RA Session II, Taysha Gene Therapies CEO (Taysha)

Taysha Gene Ther­a­pies gears up to ap­proach reg­u­la­tors with a new can­di­date for a de­bil­i­tat­ing neu­rode­gen­er­a­tive dis­ease

Af­ter launch­ing ear­ly last year, Taysha Gene Ther­a­pies quick­ly went about check­ing off key mile­stones. With­in five months, it pulled in a hefty Se­ries B round and made the jump to Nas­daq. And now, it has a late-stage can­di­date that CEO RA Ses­sion II says is just about ready for reg­u­la­tors.

The Dal­las-based biotech on Mon­day un­veiled a deal for glob­al rights to TSHA-120 — a gene ther­a­py dis­cov­ered in the lab of UT South­west­ern’s Steven Gray to treat a de­bil­i­tat­ing neu­rode­gen­er­a­tive dis­ease called gi­ant ax­on­al neu­ropa­thy (GAN). It’s the first gene ther­a­py to be ad­min­is­tered in hu­mans in­trathe­cal­ly (in­to the spinal canal), ac­cord­ing to Ses­sion, who’s cur­rent­ly lin­ing up an end-of-phase meet­ing with the FDA.

“It ba­si­cal­ly, for us, val­i­dates the sci­en­tif­ic ap­proach that we cur­rent­ly use on the rest of the port­fo­lio,” Ses­sion said.

The AveX­is and Bridge­Bio vet­er­an found­ed Taysha back in Jan­u­ary 2020 with the help of for­mer AveX­is CEO Sean Nolan. At the time, they want­ed to take what they learned build­ing the first-ever gene ther­a­py for spinal mus­cu­lar at­ro­phy and scale the process. The com­pa­ny launched in April with $30 mil­lion in Se­ries A fund­ing and a pipeline of gene ther­a­pies from UT South­west­ern, which Ses­sion has called an “un­der-rec­og­nized gem.”

Suyash Prasad

A Se­ries B round and $181 mil­lion IPO fol­lowed in Au­gust and Sep­tem­ber, re­spec­tive­ly. CMO and R&D chief Suyash Prasad called it  “three years of fi­nanc­ing in six months.”

“RA and I chat­ted fre­quent­ly dur­ing our ear­ly fi­nanc­ings,” Prasad said. “We just re­al­ized things were go­ing so well af­ter the Se­ries B, we just turned around and said, ‘Let’s go pub­lic.’”

TSHA-120 is now Taysha’s 26th pro­gram. So far, 14 pa­tients have been dosed with the AAV9 can­di­date in an open-la­bel dose-es­ca­la­tion study launched in 2015 by the NIH and the pa­tient ad­vo­ca­cy group Han­nah’s Hope. Re­searchers ob­served a halt in dis­ease pro­gres­sion at the sec­ond-high­est dose lev­el one year post-treat­ment, and six pa­tients have shown dose-de­pen­dent im­prove­ments for more than three years, ac­cord­ing to Taysha.

The biotech has rough­ly 6 months of da­ta on the high­est dose co­hort, and ex­pects to read out more da­ta lat­er this year.

“If we con­tin­ue to see that dose im­prove­ment, who knows what we would ex­pect,” Ses­sion said.

GAN is a rare au­to­so­mal re­ces­sive dis­ease of the cen­tral and pe­riph­er­al ner­vous sys­tems caused by mu­ta­tions in the gene cod­ing for gi­gax­onin. Chil­dren with GAN usu­al­ly show symp­toms be­fore the age of five, in­clud­ing pro­gres­sive sco­l­io­sis, con­trac­tures, and at­ro­phy of the spinal cord. Some pa­tients lose their abil­i­ty to walk, and end up in a wheel­chair. And too of­ten, pa­tients die in their late teens or ear­ly 20s.

There’s cur­rent­ly no treat­ment for GAN, and an es­ti­mat­ed 2,400 pa­tients in the US and Eu­rope.

“When you think about brain dis­eases and dis­eases of the cen­tral ner­vous sys­tem and the pe­riph­er­al ner­vous sys­tem, and in fact, philo­soph­i­cal­ly, for any drug ap­proach — you should re­al­ly tar­get the or­gan that’s mal­func­tion­ing, to try and lim­it any kind of off-tar­get tox­i­c­i­ty,” Prasad said.

That’s why Taysha is dos­ing the can­di­date in­trathe­cal­ly, he said. The process al­so al­lows them to start on the right side of the blood-brain bar­ri­er.

“The biodis­tri­b­u­tion of AAV9 from an IV per­spec­tive, on­ly 1% to 3% of the drug ac­tu­al­ly cross­es in­to the blood-brain bar­ri­er,” Ses­sion said. “And so as Suyash said, we could de­liv­er … a low­er to­tal dose, but put the drug ex­act­ly where it needs to be.”

Taysha is giv­ing Han­nah’s Hope $5.5 mil­lion up­front, plus up to $19.3 mil­lion in mile­stones. The ad­vo­ca­cy group stands to make low sin­gle-dig­it roy­al­ties on net sales.

“With the da­ta that we have in-house to­day, we’re ex­treme­ly ex­cit­ed about what the pos­si­ble reg­u­la­to­ry path­way could look like,” Ses­sion said. “With that be­ing said we need to have the con­ver­sa­tions with the reg­u­la­tors.”

He plans on dis­cussing with reg­u­la­tors in the US, Eu­rope and Japan “as soon as pos­si­ble.”

Up­date: Han­nah’s Hope is el­i­gi­ble for up to $19.3 mil­lion in mile­stones

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

UP­DAT­ED: Bio­gen push­es in a fresh stack of chips and starts prep­ping a glob­al R&D game plan af­ter watch­ing the cards turn on ear­ly throm­bolyt­ic da­ta

After patiently steering through a decade-long journey for its early-stage clinical work, a small Tokyo biotech has clinched a deal to out-license its lead thrombolytic agent to US heavyweight Biogen — which sees a potentially game-changing impact on the clot-busting field after taking a careful look at some upbeat Phase IIa data.

Three years after Biogen anted up $4 million to gain an option on the drug from TMS, the big US biotech is making a small bet to beef up its stroke portfolio. The BD team inked a deal to go ahead and grab rights to the drug for $18 million, with another $335 million in milestone cash on the table for a successful outcome.

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

David Baltimore (Vincent Yu/AP Images)

No­bel lau­re­ate David Bal­ti­more throws his weight be­hind I/O start­up look­ing to craft off-the-shelf CAR ther­a­pies

In the insular world of biotech, names matter — and no names shine brighter than those luminaries with a Nobel Prize on their record. Now, a Los Angeles startup working at the furthest frontiers of cell therapeutics is standing on the shoulders of a laureate of its own and looking to rewrite the fight against solid tumors.

On Tuesday, Appia Bio launched with $52 million in Series A financing led by 8VC and a platform focused on what are called invariant natural killer T cells (iNKT), an extremely rare immune cell the biotech hopes to reengineer to bust malignant tumors through a “best of both worlds” attack plan, the company said.