Gene therapy Archives

News and Trends 26 Jul 2022

Oxford Biomedica sets up new project and signs LSA

Oxford Biomedica plc, a UK-based gene and cell therapy group, has initiated a new project with Orchard Therapeutics utilizing the company’s LentiStable technology. The group also announced the signing of a new licence and supply agreement (LSA) with an undisclosed US-based private biotech company advancing a new generation of adoptive cell therapies. LentiStable technology As […]

July 26, 2022 - 4 minutesmins - By Jim Cornall

News and Trends 20 Jul 2022

Upstaza gets nod from Europe for AADC deficiency treatment

PTC Therapeutics, Inc. says Upstaza (eladocagene exuparvovec) has been given marketing authorization by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency, and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older. “Today’s approval from the European […]

July 20, 2022 - 3 minutesmins - By Jim Cornall

More News! 18 Jul 2022

Purespring Therapeutics and SwanBio Therapeutics announce agreement for use of FunSel screening platform

A licensing agreement for use of the FunSel screening platform has been announced by Purespring Therapeutics and SwanBio. Under the terms of the agreement, SwanBio will license the FunSel screening platform from Purespring to explore new targets for early-stage pipeline programs. SwanBio will have the option to develop any targets identified and to own and […]

July 18, 2022 - 2 minutesmins - By Liza Laws

News and Trends 5 Jul 2022

EditForce and Mitsubishi Tanabi Pharma to work on gene therapy for CNS

EditForce, Inc. has entered into a license agreement with Mitsubishi Tanabe Pharma Corporation (MTPC) to research, develop and commercialize potential gene therapy products for a specific target disease related to the central nervous system (CNS) by utilizing EditForce’s pentatricopeptide repeat (PPR) protein platform technology. MTPC and EditForce aim to create potential novel pharmaceuticals for the […]

July 5, 2022 - 2 minutesmins - By Jim Cornall

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

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Gene therapy

Oxford Biomedica sets up new project and signs LSA

Upstaza gets nod from Europe for AADC deficiency treatment

Purespring Therapeutics and SwanBio Therapeutics announce agreement for use of FunSel screening platform

EditForce and Mitsubishi Tanabi Pharma to work on gene therapy for CNS

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

Subscribe to our newsletter to get the latest biotech news!

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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