Taysha buys clin­ic-ready gene ther­a­py from Abeona, bring­ing Steven Gray's port­fo­lio back in one place

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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Watch out, Roche: No­var­tis inks $1.5B deal to chase down promi­nent Parkin­son’s tar­get

Novartis is plopping down $150 million in cash to pick up an experimental Parkinson’s drug and grab an option to another, a move that puts it on an increasingly popular path in the field’s search for disease-modifying therapies.

Belgium’s UCB is its partner of choice, supplying two small molecule alpha-synuclein misfolding inhibitors in a deal that can add up to nearly $1.5 billion.

Out of the pair, UCB0599 is already in Phase II trials, making Novartis confident enough to pull the trigger on co-development and commercialization, including to foot half of the R&D bill. The pharma giant will make a decision on UCB7853 once UCB wraps the ongoing Phase I program.

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Glax­o­SmithK­line, Ox­ford un­veil new part­ner­ship pit­ting buzzy R&D ad­vances against neu­ro­log­i­cal dis­ease

When GlaxoSmithKline trumpeted its return to neuroscience with a $700 million upfront deal with Alector this summer, it touted its early investments in functional genomics as a key guidepost for that deal. Now, the drug giant has partnered up with Oxford to hopefully add jet fuel to its hunt for breakthroughs in the brain.

GSK and Oxford have kickstarted a five-year collaboration aimed at spurring R&D breakthroughs across a range of hard-to-treat diseases like Alzheimer’s and Parkinson’s through the use of genomic testing and machine learning, the partners said Wednesday.

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi snaps up new vac­cine can­di­date and de­vis­es mR­NA game plan around it — but not for what you think

Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.

The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.

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