Microperimetry

Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa

Retrieved on: 
Thursday, February 8, 2024
Schepens Eye Research Institute, VISTA, GTPase, Patient, Safety, Medical genetics, RPGR, Retina, Therapy, Science, Microperimetry, Retinitis pigmentosa, Pharmaceutical industry

Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).

Key Points: 
  • Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).
  • In addition, patients in the high dose cohort also demonstrated a robust improvement in visual function, including mean retinal sensitivity.
  • XLRP is an orphan disease predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  • We look forward to announcing the initiation of our Phase 2/3 VISTA trial in the first half of 2024."

Coave Therapeutics announces Positive 12-months Data from Ongoing Phase I/II Clinical Trial of CTx-PDE6b in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

Retrieved on: 
Wednesday, May 31, 2023
Optical coherence tomography, Retina, PDE6B, Eye, Retinitis pigmentosa, Visual impairment, Visual field, Paratriathlon, Doctor of Philosophy, CNS, Associate, Central nervous system, Patient, OCT, Microperimetry, Mobility, Ageing, Phase, Therapy, Disease, Safety, Pharmaceutical industry, Medical imaging, Ophthalmology, MD

These positive data support Coave’s preparations for a registrational trial with CTx-PDE6b in this indication.

Key Points: 
  • These positive data support Coave’s preparations for a registrational trial with CTx-PDE6b in this indication.
  • To date, CTx-PDE6b has been administered to 17 patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses.
  • The treatment was administered in the more affected eye while the other eye served as an untreated control.
  • “We are highly encouraged by the safety and efficacy data observed so far in patients who have been treated with CTx-PDE6b.