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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
Sponsored by Biovian 7 Jun 2022 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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email
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 Share WhatsApp Twitter Linkedin Email