Breaking Gene Therapy News

Gene Therapy: Hate the wait? Consult Dr. Q. - Pittsburgh Post-Gazette

Aug 17, 2019 | 14:51 pm

Gene Therapy: Hate the wait? Consult Dr. Q. Pittsburgh Post-GazetteSo accepted are the Professor's bonafides on waiting in line that he has anointed himself Dr. Q. in the tradition of James Bond villainy,

Hacking Ourselves: The Promise of Gene Therapy - Interesting Engineering

Aug 17, 2019 | 08:36 am

Hacking Ourselves: The Promise of Gene Therapy Interesting EngineeringGene therapy is currently being used for developing strategies to treat a variety of genetic diseases. It has come far in recent years and is a fast becoming a ...

Will Long-Term Post-Marketing Studies Become the Norm for Gene Therapy Companies? - BioSpace

Aug 16, 2019 | 17:54 pm

Will Long-Term Post-Marketing Studies Become the Norm for Gene Therapy Companies? BioSpaceCurrent rules set by the FDA allow companies to assess whether or not long-term follow-ups are required based on risk factors determined by the company.

Gene Therapy Firm Renovacor Closes $11 Million Series A Financing - Clinical OMICs News

Aug 16, 2019 | 13:00 pm

Gene Therapy Firm Renovacor Closes $11 Million Series A Financing Clinical OMICs NewsRenovacor, a preclinical-stage gene therapy company, has announced the successful completion of an $11 million Series A financing co-led by Novartis ...

First CRISPR Law: Selling “Gene-therapy Kits” Will Be Illegal in California Unless They Carry a Warning - Technology Networks

Aug 16, 2019 | 10:57 am

First CRISPR Law: Selling “Gene-therapy Kits” Will Be Illegal in California Unless They Carry a Warning Technology NetworksFollowing unanimous support in the Legislature, the Governor Gavin Newsom signed the first bill into law addressing the emerging CRISPR technology.

Renovacor raises $11M to advance gene therapy for dilated cardiomyopathy - Cardiovascular Business

Aug 15, 2019 | 22:34 pm

Renovacor raises $11M to advance gene therapy for dilated cardiomyopathy Cardiovascular BusinessBiopharmaceutical company Renovacor, Inc., on August 14 announced it had raised $11 million in Series A financing to further develop its gene therapy-based ...

Leaders of AveXis Out Over Doctored Gene Therapy Data - The Scientist

Aug 14, 2019 | 22:50 pm

Leaders of AveXis Out Over Doctored Gene Therapy Data The ScientistBrian and Allan Kaspar, brothers at the scientific helm of Novartis's AveXis division, left the company in May, and sources tell both STAT and CNBC it's because ...

First clinical trial of drug-inducible gene therapy yields encouraging preliminary results - Medical Xpress

Aug 14, 2019 | 20:00 pm

First clinical trial of drug-inducible gene therapy yields encouraging preliminary results Medical XpressImmunotherapeutic drugs are a potent way of transforming the immune system into a ferocious guard dog that can sniff out and destroy tumor cells. But for some ...

AAV9 gene therapy vector dramatically increases life span in krabbe disease mouse model - EurekAlert

Aug 14, 2019 | 16:20 pm

AAV9 gene therapy vector dramatically increases life span in krabbe disease mouse model EurekAlertAn optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vec-tor used to deliver the galactosylceramidase gene to a mouse model of ...

Ebola success, Moon bears and gene-therapy delay - Nature.com

Aug 14, 2019 | 14:00 pm

Ebola success, Moon bears and gene-therapy delay Nature.comTwo Ebola drugs show promise in trial Two Ebola drugs have proved so effective in a clinical trial that researchers will make the treatments available to anyone ...

Biotech targeting rare heart mutation with gene therapy scores $11M in Series A haul - Endpoints News

Aug 14, 2019 | 13:11 pm

Biotech targeting rare heart mutation with gene therapy scores $11M in Series A haul Endpoints NewsIn some ways, cardiomyopathies appear to be more amenable to precision medicine than cancer — failing hearts are thought to be generally homogenous; ...

RTP-based AskBio expands gene therapy target list with acquisition of Scottish biotech - WRAL Tech Wire

Aug 13, 2019 | 12:09 pm

RTP-based AskBio expands gene therapy target list with acquisition of Scottish biotech WRAL Tech WireRESEARCH TRIANGLE PARK - Emboldened by a recent cash infusion of $225 million from investors, AskBio, a clinical stage gene therapy company in ...

Toddler denied $2.1m gene therapy will now get it for free - STAT

Aug 13, 2019 | 01:11 am

Toddler denied $2.1m gene therapy will now get it for free STATA 2 1/2-year-old girl deemed ineligible for the world's most expensive drug will get the $2.1 million medicine to treat her rare genetic disease after all.

Bio Roundup: Surprise News, a Gene Therapy Mess & a CAR-T Step - Xconomy

Aug 9, 2019 | 09:00 am

Bio Roundup: Surprise News, a Gene Therapy Mess & a CAR-T Step XconomyGene therapy has become one of the hottest fields in biomedicine, with two treatments approved in the US already and several more on the way. But surprise.

Medicare to Cover Breakthrough Cancer Gene Therapy - WebMD

Aug 8, 2019 | 09:00 am

Medicare to Cover Breakthrough Cancer Gene Therapy WebMDStudies have shown that CAR-T is effective against certain types of cancers, but can cause severe side effects.

Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA - The Scientist

Aug 7, 2019 | 09:00 am

Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA The ScientistTThe pharmaceutical company Novartis has disclosed to the US Food and Drug Administration that some testing data for its newly approved gene therapy were ...

FDA: Gene therapy maker submitted manipulated data before drug was approved - Washington Post

Aug 6, 2019 | 09:00 am

FDA: Gene therapy maker submitted manipulated data before drug was approved Washington PostThe FDA said Tuesday the maker of a newly approved $2 million gene therapy was aware that some of the testing data in its application to the agency was ...

Creative leadership needed for cell and gene therapy companies - STAT

Aug 2, 2019 | 09:00 am

Creative leadership needed for cell and gene therapy companies STATWhen it comes to leadership, cell and gene therapy companies are carving out unique paths. Creativity and diversity are essential for building teams.

Research team redefines the footprint of viral vector gene therapy - Science Daily

Jul 30, 2019 | 09:00 am

Research team redefines the footprint of viral vector gene therapy Science DailyBuilding on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, researchers ...

How do you toast a gene therapy trial? With tea.| NHGRI - National Human Genome Research Institute

Jul 23, 2019 | 09:00 am

How do you toast a gene therapy trial? With tea.| NHGRI National Human Genome Research InstituteIt's teatime and doctors, researchers and patients are at the table. They celebrate a decade of work and launch the first in-human gene therapy trial for children ...

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