fiercebiotechNovember 02, 2018
Tag: rare diseases , gene therapy , GlaxoSmithKline
Orchard Therapeutics priced its IPO at $14 a share, raising $200 million and exceeding the $173 million goal it set earlier this month. The funds will bankroll the pivotal trials and regulatory filings for three gene therapies in the U.S. and Europe.
The company hopes to get its three lead assets through regulatory submission:
OTL-101, a treatment for ADA-SCID, also known as "bubble boy syndrome."
OTL-103, for Wiskott-Aldrich syndrome, a disease with immunological deficiency and reduced ability to form blood clots.
OTL-200, for the lysosomal storage disorder metachromatic leukodystrophy (MLD).
The IPO haul will also advance OTL-102, for X-linked chronic granulomatous disease through proof of concept, the company said in an SEC filing earlier this month. Orchard’s earlier-stage programs in Sanfillipo syndrome and beta thalassemia, as well as some preclinical programs, will benefit too.
While Orchard has a clutch of late-stage pipeline hopefuls, the biotech became a commercial-stage company when it acquired GlaxoSmithKline’s rare disease gene therapies. These included Strimvelis, another ADA-SCID treatment that has been approved in the European Union, as well as late-stage assets for MLD and WAS and the program in beta thalassemia.
RELATED: Orchard files for $173M IPO to run pivotal gene therapy trials
The company, based in Boston and London, started out in 2016 with a $33 million series A and picked up another $110 million in its series B round last December. Shortly after the GSK deal, Orchard raised another $150 million in an oversubscribed series C. Add to that the $200 million IPO and Orchard has considerably deep pockets to advance multiple assets.
If all goes according to plan, Orchard will close out 2021 with four approved gene therapies, including Strimvelis. While the "bubble boy" drug has been sold in Europe since 2016, it has had a slow commercial start and could be supplanted by OTL-101.
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