ASC-618 / ASC Therapeutics, Expression Therap - LARVOL DELTA

Structural, functional and carcinogenicity as essential liver biomarkers for gene therapy of hemophilia A (ISTH 2023) - P1/2 | "The prospective clinical study will assess these biomarkers in hemophilia A patients.Conclusion(s): The ASC618 gene therapy clinical program aims to evaluate overall safety with a focus on liver structure, function and carcinogenicity, with efficacy endpoints assessing its potential to provide durable therapeutic benefits, reduction of the treatment burden and significant improvement of quality of life of patients."

Biomarker • Gene therapy • Fibrosis • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Immunology • Liver Cirrhosis • Osteoarthritis • Rare Diseases • Rheumatology • HSPA5

Development of AAV Transduction and Potency Assays to Evaluate Quality Attributes of ASC618, a Second Generation AAV8-Based Hemophilia A Gene Therapy (ASGCT 2023) - P1/2 | "The potency of ASC618 GMP lots relative to that of a reference standard (ASC618 Engineering Lot), which is evaluated by two assays that we have developed: a Potency-ELISA assay and a Potency-Activity assay, which XYNTHA® Antihemophilic Factor VIII (Recombinant) was used to generate standard curves for both assays. By using the transduction, Potency-ELISA, and Potency-Activity assays, three independent batches of ASC618 drug substance (DS) or drug product (DP) have been tested. The relative transduction confirmed high consistency with the relative potency in both ELISA and Activity assays."

Gene therapy • Gastrointestinal Cancer • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Liver Cancer • Oncology • Rare Diseases • Solid Tumor

ASC618 Gene Therapy in Hemophilia A Patients (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: ASC Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Clinical Study of Adenovirus-Associated Viral Vector-Mediated Gene Therapy of Human Factor VIII in Severe and Moderately Severe Hemophilia A (ISTH 2022) - P1/2 | "Preclinical work demonstrated that in the C57Bl/6 murine model, the cynomolgus monkey, and the humanized liver mouse model (FRG-KO), ASC618 was well tolerated at all doses evaluated with no toxicologically significant microscopic findings (Veselinovic 2020). Conclusion(s): ASC618 will be evaluated in this clinical program for its potential to provide durable therapeutic benefit, reduce treatment burden on patients and significantly improve their quality of life."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Osteoarthritis • Rare Diseases • Rheumatology

ASC618, a Second Generation of FVIII Gene Therapy for Hemophilia A, Exhibits Major Transduction and Transgene Expression in the Target Liver Tissues: Results of IND-Enabling Pharmacokinetics Studies in Mice and Non-Human Primates (ASGCT 2022) - P1/2 | "These results were included in the ASC618 IND submission and the program received IND clearance from the U.S. FDA in July 2021. A phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618 (NCT04676048) will be conducted."

PK/PD data • Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Pharmacology, Toxicology and Safety Studies of ASC618, a Second Generation Factor VIII Gene Therapy for Hemophilia A (ASGCT 2022) - P1/2 | "The no-observed-effect level (NOEL) has also not been established since the lowest tested dose 2E11 was found to be efficacious. Based on these results and dose translation approach the program received IND clearance from the U.S. FDA and a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618 (NCT04676048) will be conducted."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Development of a New Transduction Assay to Evaluate In-Vitro Relative Infectivity of ASC618, a Second Generation AAV8-Based Bioengineered Factor VIII Gene Therapy (ASGCT 2022) - P1/2 | "Since relative errors in these measurements are within 30% for all tested QC samples, we suggest that this new Transduction Assay has an acceptable accuracy to evaluate the relative infectivity of an ASC618 test lot as a reference standard. This approach was presented in the ASC618 IND submission and the program received IND clearance from the U.S. FDA and a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618 (NCT04676048) will be conducted."

Preclinical • Gastrointestinal Cancer • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Infectious Disease • Liver Cancer • Oncology • Rare Diseases • Solid Tumor

ASC618 Gene Therapy in Hemophilia A Patients (clinicaltrials.gov) - P1/2; N=12; Not yet recruiting; Sponsor: ASC Therapeutics; Trial completion date: Jul 2026 ➔ Dec 2026; Initiation date: Jul 2021 ➔ Apr 2022; Trial primary completion date: Jul 2023 ➔ Dec 2023

Clinical • Trial completion date • Trial initiation date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A (PipelineReview) - "ASC Therapeutics...announced that the U.S. Food & Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for ASC618, an investigational second-generation gene therapy for patients with severe and moderately severe hemophilia A. The transformational Adeno-Associated Virus (AAV) construct contains a proprietary B-domain deleted codon-optimized bioengineered chimeric Factor VIII (FVIII) gene and a minimal-length liver-specific promoter, shown in pre-clinical studies to produce therapeutic levels of FVIII protein at doses that are significantly lower than other constructs expressing the native human FVIII currently in clinical trials."

IND • Hemophilia

ASC618 Gene Therapy in Hemophilia A Patients (clinicaltrials.gov) - P1/2; N=12; Not yet recruiting; Sponsor: ASC Therapeutics; Trial completion date: Apr 2026 ➔ Jul 2026; Initiation date: Apr 2021 ➔ Jul 2021; Trial primary completion date: Apr 2026 ➔ Jul 2023

Clinical • Trial completion date • Trial initiation date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

ASC-618 Gene Therapy in Hemophilia A Patients (clinicaltrials.gov) - P1/2; N=12; Not yet recruiting; Sponsor: ASC Therapeutics

Clinical • New P1/2 trial • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases