RGX-501 / REGENXBIO - LARVOL DELTA

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 (clinicaltrials.gov) - P=N/A; N=8; Active, not recruiting; Sponsor: Regenxbio Inc.; Enrolling by invitation ➔ Active, not recruiting; N=12 ➔ 8

Clinical • Enrollment change • Enrollment closed • Dyslipidemia • Gene Therapies • Genetic Disorders • Metabolic Disorders

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 Studio di follow-up a lungo termine per valutare la sicurezza e l'efficacia di RGX-501 (clinicaltrialsregister.eu) - P2; N=12; Ongoing; Sponsor: REGENXBIO Inc.

Clinical • New P2 trial • Cardiovascular • Dyslipidemia • Genetic Disorders • Metabolic Disorders

Regenxbio reports continued progress across programs in year-end 2018 corporate update (Regenxbio Press Release) - "As of December 31, 2018, a total of six subjects have been enrolled in two dose cohorts of the Phase I/II trial for RGX-501 for the treatment of HoFH...all three subjects enrolled at the dose of 7.5 x 10^12 GC/kg body weight (Cohort 2) experienced elevation in transaminases four to six weeks post-dosing, were asymptomatic and responded rapidly to the initiation of corticosteroids followed by a slow taper, with normalization of the transaminases...Resume enrollment in the Phase I/II clinical trial in the first half of 2019...Present interim data from Cohort 2 with steroid prophylaxis from the Phase I/II clinical trial in the second half of 2019."

Enrollment status • P1/2 data • Trial status • Cardiovascular • Dyslipidemia • Metabolic Disorders

Regenxbio Inc. (RGNX) Q4 2019 earnings call transcript (The Motley Fool) - "What we do know is from our work and the work that's the joint work between Olivier Danos' group and Peter Campochiaro's work is that with suprachoroidal delivery, we've seen good transduction without significant inflammation. And that's very different than of course what we know happens with intravitreal injection where as soon as you get to effective doses, you hand-in-hand get the inflammatory response due to the lack of immune privileged of that space. So at least based on our work with our particular vector, we seem to have much less of a risk of an inflammatory reaction. Having said that, of course we have to go into the clinic and see what we see there."

Biomarker • Media quote

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 (clinicaltrials.gov) - P=N/A; N=12; Enrolling by invitation; Sponsor: Regenxbio Inc.; Not yet recruiting ➔ Enrolling by invitation

Clinical • Enrollment open

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 (clinicaltrials.gov) - P; N=12; Not yet recruiting; Sponsor: Regenxbio Inc.

Clinical • New trial