Gene therapy Archives

In Depth 23 Nov 2017

The Future is (Syn)Bio: How and when do we get there?

Last week, Philip and I attended a meeting with some of the pioneers and leaders of the synthetic biology field. They believe biology will be everywhere in the future, but how do we get ready for it? Digital technologies are now present in almost all aspects of our lives. It seems obvious today that companies […]

November 23, 2017 - 5 minutesmins - By Clara Rodríguez Fernández

News and Trends 2 Nov 2017

Close to Launching Gene Therapy, Dutch Biotech Raises Over $91M on the Nasdaq

Update (2/11/2017): uniQure has closed its public offering raising over $91m (€78M) on the Nasdaq, taking advantage of a massive stock jump triggered by the announcement that an improved version of its gene therapy for hemophilia B will enter Phase III trials. Originally published on 24/10/2017 UniQure launched a public offering, perfectly timed with a rise in […]

November 2, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 19 Oct 2017

Dutch Biotech plans Pivotal Clinical Trial with First Gene Therapy for Hemophilia

uniQure is getting ready to start a Phase III clinical trial in 2018 with a new version of its gene therapy for hemophilia B. uniQure has officially launched a plan to bring the first gene therapy for hemophilia to the market. After multiple meetings with the FDA and the EMA, the Dutch biotech has received permission […]

October 19, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 11 Oct 2017

French Gene Therapy to Cure Retinitis Pigmentosa Enters the Clinic

Horama has been authorized to start a Phase I/II trial in France testing HORA-PDE6B, a one-off gene therapy with the potential to cure retinitis pigmentosa. Based in Paris, Horama develops gene therapies to treat genetic diseases affecting vision. The company will now start a Phase I/II clinical trial that will recruit 12 patients at the Nantes […]

October 11, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 6 Oct 2017

British-Finnish Alliance to Bring Gene Therapy to the Masses

FIT Biotech and Avacta hope to combine their technologies to produce highly specific, best-in-class gene therapy treatments. The two companies have spotted an opportunity to bring together their technologies and form a leading gene therapy system in a market that could be worth over $4.3B (€3.7B) by 2024. FIT Biotech, based in Finland, has been working on […]

October 6, 2017 - 2 minutesmins - By Alex Dale

News and Trends 28 Sep 2017

Oxford Spinout goes for $75M IPO on Nasdaq to Fund Gene Therapy Programs

Nightstar has gone public on the American market with the aim of raising $75M for its gene-based retinal disease cures. Spun out from Oxford in 2013, Nightstar is developing and commercializing one-shot gene therapies for various eye diseases that could lead to blindness. This morning, it debuted on the Nasdaq with a projected $75M (£56M / €64M) […]

September 28, 2017 - 2 minutesmins - By Evelyn Warner

News and Trends 27 Sep 2017

French Gene Therapy for Wilson’s Disease gets Orphan Status

Vivet Therapeutics has been granted Orphan Drug Designation for its gene therapy for Wilson’s disease, VTX-801, by both the FDA and the European Commission. The French biotech Vivet Therapeutics is developing what could eventually become the first gene therapy for Wilson’s disease. Seeing its potential, both the FDA and the EC have decided to grant […]

September 27, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Best in Biotech 26 Sep 2017

These Three Fields Are Proving Biotech is The Innovation Powerhouse in Healthcare

Biotech is the best source of innovation in R&D, making its companies the darlings of pharma. Which therapeutic fields belong to biotech? “Biotech is typically first in highly innovative fields,” Matt Kapusta, CEO of uniQure told us. Before he became CEO, uniQure brought the world’s first gene therapy to market — completely on its own. Biotech […]

September 26, 2017 - 8 minutesmins - By Evelyn Warner

News and Trends 13 Sep 2017

Italian Biotech Closes €7M Series B for Stem Cells to Treat Cancer

Genenta Science has raised $8M (€7M) in the Series B fundraising round to continue investigating its use of hematopoietic stem cells to treat cancer. Based in Milan, Genenta can add €7M to the €10M secured in Series A of fundraising. Italian, British and Swiss private investors led the investment, and it will allow the biotech to start a second […]

September 13, 2017 - 2 minutesmins - By Alex Dale

News and Trends 12 Sep 2017

French Startup Raises €11M in Series A to Reinvent DNA Synthesis

DNA Script has gathered the support of key international investors to take a new, faster DNA synthesis technology closer to the market. In a Series A round led by Illumina Ventures and joined by Merck Ventures and existing investors Sofinnova Partners, Kurma Partners, and Idinvest Partners, the young French startup DNA Script has raised a total […]

September 12, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 8 Sep 2017

Spanish Scientists Identify How Gene Activity in Bacteria is Controlled

A group in Barcelona has identified DNA close to genes that influence gene activity, which could be used in disease treatment and industrial biotechnology. Sections of DNA, termed ‘control dials‘, regulate transcription, and the amount of gene product produced. Reported in Nature Communications, these findings could help to control dangerous genes during a bacterial infection […]

September 8, 2017 - 2 minutesmins - By Alex Dale

News and Trends 7 Sep 2017

CAR-T is Nearly Here: How Will It Be Delivered?

As we anticipate the first-ever CAR-T therapy market approval we look at the important aspect of treatment delivery and viral vector strategies. Cell and gene therapy is increasingly coming into its own with some of the major pharma players releasing big headlines. GlaxoSmithKline (GSK) released the first ex vivo stem cell therapy in May 2016 […]

September 7, 2017 - 4 minutesmins - By External Contributor

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