Gene therapy Archives

News and Trends 27 Jun 2022

Trial for adults with late onset Pompe disease halted due to serious adverse event

The US Food and Drug Administration (FDA) has placed a clinical hold on a trial being run for Astrellas Pharma Inc after one of the participants had a serious adverse event (SAE) of peripheral sensory neuropathy. Astrellas made the announcement after the trial, which is evaluating AT845, an investigational adeno-associated virus (AAV) gene replacement in […]

June 27, 2022 - 3 minutesmins - By Liza Laws

News and Trends 23 Jun 2022

Krystal Biotech looks for FDA approval for dystrophic epidermolysis bullosa treatment

U.S. gene therapy company Krystal Biotech, Inc, has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of B-VEC (beremagene geperpavec) for the treatment of patients with dystrophic epidermolysis bullosa (DEB). B-VEC is an investigational non-invasive, topical gene therapy designed to treat DEB at the molecular level by […]

June 23, 2022 - 3 minutesmins - By Jim Cornall

conveyor belt of medical vials for scale up production

News and Trends 21 Jun 2022

Immuneel Therapeutics raises $15M and starts patient trials for CAR-T cancer therapies

Indian biotech start-up Immuneel Therapeutics has announced it has started patient dosing in a CAR-T trial named “IMAGINE.” The Indian company focuses on creating access to Chimeric Antigen Receptor T cell therapy (CAR-T) and other cellular immunotherapies for the management and treatment of cancers. The phase II trial is the first industry sponsored CAR-T trial […]

June 21, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 20 Jun 2022

Boost for dementia gene-based therapies

The Cell and Gene Therapy Catapult (CGT Catapult) and the UK Dementia Research Institute (UK DRI) have announced a collaboration to accelerate the clinical development of adeno-associated virus (AAV) based gene therapies for dementia. Since 2015, dementia has been one of the leading causes of mortality in the UK. While new and improved treatments for […]

June 20, 2022 - 3 minutesmins - By Jim Cornall

Gene therapy: can CDMOs solve the manufacturing bottleneck?

The gene therapy market is booming. Viral vectors, the key delivery systems for gene therapies, are playing an increasing role in the development of biologics. But the growing need for viral vectors and their plasmid building blocks have resulted in a manufacturing bottleneck. Can contract manufacturing organizations cope with the rising demand? In 2019, Zolgensma, a […]

June 7, 2022 - 7 minutesmins - By Ute Boronowsky

News and Trends 31 May 2022

Therapeutic RNA company receives additional funding

UK biotech company Transine Therapeutics, which is developing a novel class of therapeutic RNAs, has successfully raised £4.6M ($5.8M) in additional seed funding. The financing was led by new investor Epidarex Capital with participation from existing investor the Dementia Discovery Fund (DDF). Transine said the additional funds will enable it to further develop its SINEUP […]

May 31, 2022 - 2 minutesmins - By Jim Cornall

In Depth 4 Apr 2022

Portable Sequencing Is Reshaping Genetics Research

Portable sequencing is making it possible for biologists to perform DNA analysis anywhere in the world. How is this technology reshaping the way they work? Thanks to nanopore technology, scientists can now collect samples and sequence them anywhere. It is the concept of backpacking applied to scientific research. French molecular biologist Anne-Lise Ducluzeau has experienced […]

April 4, 2022 - 11 minutesmins - By Carlos de Rojas

gene therapy bacteria ilya pharma lactic acid

News and Trends 23 Mar 2022

Ilya Pharma Paves Way for Bacterial Gene Therapy with €8.5M Funding

Cell and gene therapies are taking off in a big way in Europe. As the buzz grows, the Swedish firm Ilya Pharma has raised €8.5M to lay down the foundations of bacterial gene therapies for enterocolitis and diabetic ulcers. Investor interest in cell and gene therapy is ramping up fast around the world, with a […]

March 23, 2022 - 4 minutesmins - By Jonathan Smith

News and Trends 16 Feb 2022

Startup Bags €50M Series A to Smash Gene Therapy Vector Limits

After closing Spain’s largest biopharma Series A round, the company SpliceBio has set its foundations in the gene therapy field and joins the push to deliver bigger therapeutic genes into patients. SpliceBio was founded in 2012 to tinker with a type of protein called inteins, which can stick peptides together to form new proteins. The […]

February 16, 2022 - 5 minutesmins - By Jonathan Smith

Gene therapy genenta science genflow biosciences

News and Trends 21 Jan 2022

Two Gene Therapy Players Launch IPOs on Stormy Stock Market

After a drought of more than a year, the European gene therapy sector saw initial public offerings from Genenta Science and Genflow Biosciences. The firms aim to shine as biotech stocks elsewhere perform poorly. Until recently, European gene therapy companies were going through a dry spell in terms of launching initial public offerings (IPO). The […]

January 21, 2022 - 4 minutesmins - By Jonathan Smith

News and Trends 14 Jan 2022

Europe Trailed US Despite Record Gene and Cell Therapy Funding in 2021

Cell and gene therapy developers globally raised an all-time annual record in 2021. However, European firms missed out on the funding growth. Companies around the world developing cell and gene therapies raised €20.1B ($23.1B) over 2021, said the advanced therapy advocacy organization the Alliance for Regenerative Medicine (ARM) in a briefing this week. This bumper […]

January 14, 2022 - 6 minutesmins - By Jonathan Smith

Best in Biotech 22 Dec 2021

The Biggest European Biotech News of 2021

In spite of the challenges posed by the Covid-19 pandemic, the European biotech industry has emerged stronger than ever this year. These were the key moments for the sector in 2021. The year 2021 has seen skyrocketing fortunes for many European biotech players. Investment firms, for instance, raised far more funding than in 2020, which […]

December 22, 2021 - 10 minutesmins - By Jonathan Smith

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Gene therapy

Trial for adults with late onset Pompe disease halted due to serious adverse event

Krystal Biotech looks for FDA approval for dystrophic epidermolysis bullosa treatment

Immuneel Therapeutics raises $15M and starts patient trials for CAR-T cancer therapies

Boost for dementia gene-based therapies

Gene therapy: can CDMOs solve the manufacturing bottleneck?

Therapeutic RNA company receives additional funding

Portable Sequencing Is Reshaping Genetics Research

Ilya Pharma Paves Way for Bacterial Gene Therapy with €8.5M Funding

Startup Bags €50M Series A to Smash Gene Therapy Vector Limits

Two Gene Therapy Players Launch IPOs on Stormy Stock Market

Europe Trailed US Despite Record Gene and Cell Therapy Funding in 2021

The Biggest European Biotech News of 2021

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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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