drugset
Drugs / LYS-SAF302
last change Mar 2022 re-read 3 minutes ago

LYS-SAF302

targets SGSH

Developed for
mucopolysaccharidosis type 3A
Investigated by
LYSOGENE

Trials 1

today NCT03612869 · Ph 2/3
20192020202120222023202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 2/3 NCT03612869 Dec 2018 → Mar 2022 mucopolysaccharidosis type 3A LYSOGENE Unknown No outcome recorded

Press releases naming this drug 2 releases

DateIssuerRelease
2019-02-14 Sarepta Therapeutics, Inc. Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A) sarepta.com ↗
2018-10-15 Sarepta Therapeutics, Inc. Sarepta and Lysogene Announce Exclusive License Agreement for LYS-SAF302, a Late-stage Gene Therapy for the Treatment of MPS IIIA, and Grant of Option Rights to an Additional CNS Gene Therapy Candidate sarepta.com ↗

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as LYS-SAF302 ClinicalTrials.gov intervention name — accepted as the source's own label NCT03612869 ↗
Route Other “One-time intracerebral administration of adeno-associated viral vector serotype rh10” NCT03612869 ↗
Target SGSH “deliver a functional copy of the SGSH gene to the brain” NCT03612869 ↗