Kyowa Kirin to acquire rights to OTL-201 for Sanfilippo syndrome type A

The experimental gene therapy uses patients' own hematopoietic stem cells

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Note: This story was updated Dec. 11, 2023, to correct that Kyowa Kirin has entered into an agreement to acquire Orchard Therapeutics, which will be finalized next year. The story also corrected that OTL-200, Libmeldy, is under priority review by the U.S. Food and Drug Administration with a review date of March 18.

Kyowa Kirin’s portfolio will include OTL-201, a gene therapy for Sanfilippo syndrome type A, after the Japanese pharmaceutical company announced the planned acquisition of Orchard Therapeutics.

The deal is expected to be finalized early next year.

“This is an exciting opportunity designed to accelerate the realization of our shared vision of ending the devastation caused by severe genetic diseases and deliver life-changing value in medical care,” Bobby Gaspar, MD, PhD, co-founder and CEO of Orchard Therapeutics, said in a press release.

People with Sanfilippo type A, also known as mucopolysaccharidosis type IIIA (MPSIIIA), carry mutations in the SGSH gene. This gene contains the instructions for an enzyme called sulfamidase that helps break down a large sugar molecule called heparan sulfate.

Lack of the enzyme leads to a toxic accumulation of heparan sulfate and other long sugar molecules inside cells of the brain and other tissues, eventually resulting in cell death. This leads to delays in development, behavior challenges, and impaired cognition.

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Therapy uses patients’ own hematopoietic stem cells that are genetically modified

OTL-201 is one of several gene therapies developed by Orchard Therapeutics that use the patient’s own blood-forming stem cells, also known as hematopoietic stem cells (HSCs), as a treatment for genetic disorders. In this case, after the cells are harvested from a patient, they are genetically modified in the lab to carry a working version of SGSH gene. The modified stem cells are then infused back to the patient to rescue the production of the missing enzyme.

With the acquisition, Kyowa Kirin expects to accelerate the development of Orchard Therapeutics’ gene therapy program for mucopolysaccharidoses, as well as its early research programs for other diseases.

“We are excited to announce that we have signed the Transaction Agreement to acquire Orchard Therapeutics, one of the leading providers of HSC gene therapy,” said Takeyoshi Yamashita, PhD, director of the board, chief medical officer, senior managing executive officer of Kyowa Kirin. “With this transaction, we anticipate being able to use a new modality that can have a profound impact on patients’ lives.”

OTL-201 is being tested in an ongoing Phase 1/2 trial (NCT04201405), which has enrolled five children with Sanfilippo type A. The trial, which enrolled its first patient in April 2020, intends to evaluate the safety and tolerability of OTL-201 and its effectiveness in promoting the increase of sulfamidase.

Recent study results have found that after median of two years following the gene therapy infusion, the majority of children showed improvements in cognitive skills, with three children showing normal cognitive development.  During this period, no serious side effects have been reported, and the gene therapy has generally been well tolerated.

The results so far confirmed an increase in sulfamidase to levels higher than those normally found in the blood and cerebrospinal fluid (the fluid that surrounds the brain and spinal cord) of healthy children.

“We remain as true to our mission as ever, and joining Kyowa Kirin’s global network ensures we are well-resourced to progress […], capitalize on opportunities for global expansion, as well as advance our next-in-line neurometabolic programs in MPS disorders and earlier-stage research programs. We look forward to collaborating with our new colleagues at Kyowa Kirin to fully unlock the curative potential of HSC gene therapy for the benefit of patients and society,” Gaspar said.

Orchard Therapeutics platform also includes OTL-203, an investigational gene therapy for Hurler’s syndrome, also known as mucopolysaccharidosis type I. Another Orchard HSC-based gene therapy, OTL-200, marketed as Libmeldy (atidarsagene autotemcel), is approved in the U.K. and in the European Union for the treatment of patients with early-onset metachromatic leukodystrophy (MLD), a rare genetic disorder characterized by accumulation of certain fat molecules, particularly in cells of the nervous system. In the U.S., it is currently under priority review, with a decision by the U.S. Food and Drug Administration expected March 18.