Orchard Therapeutics, a gene therapy company developing and commercialising medicines for rare diseases, is set to be acquired by Japanese pharmaceutical firm Kyowa Kirin for $477.6m, with a focus on creating new drugs.

Orchard Therapeutics’ technology harnesses a patient’s own genetically modified hematopoietic stem cells (HSCs) to potentially correct the underlying cause of a genetic disease using a single administration. It was founded in 2015 through a partnership between UCL Business (UCLB), the commercialisation company for UCL, and F-Prime Capital Partners. Its revolutionary treatments are the result of decades long gene therapy research and clinical work led by UCL Professors Bobby Gaspar and Adrian Thrasher, both based at UCL Great Ormond Street Institute of Child Health.

Orchard Therapeutics’ portfolio comprises Libmeldy (atidarsagene autotemcel), also known as OTL-200, intended for eligible patients with early-onset metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system.

In the most severe form of MLD, babies develop normally, but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them.

Libmeldy is approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of “late infantile” and “early juvenile” MLD patients. It is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) goal date of 18th March, 2024.

Using the same HSC gene therapy technology platform, Orchard Therapeutics is progressing two clinical-stage programmes known as OTL-203 and OTL-201 for the treatment of another group of severe paediatric neurometabolic disorders: mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome, respectively.

This acquisition will also allow Kyowa Kirin to accelerate the development of Orchard Therapeutics’ next-in-line MPS programmes, as well as its early research programmes, which include a severe, genetic form of Crohn’s disease and frontotemporal dementia (FTD).

“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, Ph.D., 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. Orchard Therapeutics is a company with a steady track record in this field and has already launched its HSC gene therapy in Europe and filed for review in the U.S. Our hope is to combine the strengths of Kyowa Kirin and Orchard Therapeutics with mutual respect to realize the successful creation and delivery of life-changing value for patients living with rare and life-threatening inherited diseases.”

“This is an exciting opportunity designed to accelerate the realisation of our shared vision of ending the devastation caused by severe genetic diseases and deliver life-changing value in medical care,” said Bobby Gaspar, co-founder and chief executive officer of Orchard Therapeutics. “We remain as true to our mission as ever, and joining Kyowa Kirin’s global network ensures we are well-resourced to progress anticipated commercialization of OTL-200 in the U.S., if approved, continue investing in initiatives aimed at accelerating Libmeldy growth in Europe, capitalise on opportunities for global expansion, as well as advance our next-in-line neurometabolic programmes in MPS disorders and earlier-stage research programmes. 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.”

Simon Goldman, Investment Director, UCLTF, added: “Kyowa Kirin’s move to acquire Orchard is terrific news for the company, and for the ex vivo lentiviral gene therapy space in general – which remains one of the safest and most efficacious gene therapy modalities. This is also a great outcome and validation of the innovation ecosystem at UCL, which was a critical part of the technology and the company’s development. As founding investors in Orchard, we’re immensely pleased and look forward to further positive developments for rare disease patients globally.”

Find out more.

• Share