Gene Therapy Clinical Articles & Analysis
38 news found
The proceeds will enable ViGeneron to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy programs. ...
Efficient transfer of larger gene via ViGeneron’s proprietary technology platforms REVeRT and vgAAV ViGeneron advances its preclinical programs for Stargardt disease and Retinitis Pigmentosa into IND-enabling activities and clinical stage development ViGeneron GmbH, a next-generation gene therapy company, today ...
Gene expression and therapy involves the introduction of foreign nucleic acids into target cells to modify or compensate for gene defects, thereby treating diseases such as cancer, diabetes, autoimmune diseases, and infectious diseases. ...
Eikonoklastes Therapeutics, a preclinical biotech company developing treatments for today’s most challenging diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Eikonoklastes’ AAV-based gene therapy, ...
In this webinar, I look forward to exploring current and new strategies for optimising the upstream AAV vector production process, a vital first step towards advancing gene therapies to meet clinical and commercial yields.” Dr. Jean-Simon Diallo, CEO of Virica Biotech. Please join experts Amine Kamen, Ph.D., McGill University; Sarah ...
PT, in Carlsbad, CA Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that Chief Medical Officer Maria Escolar, M.D, MS., will present updated clinical data from the RESKUE Phase 1/2 clinical trial for FBX-101—the Company’s novel gene ...
Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced further collaboration for their manufacturing partnership to include ...
Myrtelle Inc., (Myrtelle), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance ...
Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that company leadership members will be making presentations and taking one-on-one meetings at three upcoming investor relations conferences. ...
Enables gene therapy clients to accelerate AAV manufacturing with seamless incorporation into the Company’s HEK 293 platform suspension process with access to end-to-end capabilities New offerings of Research-Grade and GMP-Pathway expedite Phase 1/2 clinical trial timelines, with GMP-Grade available in 2023 Data to support ...
“We continue our ambitious mission to enable access to life changing gene therapies by fulfilling a key goal of bringing them from concept to reality,” said Timothy J. ...
About Forge Biologics Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP ...
FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium FBX-101 is a systemic AAV gene therapy following unrelated cord blood transplantation (“UCBT”) and has been well tolerated through Day 180 ...
Food and Drug Administration (FDA) to launch the first-in-human clinical trial of ASC618. The study (NCT04676048) is expected to assess the one-time gene therapy’s safety and preliminary effectiveness in up to 12 adults with moderate to severe hemophilia A. ...
(NYSE: CRL) and ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in vivo gene replacement, gene editing and allogeneic cell therapies, today announced they have agreed to manufacture ASC618, a second-generation gene therapy for hemophilia A. ...
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease. The results, ...
Genenta Science (NASDAQ: GNTA), a clinical-stage biotechnology company pioneering the development of hematopoietic stem progenitor cell immuno-gene therapy for cancer, will present clinical data from its Phase 1/2a dose-escalation study of its clinical-stage candidate Temferon™ in patients with glioblastoma ...
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported new interim data, including on new visual motor integration, motor coordination and visual perception measures, from a collaborator-sponsored, ongoing Phase 1/2 gene ...
We are thrilled to bring a second-generation gene therapy treatment to patients with Hemophilia A.” The Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) is the world’s largest gathering of gene and cell therapy professionals and provides an international forum ...
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2022 and provided a business update. ...