RGX-111 / REGENXBIO, Nippon Shinyaku - LARVOL DELTA

First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT. (PubMed, Mol Ther) - "His neurodevelopment is significantly above the natural history of untransplanted Hurler syndrome patients. This single patient experience suggests central nervous system-directed gene therapy demonstrates therapeutic potential for severe MPS I and other genetic neurodegenerative diseases."

First-in-human • Journal • P1 data • Bone Marrow Transplantation • CNS Disorders • Developmental Disorders • Gene Therapies • Hurler Syndrome • Psychiatry • Transplantation • IDUA

RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov) - P1/2 | N=21 | Suspended | Sponsor: REGENXBIO Inc. | Trial completion date: Oct 2024 ➔ Mar 2029 | Active, not recruiting ➔ Suspended | Trial primary completion date: Mar 2023 ➔ Jul 2027

First-in-human • Trial completion date • Trial primary completion date • Trial suspension • Gene Therapies • Hurler Syndrome • IDUA

RGX-111 AN INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE I (MPS I): INTERIM ANALYSIS OF DATA FROM THE FIRST IN HUMAN STUDY (SSIEM 2023) - P1/2 | "RGX-111 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS I patients."

First-in-human • Gene therapy • P1 data • Gene Therapies • Hurler Syndrome • IDUA

REGENXBIO and Nippon Shinyaku Announce Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for MPS Diseases (PRNewswire) - "REGENXBIO Inc...and Nippon Shinyaku Co., Ltd...announced a strategic partnership for the development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome. Under the terms of the agreement, REGENXBIO will receive $110 million at closing and up to an additional $700 million if certain milestones are achieved, consisting of $40 million in potential development and regulatory milestones and $660 million in potential sales milestones. REGENXBIO will also receive meaningful double-digit royalties on net sales in the U.S. and Asia (collectively, the 'Licensed Territory')....REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway."

FDA approval • Licensing / partnership • Hunter Syndrome • Hurler Syndrome

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I. (PubMed, Mol Ther Methods Clin Dev) - "Histologic analysis showed minimal GAG storage and lysosomal pathology. We thus report a minimal effective dose of 1010 vg (5 × 1011 per kg) RGX-111 for IV and IT routes of administration in MPS I mice, which prevented neurocognitive deficit, cardiac insufficiency, and skeletal manifestations, as a model for genetic therapy of human MPS I."

Journal • Preclinical • Bone Marrow Transplantation • Congestive Heart Failure • Gene Therapies • Heart Failure • Hurler Syndrome • Metabolic Disorders • Transplantation • IDUA

Long Term Follow-Up for RGX-111 (clinicaltrials.gov) - P=N/A | N=8 | Enrolling by invitation | Sponsor: REGENXBIO Inc.

New trial

REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 (PRNewswire) - "REGENXBIO...announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel (August 29 – September 1). Investigators will deliver encore presentations of interim clinical trial data from REGENXBIO's pipeline of one-time investigational AAV Therapeutics for progressive, neurodegenerative lysosomal storage disorders, as well as initial interim data from the single-patient, investigator-initiated study of RGX-181 for the treatment of CLN2 disease."

Clinical data • CNS Disorders • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

RGX-111 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis of Data from the First in Human Study (ASGCT 2023) - P1/2 | "Interim results of this trial will be presented. Additionally, follow-up will be reported for a severe MPS I child treated at age 21 months utilizing a single-patient, investigator-initiated Investigational New Drug application."

Gene therapy • P1 data • Alzheimer's Disease • Bone Marrow Transplantation • CNS Disorders • Cognitive Disorders • Gene Therapies • Hurler Syndrome • Transplantation • Ventriculomegaly

RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov) - P1/2 | N=8 | Active, not recruiting | Sponsor: REGENXBIO Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2024 ➔ Oct 2024 | Trial primary completion date: Oct 2022 ➔ Apr 2023

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Hurler Syndrome

RGX-111 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis of Data from the First in Human Study (ASGCT 2022) - P1/2 | "Interim results of this trial will be presented. Additionally, two-year follow-up will be reported for a severe MPS I child treated at age 21 months utilizing a single-patient, investigator-initiated Investigational New Drug application."

P1 data • Alzheimer's Disease • Bone Marrow Transplantation • CNS Disorders • Cognitive Disorders • Gene Therapies • Hurler Syndrome • Transplantation • Ventriculomegaly

RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov) - P1/2; N=11; Recruiting; Sponsor: Regenxbio Inc.; Trial completion date: Jul 2023 ➔ Mar 2024; Trial primary completion date: Nov 2021 ➔ Oct 2022

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Hurler Syndrome

REGENXBIO REPORTS SECOND QUARTER 2020 FINANCIAL RESULTS AND OPERATIONAL HIGHLIGHTS (Regenxbio Press Release) - "RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS I): In July, REGENXBIO reported data under a single-patient investigator-initiated Investigational New Drug application (IND) conducted at CHOC Children's, which demonstrated encouraging biomarker activity and continued progression of neurocognitive development....Recruitment and patient screening are ongoing in the Company's Phase I/II clinical trial evaluating RGX-111 for the treatment of MPS I. REGENXBIO expects to provide a program update by the end of 2020."

Trial status • Genetic Disorders • Hurler Syndrome

REGENXBIO Provides Update on Progress of Clinical Programs for Rare Genetic Neurodegenerative Diseases (PRNewswire) - "REGENXBIO Inc....today announced that it has completed dosing of three patients in Cohort 2 of the Company's Phase I/II study of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and reported encouraging data under a single-patient investigator-initiated Investigational New Drug (IND) application for RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) conducted at CHOC Children's....'We are encouraged by the initial data from the first patient dosed with RGX-111, and we look forward to advancing our RGX-111 Phase I/II study'...Additional data from both cohorts in this study and a program update will be available in the second half of 2020."

Enrollment status • P1/2 data • Trial status • Genetic Disorders • Hurler Syndrome

RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov) - P1/2; N=5; Recruiting; Sponsor: Regenxbio Inc.; Phase classification: P1 ➔ P1/2; Trial completion date: Jan 2022 ➔ Jul 2023; Trial primary completion date: Jan 2020 ➔ Nov 2021

Clinical • Phase classification • Trial completion date • Trial primary completion date

RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov) - P1; N=5; Recruiting; Sponsor: Regenxbio Inc.; Trial completion date: Aug 2021 ➔ Jan 2022; Initiation date: Sep 2018 ➔ Apr 2019; Trial primary completion date: Aug 2019 ➔ Jan 2020

Clinical • Trial completion date • Trial initiation date • Trial primary completion date