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

GSK has landed a new first from UNICEF — the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”