Gene therapy Archives

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

Altheia Science gene therapy autoimmune disease

News and Trends 19 Dec 2018

Italian Biotech Raises Big Series A for ‘Gene Therapy 2.0’

Altheia Science, based in Milan, has raised €17M to fund the development of gene therapies for autoimmune diseases with the remarkable potential to be curative. “The success of our fundraising campaign is unprecedented over a Series A in Italy,” said Paolo Rizzardi, co-founder and CEO of Altheia. The funds will go towards taking the company’s […]

December 19, 2018 - 3 minutesmins - By Clara Rodríguez Fernández

In Depth 11 Dec 2018

Easy as ATCG: Commercialization of DNA as Data Storage

You could be storing information in DNA sooner than you might think. Although the technology is still quite early — the first video encoded in DNA, revealed last year, was quite simple — the rate at which the cost of writing DNA is going down might make DNA data storage a reality in the coming […]

December 11, 2018 - 5 minutesmins - By External Contributor

News and Trends 7 Dec 2018

Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year

A 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 granulomatous disease (X-CGD). The gene therapy was trialed in patients between the ages of 2 and 27 with the rare disease, which is caused by a rare genetic mutation that […]

December 7, 2018 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 6 Dec 2018

Update: Liver Gene Therapy 8 Times Stronger Than Usual

Update (06/12/2018): uniQure has incorporated Synpromics’ strong liver promoter into a preclinical gene therapy candidate for treating hemophilia A. Published on 08/10/2018 Synpromics and uniQure have unveiled new technology for precise genetic control that could make gene therapy safer and more precise. The team has developed a gene therapy system that is highly selective for […]

December 6, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

uniQure Freeline aruvant blood disorders

News and Trends 4 Dec 2018

Gene Therapies for Blood Disorders Marching Through the Clinic

Gene therapy holds great potential as a cure for blood disorders such as hemophilia and sickle cell disease, which have limited treatment options. This week, several gene therapy companies have released promising clinical trial results in hemophilia and sickle cell anemia. Presenting at the 2018 annual meeting of the American Society of Hematology, Dutch company uniQure […]

December 4, 2018 - 3 minutesmins - By Jonathan Smith

News and Trends 26 Nov 2018

First Gene Therapy for Inherited Blindness Approved in Europe

Novartis has received approval for Luxturna, a gene therapy that can restore vision in people with a specific genetic mutation that causes progressive vision loss. The therapy is indicated for people with mutations in the two copies of a gene, called RPE65, that cause retinal cells to die over time, resulting in progressive loss of […]

November 26, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

Orchard therapeutics gene therapy header

News and Trends 1 Nov 2018

Gene Therapy Company’s IPO Shoots it to Billion-Euro Biotech List

Orchard Therapeutics, a British company developing gene therapy for rare genetic diseases, has launched its Nasdaq IPO and is now reportedly worth $1.25B (€1B). This IPO propels Orchard to the handful of European biotechs worth €1B or more. The new money might help with the marketing and manufacture of Strimvelis, an EMA-approved gene therapy for […]

November 1, 2018 - 2 minutesmins - By Jonathan Smith

axovant parkinsons disease neuron dopamine

News and Trends 25 Oct 2018

Parkinson’s Disease Gene Therapy Clinical Trial Launched in UK

A gene therapy for Parkinson’s disease developed by Swiss biotech Axovant has been used on the first patient in a Phase I/II trial at University College London. Parkinson’s disease is a neurodegenerative disease, caused by a lack of the neurochemical dopamine in regions of the brain handling motor functions causing debilitating symptoms such as rigidity […]

October 25, 2018 - 2 minutesmins - By Jonathan Smith

Interview 23 Oct 2018

Bridging the Academia-Industry Divide: Lessons from a Serial Biotech Entrepreneur

What does it take to be a successful biotech entrepreneur? I put this and other similar questions to Dinko Valerio, a highly experienced Dutch geneticist turned serial entrepreneur and investor. While there are many great scientists, it takes a certain something to be able to cross over into industry and build a profitable business. Dinko […]

October 23, 2018 - 8 minutesmins - By Helen Albert

News and Trends 24 Sep 2018

Gene Therapy Improves Eyesight of Patients With Inherited Blindness

UK startup Nightstar Therapeutics has reported good safety and early signs of visual improvements in a Phase I/II trial with a gene therapy for a rare form of inherited blindness. One month after being treated with Nightstar’s gene therapy, five of the 18 patients enrolled in the trial have already shown improvements in their vision. […]

September 24, 2018 - 2 minutesmins - By Jonathan Smith

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

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Gene therapy

Topical Gene Therapy Shows Early Promise for Inherited Skin Disease

Looking Back on 2018: Our 10 Most Popular Interviews

Italian Biotech Raises Big Series A for ‘Gene Therapy 2.0’

Easy as ATCG: Commercialization of DNA as Data Storage

Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year

Update: Liver Gene Therapy 8 Times Stronger Than Usual

Gene Therapies for Blood Disorders Marching Through the Clinic

First Gene Therapy for Inherited Blindness Approved in Europe

Gene Therapy Company’s IPO Shoots it to Billion-Euro Biotech List

Parkinson’s Disease Gene Therapy Clinical Trial Launched in UK

Bridging the Academia-Industry Divide: Lessons from a Serial Biotech Entrepreneur

Gene Therapy Improves Eyesight of Patients With Inherited Blindness

Subscribe to our newsletter to get the latest biotech news!

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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