A rare dis­ease gene ther­a­py from Rock­et Phar­ma­ceu­ti­cals has gar­nered FDA ap­proval af­ter an ear­li­er re­jec­tion for man­u­fac­tur­ing prob­lems.

The FDA on Thurs­day grant­ed ac­cel­er­at­ed ap­proval to Rock­et Phar­ma’s gene ther­a­py for the se­vere form of a rare im­mune dis­or­der called leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-I). The gene ther­a­py, to be mar­ket­ed as Kres­la­di, was ap­proved to treat cer­tain ba­bies and chil­dren with the dis­ease who don’t have a matched sib­ling stem cell donor.

The com­pa­ny did not im­me­di­ate­ly dis­close the list price for Kres­la­di.

LAD-I is caused by mu­ta­tions in the IT­GB2 gene and leads the im­mune sys­tem to stop work­ing prop­er­ly. Pa­tients with se­vere dis­ease face se­ri­ous and po­ten­tial­ly dead­ly bac­te­r­i­al and fun­gal in­fec­tions. Cur­rent­ly, the on­ly po­ten­tial cure is stem cell trans­plant from a donor, but that bears its own se­ri­ous risks.

New clin­i­cal tri­al da­ta on the in vi­vo CAR-T ther­a­py that As­traZeneca ac­quired last year sug­gest that while the ex­per­i­men­tal treat­ment can curb mul­ti­ple myelo­ma in some pa­tients, it may not be safer than the cell ther­a­pies en­gi­neered in a lab.

Com­pared to con­ven­tion­al CAR-T cell ther­a­pies, which have up­end­ed mul­ti­ple myelo­ma treat­ment but are chal­leng­ing to make and give, in vi­vo cell ther­a­pies are meant to be a sim­ple in­fu­sion that in­structs a pa­tien­t's own cells to fight dis­ease.

The modal­i­ty is still in the ear­ly days of de­vel­op­ment, and there have been lim­it­ed da­ta re­port­ed on how the class of treat­ments fare in pa­tients. The lat­est Na­ture Med­i­cine study by re­searchers from Tongji Hos­pi­tal in Wuhan gives a deep­er view on the nu­ances around the safe­ty of the first as­set that was re­port­ed to en­ter clin­i­cal stud­ies, a mul­ti­ple myelo­ma ther­a­py called ESO-T01 which is now owned by As­traZeneca.

Plus, news about Zenas and Ys­ios Cap­i­tal:

🌀 Anap­tys­Bio's spin­out: The in­de­pen­dent pub­lic com­pa­ny, called First Tracks Bio­ther­a­peu­tics, is ex­pect­ed to start trad­ing on April 20. It will launch with $180 mil­lion in cash and three lead as­sets in the clin­ic for au­toim­mune and in­flam­ma­to­ry dis­eases. Mean­while, Anap­tys will fo­cus on man­ag­ing its “roy­al­ty streams,” in­clud­ing col­lab­o­ra­tions with GSK for Jem­per­li and with Van­da for im­si­dolimab. — Nicole De­Feud­is

📊 Af­fi­body touts dura­bil­i­ty for der­ma­tol­ogy can­di­date: The Swedish biotech said its ex­per­i­men­tal hidradeni­tis sup­pu­ra­ti­va ther­a­py izok­ibep showed “sus­tained ef­fi­ca­cy” in pa­tients who con­tin­ued to take the drug for an­oth­er 16 weeks in a Phase 3 tri­al. Pa­tients who had start­ed on place­bo but crossed over to treat­ment with izok­ibep saw “no­table rates of re­sponse.” CEO David Be­jk­er said in a news re­lease that the re­sults “strength­en our con­fi­dence in izok­ibep.” Ace­lyrin had li­censed the can­di­date, but hand­ed back the rights last year. — Nicole De­Feud­is