Ann: ATL1102 DMD Paediatric Investigation Plan submitted to EMA, page-27

Good point about monitoring Heart function Sammy...in hindsight this should have been covered in P2 but here we are...

Your thinking could well be spot on, importantly that framework covers 100% of DMD boys.

Sarepta currently only have a drug for 20% of the boys, and revenues expected to top $500m per year in 2021.

Commercially speaking - the rewards for Big Pharma here are massive if we get success in P2B for ambulant and non-ambulant boys.

Another reason why the $1B Roche <-> Sarepta deal should be the benchmark for partnership.

My comments re "combination therapy" stem from Sarepta's predicament with Exondys and needing to provide FDA with efficacy data to protect revenues...let alone their gene therapy.


Exciting times.