Breaking Gene Therapy News

CRISPR gene therapy regulates hunger, staves off severe obesity in mice - Digital Trends

Dec 14, 2018 | 20:42 pm

CRISPR gene therapy regulates hunger, staves off severe obesity in mice Digital TrendsResearchers from University of California, San Francisco have demonstrated how CRISPR gene editing can be used to prevent severe obesity in mice.

Exclusive Q&A: AveXis President David Lennon Talks Gene Therapy In 2019 - Investor's Business Daily

Dec 14, 2018 | 14:01 pm

Exclusive Q&A: AveXis President David Lennon Talks Gene Therapy In 2019 Investor's Business DailyAveXis President David Lennon opens up about his forecasts for the gene therapy market in 2019 as the Novartis subsidiary works in spinal muscular atrophy.

Biogen pulls out of $1 billion partnership with AGTC after gene therapy trial failure - MedCity News

Dec 13, 2018 | 20:36 pm

Biogen pulls out of $1 billion partnership with AGTC after gene therapy trial failure MedCity NewsThe failure of a gene therapy trial has led to the termination of a three-year-old partnership between one of the largest biotechnology companies and the ...

Orchard Therapeutics to Build Out Gene Therapy Manufacturing Site in Fremont, CA - Genetic Engineering & Biotechnology News

Dec 13, 2018 | 11:05 am

Orchard Therapeutics to Build Out Gene Therapy Manufacturing Site in Fremont, CA Genetic Engineering & Biotechnology NewsOrchard Therapeutics said it will build out a gene therapy manufacturing facility in Fremont, CA, that will employ 100 people and expand its manufacturing ...

Dan and Jennifer Gilbert commit $12 million to pioneer gene therapy research - Crain's Detroit Business

Dec 12, 2018 | 18:22 pm

Dan and Jennifer Gilbert commit $12 million to pioneer gene therapy research Crain's Detroit BusinessQuicken Loans Inc. founder and Chairman Dan Gilbert and his wife, Jennifer, have pledged $12 million to fund pioneering gene therapy research aimed at ...

Gene therapy for Crigler-Najjar syndrome - EurekAlert

Dec 11, 2018 | 15:26 pm

Gene therapy for Crigler-Najjar syndrome EurekAlertGenethon announces dosing of the first patient with Crigler-Najjar Syndrome, a rare liver disorder, in the European phase I/II gene therapy clinical trial - CareCN.

Researchers refine gene therapy approach for hemophilia A - Healio

Dec 10, 2018 | 18:19 pm

Researchers refine gene therapy approach for hemophilia A HealioSAN DIEGO — Patients with severe hemophilia A achieved factor VIII activity levels sufficient to reduce or prevent spontaneous hemorrhage after a single IV ...

Gene Therapy: New Treatments Reprogram Patients' Immune Cell - SelectScience

Dec 10, 2018 | 17:24 pm

Gene Therapy: New Treatments Reprogram Patients' Immune Cell SelectScienceGene and cellular therapies are at the forefront of several recent clinical trials for many patient conditions including hematologic cancers, solid tumors and ...

Orchard's Gene Therapy for Rare Immune Disease Works for Over a Year - Labiotech.eu

Dec 7, 2018 | 17:27 pm

Orchard's Gene Therapy for Rare Immune Disease Works for Over a Year Labiotech.euA single dose of a gene therapy developed by Orchard Therapeutics can restore immune function for at least a year in patients with X-linked chronic ...

XyloCor closes $17M Series A round to develop gene therapy for refractory angina - MedCity News

Dec 6, 2018 | 09:00 am

XyloCor closes $17M Series A round to develop gene therapy for refractory angina MedCity NewsA company developing gene therapies for cardiovascular disease has secured its first major round of private financing. Philadelphia-based XyloCor ...

Sickle Cell Gene Therapy Makes Another Advance - WebMD

Dec 4, 2018 | 09:00 am

Sickle Cell Gene Therapy Makes Another Advance WebMDThe therapy targets the genetic flaw that causes sickle cell. In a small group of patients, researchers said the therapy appears safe and effective enough to keep ...

Bluebird's gene therapy improved outcomes for patients in clinical trial - STAT

Dec 3, 2018 | 09:00 am

Bluebird's gene therapy improved outcomes for patients in clinical trial STATBoth Bluebird and Global Blood Therapeutics are seeking to take advantage of flexibility at the FDA and a desire to see new treatments reach the market.

Novel Gene Therapy Promising for Sickle Cell Disease - Rare Disease Report

Dec 3, 2018 | 09:00 am

Novel Gene Therapy Promising for Sickle Cell Disease Rare Disease ReportThe therapy attempts to restore fetal hemoglobin production.

Misfortune strikes a Bluebird patient, highlighting risks of gene therapy - STAT

Dec 2, 2018 | 09:00 am

Misfortune strikes a Bluebird patient, highlighting risks of gene therapy STATSAN DIEGO — People get excited about gene therapy for the potential to cure some pretty horrible diseases, but the safety risks that patients take on with the ...

Autologous gene therapy shows promise for sickle cell disease - Healio

Dec 2, 2018 | 09:00 am

Autologous gene therapy shows promise for sickle cell disease HealioSAN DIEGO — An adult with sickle cell disease responded positively to autologous gene therapy, providing the first proof that this approach could be effective ...

Parkinson's Gene Therapy Wires New Brain Circuits - WebMD

Nov 28, 2018 | 09:00 am

Parkinson's Gene Therapy Wires New Brain Circuits WebMDIn a new study, gene therapy did not change the abnormal brain circuitry of Parkinson's disease. Instead, it essentially rewired a small area of the brain, ...

Gene therapy for blood disorders - Medical Xpress

Nov 27, 2018 | 09:00 am

Gene therapy for blood disorders Medical XpressGene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us.

First Gene Therapy for Inherited Blindness Approved in Europe - Labiotech.eu

Nov 26, 2018 | 09:00 am

First Gene Therapy for Inherited Blindness Approved in Europe Labiotech.euNovartis has received approval for Luxturna, a gene therapy that can restore vision in people with a specific genetic mutation that causes progressive vision loss.

Potential Gene Therapy for Hemophilia A, AMT-180, May Treat Patients Regardless of Inhibitor Status, uniQure Says - Hemophilia News Today

Nov 26, 2018 | 09:00 am

Potential Gene Therapy for Hemophilia A, AMT-180, May Treat Patients Regardless of Inhibitor Status, uniQure Says Hemophilia News TodayRead uniQure's update on its potential gene therapies, including AMT-180 in early testing for hemophilia A patients with or without factor VIII inhibitors.

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