Gene therapy Archives

bluebird bio gene therapy beta thalassemia

News and Trends 4 Jun 2019

First Gene Therapy for Beta Thalassemia Approved in Europe

The US biotech company bluebird bio has obtained EU conditional market approval for a gene therapy that could remove the need for blood transfusions for people with the blood disorder beta-thalassemia. Called Zynteglo, the gene therapy is aimed at treating people with beta-thalassemia who are over 12 years of age and have any but the […]

June 4, 2019 - 3 minutesmins - By Jonathan Smith

gensight gene therapy lhon genetic blindness

News and Trends 29 May 2019

After Unexpected Setback, GenSight Seeks Approval for Blindness Gene Therapy

The French biotech GenSight will apply for regulatory approval for its gene therapy despite the treatment unexpectedly resulting in the improvement of the eyes treated with a placebo in a phase III trial. GenSight is developing a gene therapy designed to treat progressive vision loss caused by the rare genetic condition Leber hereditary optic neuropathy. […]

May 29, 2019 - 2 minutesmins - By Jonathan Smith

News and Trends 29 May 2019

€60M Investment to Boost Oxford Biomedica’s Gene and Cell Therapies

The Danish investor Novo Holdings is handing Oxford Biomedica up to €60.6M (£53.5M) to develop gene and cell therapies treating cancer and genetic blindness. The investment will bag Novo Holdings a stake of up to 10.1% in the UK biotech. Oxford Biomedica will use the money to repay a loan totalling €49M ($55M) from the […]

May 29, 2019 - 2 minutesmins - By Jonathan Smith

News and Trends 14 May 2019

First Potential Gene Therapy for Hemophilia B Restores Blood Clotting in Phase II

A gene therapy developed by the Dutch biotech uniQure increased the activity of a blood clotting protein enough to stop bleeds in patients with the blood disorder hemophilia B. Hemophilia B is a rare genetic condition in which patients have a mutation in the blood clotting protein factor IX (FIX), reducing its activity below 6% […]

May 14, 2019 - 3 minutesmins - By Jonathan Smith

News and Trends 30 Apr 2019

Orchard’s Gene Therapy Shows Potential in Treating Patients with Beta Thalassemia

Patients with the life-threatening blood disorder beta-thalassemia required fewer blood transfusions after receiving a gene therapy developed by the UK company Orchard Therapeutics, with four out of nine no longer depending on transfusions. Beta thalassemia is a genetic disorder that affects hemoglobin, a protein that blood cells use to carry oxygen. In the most severe […]

April 30, 2019 - 3 minutesmins - By Jonathan Smith

Interview 25 Apr 2019

European Biotechnology in 2019 and Beyond: What’s Hot and What’s Not?

We spend a lot of time focusing on specific developments in biotechnology, but sometimes it’s good to take a step back and look at the bigger picture. With this in mind, I asked three experts at our recent Refresh event in Vienna to share what is hot in European biotech right now and what trends […]

April 25, 2019 - 11 minutesmins - By Helen Albert

Interview 20 Mar 2019

Unlocking the Potential of Fast DNA Synthesis

While the price and speed of reading DNA have dropped massively, writing DNA fast and affordably still remains a challenge. Philip talked to Sylvain Gariel, COO of the French company DNA Script, who strives to reinvent DNA synthesis. DNA has become a basic necessity in any biology lab. Thanks to new technologies we can now […]

March 20, 2019 - 4 minutesmins - By Clara Rodríguez Fernández

microsoft synthace oxford biomedica gene therapy cell therapy

News and Trends 14 Mar 2019

Microsoft Aims to Make Cell Therapies Cheaper with Artificial Intelligence

Microsoft has launched a collaboration with the UK company Oxford BioMedica to use Microsoft’s artificial intelligence technology to make gene and cell therapy manufacturing easier and cheaper. Genetically programming cells and viruses to make cell and gene therapies is not as simple as programming a computer. Organisms have many layers of complexity and unpredictability, sometimes […]

March 14, 2019 - 2 minutesmins - By Jonathan Smith

biogen nightstar therapeutics gene therapy ophthalmology

News and Trends 4 Mar 2019

Biogen Acquires UK Biotech Developing Gene Therapy for Blindness

The big US biotech Biogen has agreed to acquire Nightstar Therapeutics for €773M ($877M), including Nightstar’s candidate gene therapies for inherited blindness. Expected to be complete by mid-2019, Biogen’s acquisition of Nightstar bags it the UK company’s most advanced program. Currently in phase III, the therapy targets choroidemia, an incurable genetic condition which causes progressive […]

March 4, 2019 - 2 minutesmins - By Jonathan Smith

Best in Biotech 28 Jan 2019

5 Real-Life Technologies Where Biotech Meets Science Fiction

From genetic engineering to synthetic food and immortality, biotech is getting surprisingly close to recreating science fiction in real life. Science fiction is always a step ahead of real-life science. The genre imagined space travel, wireless communication and virtual reality years before they became a reality. In fact, sci-fi has coined some scientific terms […]

January 28, 2019 - 6 minutesmins - By Clara Rodríguez Fernández

amryt skin gene therapy epidermolysis bullosa

News and Trends 8 Jan 2019

Topical Gene Therapy Shows Early Promise for Inherited Skin Disease

A gene therapy applied to the skin, developed by UK company Amryt Pharma, showed positive preclinical results for the treatment of a rare genetic skin disease. The disease, called recessive dystrophic epidermolysis bullosa (RDEB), is one of a group of diseases in which sufferers have mutations in proteins vital to the integrity of the […]

January 8, 2019 - 3 minutesmins - By Jonathan Smith

Best in Biotech 24 Dec 2018

Looking Back on 2018: Our 10 Most Popular Interviews

As 2018 draws to a close and our thoughts are torn between holiday plans and what the new year may bring, we decided to look back and revisit the people and the stories that attracted our readers the most during the year. These were well received the first time around, so we thought you might […]

December 24, 2018 - 8 minutesmins - By Helen Albert

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Gene therapy

First Gene Therapy for Beta Thalassemia Approved in Europe

After Unexpected Setback, GenSight Seeks Approval for Blindness Gene Therapy

€60M Investment to Boost Oxford Biomedica’s Gene and Cell Therapies

First Potential Gene Therapy for Hemophilia B Restores Blood Clotting in Phase II

Orchard’s Gene Therapy Shows Potential in Treating Patients with Beta Thalassemia

European Biotechnology in 2019 and Beyond: What’s Hot and What’s Not?

Unlocking the Potential of Fast DNA Synthesis

Microsoft Aims to Make Cell Therapies Cheaper with Artificial Intelligence

Biogen Acquires UK Biotech Developing Gene Therapy for Blindness

5 Real-Life Technologies Where Biotech Meets Science Fiction

Topical Gene Therapy Shows Early Promise for Inherited Skin Disease

Looking Back on 2018: Our 10 Most Popular Interviews

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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