REGENXBIO has today announced two important, positive milestones in their CNS and ocular gene therapy programs for the treatment of CLN2 disease:
- Patient dosed under a single-patient investigator-initiated study of RGX-181 (TPP1 gene delivered directly into the central nervous system (CNS)).
- Approval of CTA for RGX-381 from the UK Health Authority and plans to initiate a Phase I/II clinical trial in the first half of 2023 (TPP1 gene delivered into eye (subretina)).
For further information, please see today’s press release from REGENXBIO below. For any of our families wishing to know more, please contact our Head of Research & Medical Affairs at research@bdsraaustralia.org