‘Deprioritized’ gene therapy given as treatment for Batten disease

Child gets TSHA-118 in individual-patient study at Chicago medical center

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Note: This story was updated March 5, 2024, to clarify that Taysha Gene Therapies set aside the development of TSHA-118, along with other experimental gene therapies, to focus on another treatment for a different disorder.

A patient with CLN1 disease, also known as infantile Batten disease, has received treatment with the “deprioritized” gene therapy TSHA-118 as part of an individual-patient, investigator-initiated study at Rush University Medical Center in Chicago.

This treatment for Batten disease is no longer in active development. After its ownership changed hands a few times — most recently in 2020, when it was acquired by Taysha Gene Therapies — TSHA-118 was set aside by the company, along with a few other experimental gene therapies, in favor of advancing another treatment for a different disorder.

Now, thanks to Elizabeth Berry-Kravis, MD, PhD, a professor at Rush, the gene therapy is being given the opportunity to help a single child with CLN1.

“As a physician who treats patients with this devastating disease, I am pleased to have helped enable the dosing of an individual patient with CLN1 disease with investigational gene therapy candidate TSHA-118,” Berry-Kravis, the principal investigator and sponsor of the study, said in a joint statement from several Batten-focused organizations.

“I remain committed to helping advance care for patients and families impacted by CLN1 disease,” Berry-Kravis added.

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This specific disease type, called infantile Batten, is caused by mutations in the CLN1 gene. TSHA-118 was designed to deliver a healthy version of this gene to cells in the brain using a harmless viral vector.

The gene therapy’s development was funded in part by Taylor’s Tale, a nonprofit organization founded by the family of CLN1 patient Taylor King, who died in 2018.

In the joint statement, Taylor’s mother Sharon King, who serves as president of Taylor’s Tale, and Taylor’s sister Laura King Edwards, the vice president, said they’ll “continue to advocate so that others may benefit in the future” from this gene therapy.

“More than a decade ago, Taylor’s Tale and our dedicated volunteers and donors dared to believe in a novel gene therapy approach for CLN1 disease, catalyzing the study that led to today’s exciting announcement. While the treatment came too late to save Taylor’s life, it stands as her lasting legacy,” the women said, adding they were “thrilled for this patient and their family.”

TSHA-118 was originally developed in a laboratory, ultimately landing with Taysha.

The company developed the gene therapy for a few years, even securing clearance to start clinical testing from the Canadian government in late 2021. But in 2022, the company announced it was deprioritizing TSHA-118 and a few other experimental gene therapies to focus resources on advancing its lead therapy candidate, TSHA-102. That gene therapy for Rett syndrome is now in clinical testing.

We are pleased that we can ensure these programs are provided to the right advocates, clinicians and scientific experts who can potentially move these programs forward for the benefit of patients.

Taysha announced in a statement that it had provided clinical material to support the investigator-initiated study at Rush. Further, the company said it’s on the lookout for other potential partnerships and opportunities to further develop TSHA-118 and its other deprioritized therapy candidates.

“As we continue to focus on advancing our lead TSHA-102 program for the treatment of Rett syndrome, we are pleased that we can ensure these programs are provided to the right advocates, clinicians and scientific experts who can potentially move these programs forward for the benefit of patients,” said Sean P. Nolan, Taysha’s chairman and CEO.