News and Trends 13 Jul 2023 Gene therapy company EG 427 raises €18M EG 427, a French biotechnology company leading the development of pinpoint DNA medicine solutions, has announced the final closing of a Series A financing. The gene therapy company raised an additional €5 million ($5.6 million) in the final closing from a combination of existing investors and new family office investors, bringing the total raised in […] July 13, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 11 Jul 2023 NK:IO awarded £1.6M grant to develop NK cell therapy NK:IO, a U.K. company working on natural killer (NK) cell biology applied to the development of exceptionally potent, off-the-shelf cell therapies targeting solid tumors, has been awarded £1.6 million ($2.1 million) in grant funding from Innovate UK’s New Cancer Therapeutics program. The funding will support preclinical development, including development of a manufacturing process in collaboration […] July 11, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 30 Jun 2023 FDA approves BioMarin gene therapy for hemophilia A BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […] June 30, 2023 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 27 Jun 2023 Astraveus exits stealth to advance cell and gene therapy platform Astraveus SAS has emerged from stealth with the completion of an oversubscribed €16.5 million ($18.1 million) Series Seed financing. The company creates modular, microfluidic cell foundries that transform cell and gene therapy (CGT) manufacturing. Astraveus has developed the Lakhesys platform, an end-to-end cell foundry that uses deep process optimization and single-use, microfluidic bioprocessors to deliver […] June 27, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 23 Jun 2023 Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […] June 23, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 19 Jun 2023 Gene therapy: a promising solution for sickle cell disease Sickle cell disease is debilitating, and many people who suffer with it find that their lives are defined by pain. This means a curative treatment would be completely life–altering, and, as we observe World Sickle Cell Day on June 19, we explore how gene therapy could be the most promising solution for curing sickle cell […] June 19, 2023 - 6 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
Podcast 16 Jun 2023Beyond Biotech podcast 50 The International Society for Cell & Gene Therapy (ISCT), the global society of more than 3,000 members dedicated to the translation of cell and gene therapies (CGT) into safe and effective treatments to improve patient lives, recently hosted the largest translation-focused event connecting academic, regulatory and industry experts from around the world to collaborate and […] June 16, 2023 Share WhatsApp Twitter Linkedin Email
News and Trends 8 Jun 2023 Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM). There are a large number of genetically defined and complex muscle […] June 8, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 7 Jun 2023 AAVantgarde closes €61M financing to move gene delivery program to the clinic AAVantgarde Bio, a clinical-stage, Italian-based international biotechnology company with two proprietary adeno-associated viral (AAV) vector platforms for large gene delivery, has closed a €61 million ($65.1 million) Series A financing. The amount represents the largest series A round ever in Italian biotech. AAVantgarde has two proprietary, AAV-based large gene delivery platforms: one leveraging DNA recombination, […] June 7, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 1 Jun 2023 ElevateBio raises $401M for cell and gene therapies ElevateBio, LLC (ElevateBio), a technology-driven company focused on cell and gene therapies, has closed its $401 million Series D financing. “We have made significant strides in scaling our technologies and end-to-end capabilities in our pursuit to become the world’s most indispensable cell and gene therapy technology company. We are thrilled to welcome Khalil to our […] June 1, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 26 May 2023 Novartis buys AVROBIO cystinosis gene therapy program for $87.5M AVROBIO, Inc., a clinical-stage gene therapy company working to free people from a lifetime of genetic disease, has agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash. AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for […] May 26, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 26 May 2023 Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […] May 26, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email