… an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …
… an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 St
Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential next steps with this program and overall strategy anticipated in Q2 2022
Phase 2/3 Sirius and Celeste clinical trials of ultevur
… an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …
… an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …
… ® , which uses a cell’s own editing machinery called ADAR (adenosine deaminase acting on RNA) to make specific … the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including …
… Axiomer®, which uses a cell’s own editing machinery called ADAR (adenosine deaminase acting on RNA) to make specific … the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including …