pharmacy Times

Feb 11, 2019 | 18:53 pm

Boomerang Effect Of Gene Therapy on Oncology Contracting Specialty Pharmacy TimesManaged care is undergoing a sea change in provider reimbursement. Historically rooted in fee-for-*service*, a movement is underway to shift payment to various ...

UniQure update bolsters outlook for hemophilia B gene therapy - BioPharma Dive

Feb 11, 2019 | 18:13 pm

UniQure update bolsters outlook for hemophilia B gene therapy BioPharma DiveTreatment with uniQure's experimental gene therapy for hemophilia B led to sustained increases in Factor IX activity levels in three patients for up to 16 weeks ...

Gene therapy increases factor IX activity in severe hemophilia B - Healio

Feb 8, 2019 | 17:07 pm

Gene therapy increases factor IX activity in severe hemophilia B HealioThe investigational gene therapy AMT-061 showed increases in factor IX activity sustained at 25% to 51% of normal among patients with severe or moderately ...

Gene therapy biotech shares sink on inconclusive trial results - pharmaphorum

Feb 8, 2019 | 12:10 pm

Gene therapy biotech shares sink on inconclusive trial results pharmaphorumShares in two US gene therapy biotechs have fallen after two cutting-edge treatments failed to produce convincing results in early clinical trials.

Gene Therapy Treatment Targets Rare Mutation Tied to Blindness - Michigan Medicine

Feb 7, 2019 | 09:00 am

Gene Therapy Treatment Targets Rare Mutation Tied to Blindness Michigan MedicineEye troubles detected at infancy began to take their toll when Emma Kate Roden started walking. The toddler would often bump into things because of poor ...

A biotech tries to relieve doubts on durability of gene therapy for Fabry disease - STAT

Feb 6, 2019 | 09:00 am

A biotech tries to relieve doubts on durability of gene therapy for Fabry disease STATAn experimental gene therapy developed by Avrobio reduced a type of fat that builds up to toxic levels in cells and organs of patients with Fabry disease.

Gene Therapy Preserves Nerve Fibers in Mouse Model of Severe Neurodegeneration - Parkinson's News Today

Feb 5, 2019 | 09:00 am

Gene Therapy Preserves Nerve Fibers in Mouse Model of Severe Neurodegeneration Parkinson's News TodayAn investigational gene therapy preserved nerve axons — long projections that connect nerve cells — in a mouse model of severe axonal degeneration.

Gene Therapy: Novartis Under The Microscope With Novel Treatment - Investor's Business Daily

Feb 1, 2019 | 09:00 am

Gene Therapy: Novartis Under The Microscope With Novel Treatment Investor's Business DailyBiotechs are watching Novartis' efforts for gene therapy Zolgensma, which would follow Spark's Luxturna. Will it face the same hurdles as cell therapy?

Gene therapy cassettes improved for muscular dystrophy - EurekAlert

Feb 1, 2019 | 09:00 am

Gene therapy cassettes improved for muscular dystrophy EurekAlertExperimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the ...

They said I'd go blind. Now gene therapy has changed that - The Guardian

Jan 19, 2019 | 09:00 am

They said I'd go blind. Now gene therapy has changed that The GuardianMatthew Bishop was told there was no treatment that could save his vision. But now scientific breakthroughs in gene therapy have given him, and others, hope.

Gene therapy promotes nerve regeneration - Science Daily

Jan 18, 2019 | 09:00 am

Gene therapy promotes nerve regeneration Science DailyResearchers have shown that treatment using gene therapy leads to a faster recovery after nerve damage. By combining a surgical repair procedure with gene ...

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