Can prog­en­i­tor cells go fur­ther than gene ther­a­py in reti­nal dis­or­ders? A biotech's an­swer shines in PhI­Ib

By the time the FDA ap­proved Lux­tur­na — the pi­o­neer­ing gene ther­a­py for an in­her­it­ed reti­nal dis­ease — Hen­ry Klassen had been re­search­ing and treat­ing the group of dis­eases known as re­tini­tis pig­men­tosa for more than 20 years.

Spark Ther­a­peu­tics’ suc­cess there had not just in­spired a $4.3 bil­lion takeover by Roche, it had al­so em­bold­ened oth­er biotechs pur­su­ing a ther­a­py that would de­liv­er one of the genes tied to dif­fer­ent vari­a­tions of the dis­or­der. Night­star Ther­a­peu­tics sub­se­quent­ly scored its own buy­out with Bio­gen, and MeiraGTx re­cent­ly post­ed ear­ly but “ex­cit­ing da­ta” on its J&J-part­nered pro­gram.

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