Deal of the Year: Orchard Therapeutics

An oversubscribed $110m series B round completed in December 2017 made Orchard Therapeutics, a UK-based genetics spinout from University College London (UCL), an obvious choice for the 2018 Global University Venturing Deal of the Year award.

The series B round was co-led by investment firm Baillie Gifford and venture capital firm Ori Capital, and featured contributions from the university-owned UCL Technology Fund and Singaporean state-owned investment firm Temasek.

Further investors included financial services firm Fidelity International’s investment arm F-Prime Capital, Cowen Healthcare Investments, Juda Capital, Pavilion Capital, RTW Investments, Agent Capital and 4Bio Capital.

Orchard was launched by UCL Business, the institution’s tech transfer office, and F-Prime Capital in May 2016. It is based on research at the university’s Institute of Child Health.

The company specialises in gene-therapy technology which focuses on a condition known as adenosine deaminase severe combined immunodeficiency (Ada-Scid), an inherited immune-system disorder that is both rare and life-threatening.

Ada-Scid is also known as bubble baby disease because sufferers tend to be young and extremely susceptible to infectious diseases, meaning they require sterile environments – a bubble.

The series B round followed Orchard’s $30m series A in May 2016, led by F-Prime Capital with participation from UCL Business, UCL Technology Fund and the EU-owned European Investment Fund.

At the time of the series B round, Orchard’s chief executive Mark Rothera, said: “This financing further empowers Orchard to deliver on our vision to be a leading, global, fully-integrated company that transforms the lives of patients through innovative gene therapy.

“In collaboration with our stakeholders, we are committed to translating our outstanding clinical data into therapies for patients with the greatest need, as quickly as possible. This financing makes meeting this challenge possible.”

The series B round is being used to expedite the global roll-out of Orchard’s OTL-101, an Ada-Scid gene therapy which is the company’s lead product.

Crucially, OTL-101 has been recognised by regulators on both sides of the Atlantic as an important breakthrough over the past 12 months. In July 2017, the therapy was granted a rare paediatric disease (RPD) designation by the Food and Drug Administration (FDA) in the US.

RPD designation is awarded to drugs treating serious or life-threatening diseases that affect fewer than 200,000 patients in the US, but predominantly children. This kind of recognition can be important to researchers because it means their therapy may qualify for a priority review in a marketing application for another drug they subsequently develop.

Meanwhile, in August last year, the UK Medicines and Healthcare Products Regulatory Agency granted OTL-101 a promising innovative medicine designation. This recognises that the therapy is likely to offer significant clinical benefits. It also means the drug should be made available more quickly than might otherwise be the case.

Up to that point, Orchard said, more than 40 Ada-Scid patients had been treated with its gene therapy products at the London-based Great Ormond Street Hospital for children and at University of California Los Angeles. All patients survived the treatment, and the therapy was shown to restore immune functions.