Ann: New data supports ATL1102s broader clinical potential, page-47

And from the same WMS 2021 last Friday, this from Roche, words like "genera improvement, biological endpoints and did not achieve satistical significance"

Cant wait for a real cure like ATL1102 to suck out that 550m revenue for us to have their 7B market cap.

."In DMD, three-year data from the open-label trial, Study SRP-9001-101, evaluating the safety of a single dose of the investigational gene therapy SRP-9001 in four ambulatory children aged 4 and 7 years old with DMD, were presented. The study showed that SRP-9001 was well-tolerated with key functional assessment, measured by the North Star Ambulatory Assessment (NSAA), demonstrating an overall improvement in motor ability compared to baseline. The improvements in motor abilities were maintained thoverree years, signifying a durable response. The results support the continuation of clinical investigation to further assess the benefit/risk of SRP-9001 in patients with DMD.

Roche, and partner Sarepta, also shared data from ENDEAVOR (Study SRP-9001-103), the first clinical trial using commercially representative SRP-9001 material for the treatment of DMD. Interim results from the first 11 participants in Cohort 1 (ambulatory boys aged 4-7 years) from the open label, ongoing Phase 1b study, provides evidence that SRP-9001 showed robust expression of micro-dystrophin and no new safety signals were identified.

In addition, results from Part 1 of Study SRP-9001-102, an ongoing, randomised, double-blind, placebo-controlled clinical trial evaluating the safety, efficacy, and tolerability of a single dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary biological endpoint of change in micro-dystrophin protein expression from baseline. Participants treated with SRP-9001, generally showed an increase in NSAA total score compared to placebo at 48 weeks, although this increase did not achieve statistical significance compared to that of patients who received placebo. The safety profile was consistent with prior studies, with no new safety signals identified.

The results of these studies provide important information for SRP-9001’s ongoing clinical development programme, with outcomes from Study 101 and 102 informing the design of the Phase 3 trial for SRP-9001, due to commence globally by the end of the year."