5:47 PM
 | 
Jul 26, 2018
 |  BC Innovations  |  Product R&D

Syncona’s sister act

How Syncona's family of five gene therapy portfolio companies share recipes

With the final pieces of its gene therapy portfolio in place, Syncona Investment Management Ltd. thinks its five “sister companies” are poised to capitalize on recent regulatory and technological advances in gene therapy, and push the modality beyond rare diseases.

The firm designed its portfolio to find low-hanging fruit in gene therapy’s most accessible target organs, and to address the modality’s two biggest challenges -- manufacturing and delivery.

“We set up a road map to build essentially three therapeutics companies -- one in the eye, one in the liver, and one in the central nervous system -- and acquire or build two platforms that we think will be strategic,” said Christopher Hollowood, chief investment officer and managing partner at Syncona. “If you did that, you’d have an encompassing set of companies that cover basically all of the opportunity set for gene therapy.”

The June debuts of CNS therapeutics company SwanBio Therapeutics Ltd. and ophthalmic delivery play Orbit Biomedical Inc. marked the completion of Syncona’s strategy, which the firm mapped out in 2012 and 2013 after launching with £200 million ($263 million) from Wellcome Trust. In 2012, Wellcome added £50 million ($65.7 million) to the fund.

At the time, the company was called Syncona Partners LLP. In 2016, it was acquired by cancer-focused investment firm BACIT Ltd. to form a publicly traded life sciences investment company under the name Syncona, which has a market cap of £1.7 billion ($2.2 billion).

"If you did that, you'd have an encompassing set of companies that cover basically all of the opportunity set for gene therapy."

Christopher Hollowood, Syncona

Over the last six years, Syncona has invested more than £130 million ($171 million) building the five companies.

Its first, now most advanced, gene therapy investment was retinal disease company Nightstar Therapeutics plc, which received $106.8 million in venture funding from Syncona and its syndicate partners, and raised $86.3 million through its 2017 IPO. Nightstar’s lead program is in Phase III testing for choroideremia, and is followed by a Phase I/II-stage program in X-linked retinitis pigmentosa and two preclinical programs for rare retinal diseases.

The remaining four companies are privately held. Systemic disease play Freeline Therapeutics Ltd., whose lead program is in Phase I/II testing for hemophilia B, received a $37.7 million series A and a tranched $117 million series B from Syncona. Preclinical newcos SwanBio and Orbit received $25 million and $12 million series A rounds, respectively. Seed funding for Gyroscope Therapeutics Ltd., which has a program in preclinical development for dry age-related macular degeneration (AMD), is undisclosed (see “Five Sisters”).

Table: Five sisters

Syncona Investment Management Ltd. (LSE:SYNC) has founded five gene therapy companies since 2014. The companies are focused on the three tissues Syncona believes hold the most promise for gene therapy -- the eye, the liver and the CNS. Freeline Therapeutics Ltd. and Orbit Biomedical Inc. have respectively in-licensed IP covering gene therapy manufacturing and delivery technologies that are relevant to the other gene therapy...

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