Ver­tex is pulling back on its ge­net­ic med­i­cine re­search, end­ing all work re­lat­ed to ade­no-as­so­ci­at­ed virus­es, or AAVs — the vi­ral vec­tors com­mon­ly used to de­liv­er gene ther­a­pies in­to the body, the com­pa­ny con­firmed to End­points News.

The cuts im­pact Ver­tex’s col­lab­o­ra­tions with at least two biotech star­tups: the AAV de­sign com­pa­ny Affinia Ther­a­peu­tics and tR­NA ther­a­py com­pa­ny Tevard Bio­sciences, which us­es AAVs. It al­so calls in­to ques­tion Ver­tex’s work on CRISPR ther­a­pies for ge­net­ic mus­cle dis­eases.

Sev­er­al big­ger phar­ma­ceu­ti­cal com­pa­nies, in­clud­ing Bio­gen, Pfiz­er, Roche and Take­da, have cut or pared back on AAV gene ther­a­py re­search in re­cent years. And sev­er­al gene ther­a­py biotech com­pa­nies have trimmed their pipelines and staff as pri­vate and pub­lic in­vestors sour on the field.  

Lead­er­ship in the FDA's Of­fice of New Drugs sent an email to staff Fri­day af­ter­noon ask­ing if any­one would vol­un­teer to take on more ad­min­is­tra­tive work, fol­low­ing the fir­ing of many of the agen­cy's ad­min­is­tra­tors and project man­agers last month.

The email from the Of­fice of New Drugs lead­er­ship, which was re­viewed by End­points News, said that the FDA's Cen­ter for Drug Eval­u­a­tion and Re­search "is still work­ing on a long-term so­lu­tion for our time­keep­ing needs," re­fer­ring to the process of track­ing and pro­cess­ing FDA staffer­s' hours and ver­i­fy­ing that they're cor­rect.

"If folks are will­ing to be trained to be a time­keep­er or have pri­or time­keep­ing ex­pe­ri­ence (does not need to be re­cent), please re­spond back," the email said.

HHS did not im­me­di­ate­ly re­spond to a re­quest for com­ment.

The White House’s bud­get re­quest pro­pos­es near­ly $18 bil­lion in cuts to NIH spend­ing, and more than $3.5 bil­lion slashed at the CDC. And the changes would in­clude ma­jor con­sol­i­da­tion at both agen­cies.

Else­where in the pro­posed bud­get, HHS Sec­re­tary Robert F. Kennedy Jr. would snag $500 mil­lion to tack­le some of the Make Amer­i­ca Healthy Again ini­tia­tives that are core to his goals for the agency.

The bud­get re­quest, re­leased Fri­day, pro­pos­es that the NIH fuse in­to five new na­tion­al in­sti­tutes, down from 21. Those in­sti­tutes would be Body Sys­tems Re­search, Neu­ro­science and Brain Re­search, Gen­er­al Med­ical Sci­ences, Dis­abil­i­ty Re­lat­ed Re­search, and Be­hav­ioral Health. A se­nior White House of­fi­cial cit­ed high in­di­rect costs as part of the ra­tio­nale for the over­haul.

The FDA on Fri­day said it's pulling two fi­nal guid­ance doc­u­ments pub­lished ear­li­er this year that sought to help en­ti­ties man­u­fac­tur­ing cell- and tis­sue-based med­ical prod­ucts to make bet­ter donor el­i­gi­bil­i­ty de­ter­mi­na­tions.

At the time the guid­ance doc­u­ments were fi­nal­ized in Jan­u­ary, the FDA said it de­ter­mined a need for up­dat­ed rec­om­men­da­tions for mak­ing such el­i­gi­bil­i­ty de­ter­mi­na­tions to re­duce the risk of in­fec­tions due to sep­sis. The guid­ances, reis­sued as drafts on Fri­day, fol­lowed a na­tion­wide out­break in 2021 of more than 110 tu­ber­cu­lo­sis in­fec­tions in re­cip­i­ents of al­lo­graft bone prod­ucts. A Delaware hos­pi­tal in 2021 ini­tial­ly not­ed an "un­usu­al clus­ter of tu­ber­cu­lo­sis cas­es in pa­tients who had un­der­gone spinal surgery, in­volv­ing im­plan­ta­tion of bone al­lo­graft ma­te­r­i­al from a sin­gle prod­uct lot," the CDC said.

Cy­to­ki­net­ics said late Thurs­day that the PDU­FA date for its heart dis­ease ther­a­py afi­camten would be de­layed by three months to Dec. 26. The lag would al­low the FDA to re­view a ma­jor change to its mar­ket­ing ap­pli­ca­tion, name­ly the sub­mis­sion of a drug safe­ty pro­gram or REMS.

The ma­jor sur­prise here is that the com­pa­ny did not in­clude a REMS in its orig­i­nal NDA fil­ing. Stifel an­a­lysts de­scribed the omis­sion as “not at all the Street's ex­pec­ta­tion.” Pre­vi­ous­ly, Cy­to­ki­net­ics’ man­age­ment had said it would pro­pose a REMS pack­age when it sub­mit­ted the NDA, they wrote on a Thurs­day note.

Cy­to­ki­net­ics’ stock CYTK was down by as much as 12% pre­mar­ket Fri­day as in­vestors di­gest­ed the ar­guably un­nec­es­sary de­lay.