Gene therapy Archives

News and Trends 3 May 2018

World’s First Gene Therapy Developer Raises $128M for Hemophilia and Huntington’s

uniQure has raised a substantial public offering on the NASDAQ. The funding could help the Dutch biotech advance two new gene therapies after the withdrawal of its first treatment. Amsterdam-based uniQure has raised an ambitious $128M (€107M) in a public offering at the beginning of next week. The Dutch biotech develops gene therapies for hemophilia and Huntington’s disease, for […]

May 3, 2018 - 2 minutesmins - By Alexander Burik

News and Trends 25 Apr 2018

Cancer Treatment Targeting DNA Repair is Tested in Humans for the First Time

Onxeo has initiated a Phase I trial testing its first-in-class cancer treatment that works by inhibiting the repair of tumor DNA. Onxeo, a French biotech, has dosed its first patient in a Phase I study testing its cancer treatment AsiDNA, the first treatment of its kind, for advanced solid tumors. The news follows the Phase […]

April 25, 2018 - 1 minutemin - By Alexander Burik

News and Trends 20 Apr 2018

Gene Therapy For Beta-Thalassemia Could Eliminate Need for Transfusions

Bluebird bio has obtained results from two clinical studies showing its new gene therapy could eliminate the need for red blood cell transfusions in patients with beta-thalassemia. The company plans to file for an EU approval of its treatment later this year. US firm bluebird bio has results from two clinical studies showing its LentiGlobin gene […]

April 20, 2018 - 2 minutesmins - By Alexander Burik

Interview 18 Apr 2018

What Lies Ahead for European Cell and Gene Therapies?

Cell and gene therapies have recently made big advances in providing effective treatments for a variety of conditions, from cancers to rare diseases. Nonetheless, there are still significant challenges ahead before the technologies can become a viable treatment option for most patients. What if you could treat a disease on the level of tissue and […]

April 18, 2018 - 5 minutesmins - By Alexander Burik

News and Trends 17 Apr 2018

How Low Can the Price of a Next Generation DNA Sequencer Go?

The next generation of DNA sequencers has arrived, and with it, a clash of titans: Illumina, Thermo Fisher and Qiagen are all competing for the clinical diagnostics market. Next-generation sequencing (NGS) companies are redefining DNA sequencing. What began as small-scale PCR and Sanger sequencing efforts have now mushroomed into a $20Bn market with broad medical implications for […]

April 17, 2018 - 5 minutesmins - By External Contributor

genesight gene therapy blindness placebo

News and Trends 4 Apr 2018

Placebo Beats a Gene Therapy for Blindness in a Phase 3 Trial

GenSight has demonstrated that its gene therapy for a rare disease causing blindness can significantly improve the vision of patients. But so does the placebo. GenSight Biologics has released the results of a Phase III clinical trial testing its gene therapy GS010 in patients suffering from Leber hereditary optic neuropathy (LHON), a rare mitochondrial disease […]

April 4, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

Interview 20 Mar 2018

Why It’s the Best Time to Be in Cell and Gene Therapy

Cell and gene therapies are here, and they’re here to stay. We stopped to chat about the future of the field with Alexander Vos last week at Bio Europe Spring. The cell and gene therapy space has been given a big boost in the last couple of years. Since 2016, GSK’s gene therapy Strimvelis is available […]

March 20, 2018 - 3 minutesmins - By Clara Rodríguez Fernández

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

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Gene therapy

World’s First Gene Therapy Developer Raises $128M for Hemophilia and Huntington’s

Cancer Treatment Targeting DNA Repair is Tested in Humans for the First Time

Gene Therapy For Beta-Thalassemia Could Eliminate Need for Transfusions

What Lies Ahead for European Cell and Gene Therapies?

How Low Can the Price of a Next Generation DNA Sequencer Go?

Placebo Beats a Gene Therapy for Blindness in a Phase 3 Trial

Why It’s the Best Time to Be in Cell and 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

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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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