RG6359 / Roche - LARVOL DELTA

Comprehensive Preclinical Integration Site Analyses of SPK-3006, a Liver-Mediated AAV Gene Therapy for Pompe Disease Reveals a Low Theoretical Risk for Insertional Mutagenesis (ASGCT 2024) - "In addition we provide proof of concept for the value of minimal invasive AAV integration site analyses on cell free DNA. This work further expands our understanding of AAV integration of a liver tropic capsid in primary NHPs and human hepatocytes, serving as a resource for future AAV integration related studies in preclinical models."

Gene therapy • Preclinical • Gastrointestinal Cancer • Gene Therapies • Genetic Disorders • Hepatocellular Cancer • Oncology • Pompe Disease • Solid Tumor

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2 | N=4 | Active, not recruiting | Sponsor: Spark Therapeutics | N=30 ➔ 4 | Trial completion date: Oct 2027 ➔ Apr 2032 | Trial primary completion date: Oct 2023 ➔ Apr 2032

Enrollment change • Trial completion date • Trial primary completion date • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2 | N=30 | Active, not recruiting | Sponsor: Spark Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2; N=30; Recruiting; Sponsor: Spark Therapeutics; N=20 ➔ 30; Trial completion date: Oct 2023 ➔ Oct 2027

Clinical • Enrollment change • Trial completion date • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (clinicaltrials.gov) - P=N/A; N=61; Completed; Sponsor: Spark Therapeutics; Recruiting ➔ Completed; Trial completion date: Jun 2022 ➔ Oct 2020; Trial primary completion date: Jun 2022 ➔ Oct 2020

Trial completion • Trial completion date • Trial primary completion date • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (clinicaltrials.gov) - P=N/A; N=60; Recruiting; Sponsor: Spark Therapeutics; Active, not recruiting ➔ Recruiting

Enrollment open • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2; N=20; Recruiting; Sponsor: Spark Therapeutics; Suspended ➔ Recruiting; Trial completion date: May 2023 ➔ Aug 2023; Initiation date: May 2020 ➔ Aug 2020; Trial primary completion date: May 2023 ➔ Aug 2023

Clinical • Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (clinicaltrials.gov) - P=N/A; N=60; Active, not recruiting; Sponsor: Spark Therapeutics; Recruiting ➔ Active, not recruiting

Enrollment closed • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2; N=20; Suspended; Sponsor: Spark Therapeutics; Recruiting ➔ Suspended

Clinical • Trial suspension • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) (clinicaltrials.gov) - P1/2; N=20; Recruiting; Sponsor: Spark Therapeutics; Not yet recruiting ➔ Recruiting; Trial completion date: Dec 2022 ➔ Mar 2023; Initiation date: Dec 2019 ➔ Mar 2020; Trial primary completion date: Dec 2022 ➔ Mar 2023

Clinical • Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date

A Gene Transfer Study for Late-Onset Pompe Disease (clinicaltrials.gov) - P1/2; N=20; Not yet recruiting; Sponsor: Spark Therapeutics

Clinical • New P1/2 trial

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (clinicaltrials.gov) - P=N/A; N=60; Recruiting; Sponsor: Spark Therapeutics; Not yet recruiting ➔ Recruiting; Trial completion date: Feb 2022 ➔ Jun 2022; Trial primary completion date: Feb 2022 ➔ Jun 2022

Enrollment open • Trial completion date • Trial primary completion date

Efficacy and Safety Evaluation of Investigational Liver Gene Transfer for Secretable GAA in the Treatment of Pompe Disease (ASGCT 2019) - "While pre-clinical efficacy and safety studies are ongoing, no toxicity has been observed in any of the studies to date. These results support moving SPK-3006 into clinical development."

Clinical

Neutralizing Antibody Screening Study With a Retrospective Component in Participants With Late-Onset Pompe Disease (clinicaltrials.gov) - P=N/A; N=60; Not yet recruiting; Sponsor: Spark Therapeutics

New trial