The group says it will use the cash to progress towards registration of its three most advanced clinical programmes: OTL-101 for adenosine deaminase severe combined immunodeficiency; OTL-200 for metachromatic leukodystrophy; and OTL-103 for Wiskott–Aldrich syndrome (WAS).

The funds will also be used to support the clinical and preclinical development of the Orchard’s rare disease gene therapy pipeline, the firm noted.

"We are thrilled to have such strong support from both new and existing investors in this financing round," said Mark Rothera, Orchard’s president and chief executive.

"This financing provides Orchard with additional capital to rapidly progress our most advanced clinical programs to commercialisation. We are advancing our pipeline of potentially transformative gene therapies in primary immune deficiencies and neurometabolic disorders to reach patients as quickly as possible," added Frank Thomas, the firm’s chief financial and chief business officer.

Back in April the group bought GlaxoSmithKline’s portfolio of approved and investigational rare disease gene therapies, giving the drug giant a 19.9 percent equity stake along with a seat on Orchard’s board.

The programmes acquired by Orchard include Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved in Europe in 2016, and two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy and Wiskott Aldrich syndrome, and one clinical programme for beta thalassaemia.

-------------------------------------------------------------------------------------------------------------------------------

Editor's Note:

To apply for becoming a contributor of en-CPhI.cn,

welcome to send your CV and sample works to us,

Email: Julia.Zhang@ubmsinoexpo.com.