UK biotech Orchard plans $172.5m US IPO to fund rare disease R&D

UK biotech Orchard Therapeutics is planning a US IPO to expand use of its Strimvelis gene therapy, and further develop its rare disease pipeline.

A filing with the US Securities and Exchange Commission shows the biotech wants to raise $172.5 million to make Strimvelis available to more patients with adenosine deaminase-severe combined immunodeficiency (ADA-SCID) – also known as ‘bubble boy disease’

The UK biotech acquired Strimvelis from GlaxoSmithKline earlier this year, along with a pipeline of other gene therapies.

It already raised $150 million from a round of private funding in August but wants more cash as it chases blockbuster-level revenues from its maturing pipeline of rare disease drugs.

As part of the deal GSK took a 20% stake in the company and said that Orchard must make Strimvelis available to more patients until an alternative gene therapy such as its own OTL-101 becomes available.

The European Medicines Agency has designated its OTL-300 for beta thalassaemia for its Priority Medicine (PRIME) scheme, which accelerates development of promising new therapies for serious diseases.

Also in the pipeline is OTL-200 for metachromatic leukodystrophy, a rare genetic disorder that affects lipid metabolism.

It is also developing OTL-103, a rare immune condition that stems from a reduced ability to form blood clots, and abnormal or non-functional white blood cells.

Orchard said it plans to file all three of these pipeline drugs in the “near term”.

Orchard’s technology works by harvesting a patient’s own stem cells, and modifying them to produce a functional copy of a missing or faulty gene in the lab.

This results in a drug that can be reintroduced into the patient, which corrects the genetic problem causing the disease.

The biotech said the technology has the potential to be used in a range of other diseases, adding that annual revenues from its five lead drugs could exceed $2 billion annually.