Protocols

Amicus blueprints growth plans for Philly-based gene therapy group; Cash-strapped Compugen restructures, cuts 35 staffers

→ As Roche puts down its foot in Philadelphia as the base for its budding gene therapy operations built around new subsidiary Spark Therapeutics, Amicus $FOLD is blueprinting its own gene therapy group in the city.

Jeff Castelli

Amicus first made its foray into gene therapy via the acquisition of Celenex, which came with 10 programs in neurologic lysosomal storage disorders from Batten disease to Tay Sachs. Shortly thereafter, the rare disease specialist teamed up with James Wilson at the University of Pennsylvania to advance gene therapy treatments for Pompe disease, Fabry disease, CDKL5 deficiency and one other undisclosed rare metabolic disorder.

When completed later this year, its new gene therapy center — which will also be its new global R&D hub — will take up 75,000 square feet in uCity Square, a short walk away from Wilson’s lab. Jeff Castelli, chief portfolio officer and now head of gene therapy, will eventually lead a team of 200 at the facility alongside CSO Hung Do.

→ Another drug developer — Anaveon — is working on an improved version of an IL-2 sans the toxicity that has stymied the use of the original, Proleukin. The Swiss biotech, founded in late 2017 and spun out of the University of Zurich, has won the backing of Syncona and Novartis in a CHF 35 million (roughly $35 million) Series A round, and has big ambitions for its drug, aiming for broad use in oncology: as a cell therapy, vaccine, checkpoint inhibitor and in combination with radiotherapy. Syncona — a UK-based investment firm that counts The Wellcome Trust (also a founder) and Cancer Research UK as its investors — has also taken 47% stake in Anaveon.

→ Strapped for cash to complete an expanded Phase I immuno-oncology program, Compugen is cutting into its 100-person workforce and consolidating all operations in its Israel location. Around 35 employees are being laid off, mostly in R&D and presumably US-based. The decision, which is expected to extend the company’s cash runway through mid-2020 by saving up to $10 million per year, is a result of a strategic review following two discovery partnerships with Bristol-Myers Squibb and AstraZeneca, says CEO Anat Cohen-Dayag.

Purdue Pharma subsidiary Imbrium Therapeutics has enlisted TetraGenetics in a quest to discover and develop non-opioid, ion-channel antibody therapies for chronic pain. Featuring a $25 million upfront and biobucks up to $248 million, the deal comes as Purdue is engulfed in a political storm for its role in the opioid epidemic.

→ An anti-aging startup called Samsara Therapeutics — focused on screening for small molecules that extend healthy lifespan across species — has secured undisclosed seed funding from the aging-focused VC Apollo Ventures. The upstart, which has a partnership with Evotec, has debuted with a paper in Nature, characterizing the life-extending effects of a natural molecule derived from a Japanese herb called ashitaba consumed on the island of Okinawa, which hosts the greatest number of supercentenarians. It is also the first time Apollo has not just provided seed funding to one of its portfolio companies, but is also helping build the company by providing the full scientific team.

Mallinckrodt has inked a research collaboration with Germany’s Transimmune to uncover the mechanism of action and potential applications of photopheresis, the method of treating blood with ultraviolet light that underlies Mallinckrodt’s Therakos platform. With Transimmune’s expertise in immunotherapy, the partners are hoping to find new evidence that photopheresis can work in graft-versus-host disease organ transplant rejection and autoimmune diseases other than cutaneous T- cell lymphoma, for which the treatment is already approved.

→ Having failed to win over investors with its spin on some mid-stage cancer vaccine data and seen its stock hammered in the months since, Sellas is now pleading for help. The review of strategic alternatives, as the company calls it, covers everything from a sale, reverse merger, financing to funded partnership. Meanwhile, Sellas still has a Phase III planned for galinpepimut-S, which it’s also testing in an early trial in combination with Merck’s Keytruda


With contribution by Natalie Grover.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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