Gene therapy Archives

News and Trends 6 Mar 2018

Oxford Spin-out Moves Gene Therapy For Blindness Into Phase III Trial

Nightstar Therapeutics will begin the first Phase III trial for a gene therapy targeting choroideremia, a rare disorder leading to complete blindness. Nightstar will launch the first-ever Phase III trial testing a gene therapy for choroideremia, a genetic disorder characterized by night blindness and gradual loss of vision that eventually leads to complete blindness. The trial, STAR, […]

March 6, 2018 - 2 minutesmins - By Alexander Burik

News and Trends 20 Feb 2018

This Gene Therapy for Vision Loss is Safe and Effective After Two Years

The results of a long-term follow-up suggest that GenSight’s gene therapy for Leber hereditary optic neuropathy can provide safe and effective treatment for the genetic cause of vision loss. GenSight develops gene therapies to preserve or restore vision in patients with neurodegenerative diseases of the retina. The biotech’s lead candidate is GS010, which is currently […]

February 20, 2018 - 3 minutesmins - By Alex Dale

News and Trends 31 Jan 2018

Gene Synthesizing Biotech Gets $12M to Boost the Synbio Field

Evonetix has closed a Series A fundraising round to support the development of its gene synthesis platform, an important tool in the emerging synbio field. Evonetix, based in Cambridge, UK, is developing a scalable, high fidelity gene synthesis approach that could be a key tool in the growing synbio field. The biotech has closed a […]

January 31, 2018 - 3 minutesmins - By Alex Dale

In Depth 31 Jan 2018

Why Does Europe Lag Behind the US and China in the Gene Editing Race?

America and China lead the development of new gene editing technologies, producing GMOs and bringing CRISPR-based therapies into the clinic. Could this render European biotechs in this area obsolete? Gene editing technologies have revolutionized genetics and mark a new generation in biomedical and agricultural sciences. There are endless applications for the technology, including gene therapies for […]

January 31, 2018 - 6 minutesmins - By Ekaterina Perets

News and Trends 30 Jan 2018

Update: Pocket Device makes its Debut in Whole Human Genome Sequencing

Update (30/01/2018): Oxford Nanopore has published a study in Nature Biotechnology outlining the first use of its pocket MinION device to sequence a human genome. The device was able to read sequences of DNA that are “hundreds of times larger than usual,” helping scientists better reconstruct the whole genome sequence. MinION costs less than $1,000 and […]

January 30, 2018 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 29 Jan 2018

This Italian Biotech Will Sequence 10,000 Genomes in 2 Years

Dante Labs will use its DNA testing kit to sequence the DNA of 10,000 Europeans in an effort to improve our understanding of the human genome. Dante Labs developed the first DNA test to be made available in Europe, allowing the public to test their health from the comfort of their own homes. It has now […]

January 29, 2018 - 2 minutesmins - By Alex Dale

News and Trends 25 Jan 2018

Novartis Will Bring US Gene Therapy for Blindness to Europe

The Swiss pharma has bought into the gene editing therapy game in a deal with Spark worth $170M (€137M) for commercialization rights outside the US. Novartis first marked out its position as a gene therapy leader in 2012 when it threw its hat into the CAR-T ring. Just a few months after the FDA approved […]

January 25, 2018 - 2 minutesmins - By Evelyn Warner

gensight retinitis pigmentosa gene therapy

News and Trends 10 Jan 2018

French Biotech Starts Trials to Restore Sight in All Types of Retinitis Pigmentosa

GenSight will start a clinical trial in the UK testing a combination of gene therapy and a wearable device to restore sight in patients with retinitis pigmentosa. The UK Medicines and Healthcare Regulatory Agency (MHRA) has accepted GenSight’s application to run a first clinical trial in the UK testing a new type of therapy with the […]

January 10, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 20 Dec 2017

British Gene Therapy Gets a Huge £85M Boost in Series B

Orchard Therapeutics has raised a big round of financing to gene therapy options for many rare diseases for which there are no treatments. London-based Orchard Therapeutics has managed to raise £85M (€96M) in Series B, an amount that will certainly give an important boost to its pipeline of gene therapy treatments for rare diseases. The […]

December 20, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 19 Dec 2017

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

UniQure has published clinical data revealing that its gene therapy for hemophilia B is effective for years and lets patients stop weekly replacement therapy. From its headquarters in Amsterdam, uniQure is developing what could be one of the first gene therapies for hemophilia. A publication in the journal Blood yesterday details the results of a […]

December 19, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

granulomatous disease orchard therapeutics

News and Trends 18 Dec 2017

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

Orchard Therapeutics has licensed a gene therapy for the rare genetic disorder X-linked chronic granulomatous disease (X-CGD) from the non-profit Généthon. London-based Orchard Therapeutics is adding a new program to its pipeline of gene therapies. The company has obtained an exclusive license to a lentiviral vector to treat X-linked chronic granulomatous disease and the clinical trial data […]

December 18, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

CRISPR Therapeutics clinical trials CRISPR Cas9

News and Trends 13 Dec 2017

CRISPR Therapeutics Plans First CRISPR/Cas9 Clinical Trial in Europe for 2018

CRISPR Therapeutics and Vertex Pharmaceuticals are taking action to start a first clinical trial with CRISPR/Cas9 in Europe in 2018. The blood disorder β-thalassemia could soon be the first human disease to be treated using CRISPR/Cas9 technology in Europe. CRISPR Therapeutics and its partner Vertex Pharmaceuticals have announced the first candidate from their collaboration that will go […]

December 13, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

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Gene therapy

Oxford Spin-out Moves Gene Therapy For Blindness Into Phase III Trial

This Gene Therapy for Vision Loss is Safe and Effective After Two Years

Gene Synthesizing Biotech Gets $12M to Boost the Synbio Field

Why Does Europe Lag Behind the US and China in the Gene Editing Race?

Update: Pocket Device makes its Debut in Whole Human Genome Sequencing

This Italian Biotech Will Sequence 10,000 Genomes in 2 Years

Novartis Will Bring US Gene Therapy for Blindness to Europe

French Biotech Starts Trials to Restore Sight in All Types of Retinitis Pigmentosa

British Gene Therapy Gets a Huge £85M Boost in Series B

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease

CRISPR Therapeutics Plans First CRISPR/Cas9 Clinical Trial in Europe for 2018

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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

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Advertise with us

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