果冻影院

Orchard was founded in 2015 through a partnership between 果冻影院 Business (果冻影院B), the commercialisation company for 果冻影院, and F-Prime Capital Partners. Its revolutionary treatments are the result of decades long gene therapy research and clinical work led by 果冻影院 Professors Bobby Gaspar and Adrian Thrasher, both based at 果冻影院 Great Ormond Street Institute of Child Health.听

The significant deal with Kyowa Kirin will help build on Orchard鈥檚 track record of developing curative treatments for patients with severe and devastating genetic disorders and enable the company to create new drugs for a wider range of conditions.听

Orchard鈥檚 proprietary technology harnesses a patient鈥檚 own genetically modified hematopoietic stem cells (HSCs) to potentially correct the underlying cause of a genetic disease (restore gene function) using a single administration (dose).听

Among the therapies developed by Orchard includes Libmeldy (atidarsagene autotemcel), also known as OTL-200, designed for patients with early-onset metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body鈥檚 metabolic system.听

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 corrects the genetic cause of MLD by inserting functional copies of a faulty gene into the patient鈥檚 stem cells. The stem cells come from their own bone marrow or blood and are fed back into the body with the new genetic information.

Libmeldy is approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of 鈥渓ate infantile鈥� and 鈥渆arly juvenile鈥� 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 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鈥檚 syndrome (MPS-IH) and mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome, respectively.

This acquisition also has the potential to accelerate the development of Orchard鈥檚 next-in-line MPS programmes, as well as its early research programmes, which include a severe, genetic form of Crohn鈥檚 disease and frontotemporal dementia (FTD).听

Simon Goldman, Investment Director, 果冻影院TF, commented on the transaction: 鈥淜yowa Kirin鈥檚 move to acquire Orchard is terrific news for the company, and for the ex vivo lentiviral gene therapy space in general 鈥� 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 果冻影院, which was a critical part of the technology and the company鈥檚 development. As founding investors in Orchard, we鈥檙e immensely pleased and look forward to further positive developments for rare disease patients globally.鈥�

Following Orchard鈥檚 launch in 2015, F-Prime Capital Partners led on a 拢21 million Series A investment into Orchard. 听In 2018, Orchard announced the closing of $225 million through its initial public offering to help transform the lives of patients with serious and life-threatening rare diseases.
The 果冻影院 Technology Fund, managed by AlbionVC in collaboration with 果冻影院B, has also invested in Orchard.

Links

• 果冻影院 Great Ormond Street Institute of Child Health
• 果冻影院 Population Health Sciences

Image

• Credit: Artur Plawgo via

Media contact听

Henry Killworth听

E: h.killworth [at] ucl.ac.uk

Tel: +44 (0) 7881 833274