Gene therapy Archives

Orchard Therapeutics Gene Therapy ADA SCID FDA

News and Trends 23 Aug 2017

Update: British Biotech’s Gene Therapy for Rare ‘Bubble Baby’ Disease set on UK Fast-Track

UPDATE (23/08/2017): The UK has also prioritized OTL-101 for review as a ‘Promising Innovative Medicine.’ This is the first step on the path to the Early Access to Medicine Scheme to bring it to market faster. Original Publication 24/07/2017 Orchard Therapeutics has received rare pediatric disease designation for OTL-101, a gene therapy for the treatment of […]

August 23, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

Interview 20 Jul 2017

Video – DNA Molecular Combing for the Early Detection of Genetic Diseases

It’s been more than a month since Refresh, but the topic of our last expert track is as current as ever: DNA analysis for the early detection of genetic diseases. Charles Caulliez, Director of Corporate Business Development at Genomic Vision, started his talk by introducing the company: In 2004, Aaron Bensimon (CEO) founded Genomic Vision as […]

July 20, 2017 - 3 minutesmins - By External Contributor

In Depth 11 Jul 2017

Regenerating the Body With Stem Cells – Hype or Hope?

When the Japanese researcher Shinya Yamanaka managed to reprogram adult cells into an embryonic-like state to yield induced pluripotent stem cells (iPSCs), this was supposed to herald a revolution in regenerative medicine. But 10 years after their discovery, a therapeutic breakthrough is still outstanding. “The overall stem cell therapy field has failed today to show […]

July 11, 2017 - 9 minutesmins - By Melanie De Almeida

News and Trends 30 Jun 2017

€40M in Series C for a British Biotech Treating Blindness with Gene Therapy

NightstaRx has raised €39.5M that will go towards three clinical programs testing gene therapies for rare diseases that cause blindness. NightstaRx is developing gene therapies for genetic retinal diseases that cause blindness, with technology from the University of Oxford. Now getting closer to the market, the company has closed a Series C round with $45M (€39.5M) […]

June 30, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

More News! 23 Jun 2017

French Biotech Raises Over €22M to Launch a Gene Therapy to Restore Sight

Gensight Biologics has announced a capital increase that totals €22.5M and will support the launch of its gene therapy for blindness in Europe and the US. Gensight has decided to give an additional financial push to its lead candidate, GS010, a gene therapy to restore sight in patients with the rare genetic disease Leber hereditary optic […]

June 23, 2017 - 1 minutemin - By Clara Rodríguez Fernández

News and Trends 22 Jun 2017

French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy

Noble Généthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease. Dedicated to rare diseases since 1990 and more recently to gene therapy, Généthon is one of very few not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is […]

June 22, 2017 - 3 minutesmins - By Evelyn Warner

vivet therapeutics gene therapy fundraising

News and Trends 4 May 2017

French Gene Therapy Biotech Raises a Huge €37.5M in Series A

Vivet Therapeutics has raised a huge €37.5M in Series A to help bring a new gene therapy technology for rare liver diseases to the clinic. Vivet Therapeutics has managed to attract some of the hottest biotech investors in Europe in its Series A. Led by Novartis Venture Fund and Columbus Venture Partners, and also counting […]

May 4, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 25 Apr 2017

EMA designates uniQure’s gene therapy for hemophilia a priority

Following a Breakthrough Designation from the FDA earlier this year, the Dutch biotech has scored the European equivalent for AMT-060. Since its claim to fame, Glybera, fell from grace to become a commercial failure, uniQure has been on the rebound. The company recently has undergone several management shakeups and a strategy review, and it just made the decision to withdraw […]

April 25, 2017 - 3 minutesmins - By Evelyn Warner

SIRION Biotech cell therapy lentivirus lentiboost

News and Trends 12 Apr 2017

Taking Cell Therapy one Step Further with this Boost Reagent

Cell therapy is revolutionizing medicine, here’s how the latest technology can help overcome the major challenges stopping it from taking over the market. Offering unprecedented possibilities to treat some of the most challenging diseases, cell therapy is stealing the show in the biotech space. Strimvelis, the first hematopoietic stem cell (HSC) gene therapy is already […]

April 12, 2017 - 4 minutesmins - By External Contributor

Eyevensis Eye Gene Therapy Approval France

News and Trends 11 Apr 2017

France Approves Trials for the First Eye Gene Therapy Free of Viral DNA

Eyevensys has received regulatory approval in France to start clinical trials using eye gene therapy technology that eliminates the need for viral vectors. The French biotech Eyevensys has been cleared by the French Product Security Regulatory Agency (ANSM) to proceed into the clinic with its EyeCET platform. The company is developing this gene therapy platform […]

April 11, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Interview 3 Apr 2017

Meet the Co-Founder who says DNA is the Next Silicon

DNA Script opens the door to a whole new age of manufacturing synthetic DNA. Thomas Ybert, was only 30 when he co-founded the company – here’s his story! Based in Paris, DNA Script produces synthetic DNA using a proprietary template-free enzymatic technology. Most of the manufactured DNA is currently synthesized by chemical means, but this startup […]

April 3, 2017 - 5 minutesmins - By External Contributor

Lysogene gene therapy Sanfillipo A obs study

News and Trends 29 Mar 2017

French Gene Therapy Biotech Gets Ready for Phase II/III

Lysogene has crunched the numbers on its observational study for Sanfillipo A – now the company can move into the final phase for its Sanfilippo A therapy. Lysogene is a French biotech specializing in gene therapy of rare CNS diseases. Its lead asset is a gene therapy for the treatment of the rare genetic disorder […]

March 29, 2017 - 3 minutesmins - By Melanie De Almeida

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Gene therapy

Update: British Biotech’s Gene Therapy for Rare ‘Bubble Baby’ Disease set on UK Fast-Track

Video – DNA Molecular Combing for the Early Detection of Genetic Diseases

Regenerating the Body With Stem Cells – Hype or Hope?

€40M in Series C for a British Biotech Treating Blindness with Gene Therapy

French Biotech Raises Over €22M to Launch a Gene Therapy to Restore Sight

French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy

French Gene Therapy Biotech Raises a Huge €37.5M in Series A

EMA designates uniQure’s gene therapy for hemophilia a priority

Taking Cell Therapy one Step Further with this Boost Reagent

France Approves Trials for the First Eye Gene Therapy Free of Viral DNA

Meet the Co-Founder who says DNA is the Next Silicon

French Gene Therapy Biotech Gets Ready for Phase II/III

Subscribe to our newsletter to get the latest biotech news!

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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