Rare Daily Staff

Orchard Therapeutics, a stem cell gene therapy company focused on developing medicines for rare diseases, completed a $200 million initial public offering.

The London-based company sold 14.3 million shares at $14. Shares are trading on the Nasdaq Global Market under the ticker “ORTX.” The company sold one million more shares than anticipated at the low end of its expected range of $14 to $16. The IPO values the biotech at $1.18 billion.

Shares were trading at $14.49 in early trading.

Orchard had raised about $290 million ahead of the IPO, including completing an oversubscribed series C financing of $150 million in August to advance its gene therapy pipeline.

Orchard develops autologous ex vivo lentiviral gene therapies to treat rare primary immune deficiencies,  neurometabolic disorders, and hemoglobinopathies. Its three most advanced clinical programs are OTL-101 for adenosine deaminase severe combined immunodeficiency (ADA-SCID); OTL-200 for metachromatic leukodystrophy (MLD); and OTL-103 for Wiskott–Aldrich syndrome (WAS).

In April, Orchard acquired GlaxoSmithKline’s portfolio of approved and investigational rare disease gene therapies in exchange for a 19.9 percent stake in the biotech and future milestones and royalties. The portfolio complemented its existing pipeline of gene therapies for primary immune deficiencies and inherited metabolic disorders.

“Acquiring this portfolio further advances Orchard’s vision to be a global, fully integrated company leading the field of gene therapy for rare diseases,” Orchard CEO Mark Rothera said at that time. “The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future.”

The portfolio of gene therapy programs involved in the transaction included: Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016, two late-stage clinical programs in ongoing registrational studies for metachromatic leukodystrophy  and Wiskott Aldrich syndrome, and one clinical program for beta thalassemia.

Orchard also acquired rights to exclusively license three additional preclinical programs from Telethon/Ospedale San Raffaele upon completion of clinical proof of concept studies for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease, and globoid cell leukodystrophy.

October 31, 2018

Photo: Mark Rothera, CEO Orchard Therapeutics