$225million investment for gene therapy spinout Orchard Therapeutics

Orchard Therapeutics, has launched is initial public offering (IPO) on the Nasdaq stock exchange. The company, which specialises in developing gene therapies for complex rare diseases, sold 14.3 million shares to raise $225 million of investment.

Founded in 2015 based on the pioneering work of NIHR GOSH Biomedical Research Centre (BRC) researchers Professor Bobby Gaspar, Adrian Thrasher, Orchard Therapeutics aims to develop transformative gene therapies for immune system and metabolic disorders such as severe combined immunodeficiency (SCID), Chronic Granulomatous Disease, and metachromatic leukodystrophy (MLD). The company uses cutting-edge technology to modify a patient’s own stem cells with a copy of the missing or faulty gene, before transplanting the cells back into the body. This avoids the need for a matching stem cell donor and issues with rejection.

These technologies were developed at GOSH and the UCL Great Ormond Street Institute of Child Health (ICH) over many years with the support of the NIHR GOSH BRC and other funders and international partners including Manchester, Boston Children's Hospital and UCLA. Orchard’s latest success illustrates the impact of GOSH BRC support in helping bring new innovative medicines closer to patients who need them.

“Rare diseases like the one’s Orchard were established to develop treatments for, are often underfunded. This demonstrates serious commitment to changing that with the ultimate goal of improving the lives of very sick adults and children. I am extremely proud that Orchard has come so far in only three years,” said Professor Thrasher.

Prior to the IPO, Orchard Therapeutics raised $152 million in Series C venture capital funding, and $110million from a series B round, which will help bring its gene therapies from clinical testing through to regulatory approval. In April, Orchard announced a strategic agreement with Glaxo SmithKline (GSK) under which GSK will transfer its portfolio of rare disease gene therapies to Orchard, including Strimvelis, the first gene therapy for a rare form of SCID (ADA-SCID) that has been licenced in Europe.