ASC618

ASC Therapeutics Receives Key Regulatory Designations in U.S. and Europe to Advance its Second-Generation Gene Therapy for Hemophilia A

Retrieved on: 
Wednesday, March 30, 2022
Research, FDA, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Therapy, Fast Track, VIII, Maple syrup urine disease, Clinical trial, COMP, Allotransplantation, ASC, European Medicines Agency, IND, University, Doctor of Philosophy, Program, Food and Drug Administration, Unfolded protein response, Food, Immunotherapy, MD, Protein folding, Gene editing, Safety, Committee, DOI, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Haemophilia, EMA, Lists of diseases, FDA, HC, Patient, Pharmaceutical industry, Vaccine, Medical device, ASC618, ASC THERAPEUTICS

Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, We are very pleased with the FDA's and EMAs regulatory decisions regarding ASC618.

Key Points: 
  • Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, We are very pleased with the FDA's and EMAs regulatory decisions regarding ASC618.
  • Oscar Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams.
  • ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males1.
  • Hemophilia A gene therapy: current and next-generation approaches, Expert Opinion on Biological Therapy.

ASC Therapeutics and Professor Steven W. Pipe Publish Expert Review on Novel Gene Therapies of Hemophilia A

Retrieved on: 
Tuesday, January 11, 2022
Health, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Patient, Pathology, HC, Haemophilia, University, Haemophilia A, Liver, Clinical trial, FRG, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, US, Degenerative disease, Unfolded protein response, Allotransplantation, Safety, Pediatrics, Program, ASC, DOI, Immunotherapy, Learning, IND, DNA, Review, Advancing Human Rights, Lists of diseases, Factor VIII, Half-life, Therapy, Gene editing, Ministry of Education (Malaysia), Doctor of Philosophy, Multimedia, Coagulopathy, Protein folding, Food, Treatment, Maple syrup urine disease, FDA, VIII, Principal, Orphan, Biology, Medical device, Vaccine, Pharmaceutical industry, MD, Hemophilia A, ASC618, ASC Therapeutics, HEMOPHILIA A, ASC618, ASC THERAPEUTICS

This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:

Key Points: 
  • This publication provides an update of scientific and clinical advances in gene replacement therapies for hemophilia A with focus on:
    This press release features multimedia.
  • Oscar G. Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added We are thrilled that the journal Expert Opinion in Biological Therapyhas published this seminal review to inform the medical and scientific community of these exciting developments with gene therapies in hemophilia A.
  • The major challenges of current treatment regimens, such the short half-life of hemophilia therapeutics with need for frequent intravenous injections, justify ongoing focus on gene replacement therapies.
  • ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males.

ASC Therapeutics Appoints Gene and Cell Therapy Manufacturing Veteran Gary Potter to lead Global Operations

Retrieved on: 
Tuesday, September 28, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Unfolded protein response, GMP, Food, ASC, Therapy, Clinical trial, Gene editing, Orphan, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, CGMP, Haemophilia, CMC, Genesys, GLP, FDA, Patient, Engineering, Hematopoietic stem cell transplantation, Global Operations, Senior, Hamline University, Drug, Program, Motivation, Placenta, Allotransplantation, Technology, IND, Amgen, Automation, Genetics, Safety, R, Technology transfer, Industry, Disorder, Fine chemical, Vaccine, Pharmaceutical industry, ASC618, ASC930, ASC Therapeutics, ASC618, ASC930, ASC THERAPEUTICS

ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic and other rare disorders announces the appointment of Gary Potter as Senior Vice President, Global Operations.

Key Points: 
  • ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic and other rare disorders announces the appointment of Gary Potter as Senior Vice President, Global Operations.
  • Gary Potter will lead the global teams responsible for drug substance and drug product process development, manufacturing, and supply chain for gene and cell therapies required for all phases of clinical development and commercialization for all ASC Therapeutics programs.
  • Ruhong Jiang, ASC Therapeutics CEO said I am extraordinarily confident that Gary Potter brings the right expertise, leadership skills and motivation for our global operations, focusing initially on drug product readiness for our clinical programs for ASC618 second-generation gene therapy and ASC930 allogeneic cell therapy.
  • Gary Potter, ASC Therapeutics SVP Global Operations, stated I am thrilled to join ASC Therapeutics, a leader in transformative gene and cell therapies extraordinarily well positioned to benefit from cost-reduction opportunities in manufacturing across the world.

ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A

Retrieved on: 
Thursday, July 8, 2021
Medical devices, Hospitals, FDA, Genetics, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Branches of biology, Life sciences, Health sciences, Applied genetics, Biotechnology, Drug discovery, Medicinal chemistry, Therapy, Gene therapy, Hemophilia A, ASC618, ASC Therapeutics, HEMOPHILIA A, ASC618, ASC THERAPEUTICS

Hemophilia A accounts for most cases of hemophilia (~80%), affecting approximately 1 of every 5000 live-born males.

Key Points: 
  • Hemophilia A accounts for most cases of hemophilia (~80%), affecting approximately 1 of every 5000 live-born males.
  • ASC618 was developed based on extensive work on a second-generation gene therapy for hemophilia A from an academic team at Emory University2.
  • ASC Therapeutics has obtained exclusive global rights to ASC618 from Expression Therapeutics and has conducted IND-enabling studies in multiple animal models3.
  • Molecular Therapy 2020;28/4:167-8
    4 ASC618 Gene Therapy in Hemophilia A Patients.