Less than four months af­ter Taysha launched with a suite of AAV gene ther­a­pies de­vel­oped at UT South­west­ern — and two weeks af­ter the ex-AveX­is team raised $95 mil­lion in fresh cash — the biotech is look­ing to its chief sci­en­tif­ic ad­vi­sor for an­oth­er as­set.

The new pro­gram is ABO-202, which is de­signed to treat CLN1 dis­ease (or in­fan­tile Bat­ten dis­ease) by de­liv­er­ing a cor­rect copy of the PPT1 gene with a mod­i­fied AAV9 vec­tor.

Steven Gray

Steven Gray de­vel­oped the ther­a­py while at UNC-Chapel Hill, where he had stud­ied un­der Jude Samul­s­ki years ago. Abeona li­censed it in 2016, built out the clin­i­cal tri­al pro­to­col and man­u­fac­tur­ing process, and has since had an IND for a Phase I/II clin­i­cal tri­al cleared with the FDA.

Taysha is pay­ing on­ly $7 mil­lion up­front to get its hands on ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Abeona is el­i­gi­ble to re­ceive up to $56 mil­lion down the road for de­vel­op­ment and sales mile­stones, plus roy­al­ties.

The deal puts Taysha on track to be­gin two clin­i­cal stud­ies next year: both the CLN1 pro­gram and an­oth­er gene ther­a­py for Tay-Sachs dis­ease. Three more INDs are on the dock­et for 2021.

RA Ses­sion

“CLN1 is a pro­gres­sive mono­genic CNS dis­ease with sig­nif­i­cant un­met med­ical need, and we be­lieve the ABO-202 da­ta gen­er­at­ed thus far demon­strate great trans­la­tion­al po­ten­tial and of­fer hope to chil­dren suf­fer­ing from this dev­as­tat­ing dis­or­der,” CEO RA Ses­sion II said.

Gray, who now leads 50 trans­la­tion­al sci­en­tists and man­ages a GMP man­u­fac­tur­ing site at UT South­west­ern’s vi­ral vec­tor core, will con­tin­ue to ad­vise the com­pa­ny along­side Berge Mi­nass­ian, the di­vi­sion chief of child neu­rol­o­gy.

Rather than shep­herd­ing one as­set at a time like the first gen­er­a­tion of gene ther­a­py play­ers, Taysha had opt­ed for a port­fo­lio ap­proach to ad­vance mul­ti­ple gene ther­a­pies in par­al­lel. It’s look­ing to build three big fran­chis­es: ge­net­ic forms of epilep­sy, neu­rode­vel­op­men­tal dis­or­ders and neu­rode­gen­er­a­tive dis­or­ders. And al­though the crew has eyes on new­er tech­nolo­gies like re­dos­ing, bi­cistron­ic plas­mids, mi­croR­NA knock­down, hair­pin tech­nol­o­gy to turn on a silent al­lele and de­liv­ery through the va­gus nerve, the cor­ner­stone re­mains ade­no-as­so­ci­at­ed virus (AAV).

“If it’s not broke, you don’t fix it,” Ses­sion told End­points News back in April.

Sean Nolan

Taysha, which is chaired by for­mer AveX­is CEO Sean Nolan, has re­served rights to tap four more pro­grams out of Gray’s lab.

For Abeona, the deal with Taysha gives them time and some cash to fo­cus on its more ad­vanced pro­grams in re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, San­fil­ip­po syn­drome type A and San­fil­ip­po syn­drome type B as it re­cov­ers from a some­what tu­mul­tuous 2019.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.