Sean Nolan, RA Session II

Crossover round? Check. Top team? Check. Taysha sails to an IPO with $100M ini­tial ask

On the same day No­var­tis erased the AveX­is brand from the com­pa­ny, the ex-AveX­is crew says their new gene ther­a­py start­up is ready for an IPO.

Taysha Gene Ther­a­pies has left enough bread crumbs for biotech sleuths to fig­ure out that it’s go­ing down this path. CEO RA Ses­sion II, who hap­pi­ly chat­ted to mul­ti­ple re­porters at launch, de­clined in­ter­views for the re­cent Se­ries B (and pre­sumed crossover round); the biotech brought in a gene ther­a­py pro­gram de­vel­oped by its co-founder Steven Gray from Abeona; and days ago it tapped the ex-CMO and ex-CFO of AveX­is to join the board, chaired by their for­mer boss Sean Nolan.

What’s still re­mark­able is the speed at which Dal­las-based Taysha has moved. It’s been just a year since the com­pa­ny was formed, a lit­tle over four months since Taysha launched with $30 mil­lion and a slate of 15 pro­grams, and bare­ly a month af­ter it closed a $95 mil­lion Se­ries B. The ac­cu­mu­lat­ed deficit so far is just $27.8 mil­lion.

That casts a unique spot­light on Taysha com­pared to the 48 play­ers that have rid­den to a pub­lic list­ing on the biotech boom so far this year. Even Pas­sage Bio, the Philadel­phia-based start­up that has a sim­i­lar pact with gene ther­a­py gu­ru Jim Wil­son, took a year from Se­ries A to go pub­lic.

For now Taysha is pen­cil­ing in a $100 mil­lion raise, but these days the place­hold­er fig­ure could mean lit­tle.

Oth­er than lay­ing out the clin­i­cal tri­al time­lines for the lead pro­grams — the first study, a Phase I/II of TSHA-101 for GM2 gan­gliosi­do­sis, is planned in Cana­da for lat­er this year — the S-1 re­veals some in­ter­est­ing de­tails about the part­ner­ships that are core to the biotech.

To get UT South­west­ern on board, it turned out Taysha didn’t pay a pen­ny. In­stead, it is­sued 2 mil­lion shares of its stock to the uni­ver­si­ty, with no fu­ture mile­stones or roy­al­ty promis­es at­tached oth­er than costs of main­tain­ing patents. Gray, an ex­pert in AAV-based gene ther­a­pies for CNS dis­or­ders, is lead­ing the col­lab­o­ra­tion along­side child neu­rol­o­gy di­vi­sion chief Berge Mi­nass­ian.

By the end of 2021, Taysha al­so ex­pects to file INDs in the US for four pro­grams, all pack­aged in an AAV9 vec­tor and span­ning the three pil­lars of neu­rode­gen­er­a­tive, neu­rode­vel­op­men­tal and epilep­tic dis­or­ders:

TSHA-101, a bi­cistron­ic HexBP2A-HexA trans­gene pack­aged in­to an AAV9 vec­tor un­der the con­trol of a CAG pro­mot­er de­signed for con­di­tions like Tay-Sachs and Sand­hoff dis­ease. TSHA-102, which com­bines a neu­ronal spe­cif­ic pro­mot­er, MeP426, with the min­iMECP2 trans­gene, plus a miR­NA-Re­spon­sive Au­to-Reg­u­la­to­ry El­e­ment for the treat­ment of Rett syn­drome. TSHA-103, which tar­gets a ge­net­ic form of epilep­sy caused by SLC6A1 hap­loin­suf­fi­cien­cy dis­or­der, is con­struct­ed from a codon-op­ti­mized ver­sion of the hu­man SLC6A1 gene un­der the con­trol of a JeT pro­mot­er. TSHA-104, which ad­dress­es SURF1 de­fi­cien­cy, adopts a ver­sion of the hu­man SURF1 gene un­der the con­trol of a mod­i­fied ver­sion of the pro­mot­er CBA hy­brid in­tron, or CBh.

With 18 pro­grams now in the pipeline re­flect­ing a port­fo­lio ap­proach, Taysha is en­ti­tled to an op­tion for four more pro­grams un­der the re­search pact with UT South­west­ern, which ex­pires in No­vem­ber 2021.

Then there’s TSHA-118, a po­ten­tial treat­ment of in­fan­tile Bat­ten dis­ease in­vent­ed by Gray that Taysha re­cent­ly li­censed from Abeona. Taysha is paid on­ly $7 mil­lion up­front to get its hands on the clin­ic-ready drug, then known as ABO-202, with $3 mil­lion in li­cense fees and $4 mil­lion for the in­ven­to­ry — in­clud­ing clin­i­cal-grade CLN1 plas­mid. Mile­stones add up to $56 mil­lion.

In its fil­ing, Taysha dis­closed that it’s al­so struck a deal with Queen’s Uni­ver­si­ty at Kingston to ac­cess some of its tech­nolo­gies, in­clud­ing plas­mid pro­duc­tion, for next-gen­er­a­tion gene ther­a­pies. In ad­di­tion to $3 mil­lion up­front, it re­im­bursed around $220,000 in costs and agreed to pay an­oth­er $20 mil­lion for mile­stones.

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Brian Hubbard, Anji Pharmacetuticals CEO

Look­ing to rewrite the rules of drug li­cens­ing, start­up An­ji is on the hunt for 'dy­nam­ic eq­ui­ty' joint ven­tures

Licensing is one of the most common ways big drugmakers leverage biotech innovation to drive gains across their pipelines — and the structure of those deals is pretty well established. But one biotech with home bases in China and the US thinks it may have a better way.

On Tuesday, Cambridge-based biotech Anji Pharma closed a $70 million Series B with two late-stage molecules in the fold and a mission to rewrite the rules of drug licensing through what it calls “dynamic equity” deals and a joint venture-heavy game plan. The round was funded in whole by Chinese hedge fund CR Capital.

Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.

From left to right: Mark Springel, Kristina Wang, Lin Ao, Soufiane Aboulhouda

George Church, his stu­dents, and top VCs go na­tion­wide with a biotech train­ing camp

One night last fall, Floris Engelhardt sat down in her Boston apartment and logged onto a Zoom call, armed with a comic and a vague idea about starting a biotech.

Engelhardt was joining a student-run “match night.” A postdoc at MIT’s Bathe BioNanoLab, where researchers use DNA and RNA like Lego blocks for nanometer-sized structures, Engelhardt wanted to find real-world applications for her work. She sketched out — literally — a plan to use DNA origami, a decade-old technique for precisely folding DNA, to make therapies.

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

Sanofi calls it quits on mR­NA Covid-19 shots, scrap­ping vac­cine from $3.2B Trans­late Bio buy­out

Sanofi is throwing in the towel on mRNA-based Covid-19 vaccines.

The French drugmaker will halt development on its unmodified mRNA Covid-19 shot despite what it said were positive Phase I/II results, a spokesperson told Endpoints News on Tuesday morning. Sanofi said the reason it’s stopping the Covid-19 mRNA program, developed in partnership with its new $3.2 billion acquisition Translate Bio, is because the market is too crowded.