After cutting a deal earlier this week worth up to $1.1 billion with Crispr Therapeutics AG, it took Vertex Pharmaceuticals Inc. only two days to come up with another collaboration that could top the $1 billion mark, this time with Obsidian Therapeutics Inc. Privately held Obsidian could bring in as much as $1.3 billion if it hits specified research, development, regulatory and commercial milestones in up to five programs in as yet undisclosed indications whose treatments involve the regulating of gene editing. The partnership pairs Obsidian’s Cytodrive technology platform with Vertex’s platform capabilities in small-molecule, cell and genetic therapies. It’s a platform-to-platform deal that Paul Wotton, Obsidian’s CEO, told BioWorld is an unusual business deal structure that, in this case, could be applied to a broad area of therapeutics.

Foreign interference in U.S. biomedical research a costly enterprise

Last year, the NIH identified 507 grant recipients who potentially had undisclosed conflicts of interest, with many of those conflicts involving affiliations with foreign governments. While that number represents less than 2% of the more than 30,000 NIH grant recipients, Sen. Patty Murray (D-Wash.) said, “We cannot let the few instances of bad actors undermine the U.S biomedical research enterprise, including our ability to partner with talented researchers around the globe.” In opening a Senate Health, Education, Labor and Pensions Committee hearing April 22, Murray stressed what’s at stake. Besides the abuse of billions of dollars in U.S. tax dollars that fund the NIH grants, life-saving research could be delayed or derailed by undue foreign influence, she said.

ITM raises the roof with $108M convertible loan for radiopharmaceutical pipeline

DUBLIN – Isotopen Technologien München (ITM) AG raised €90 million ($108 million) in debt financing to complete phase III development of its lead radiopharmaceutical, solucin (no-carrier-added (nca) Lutetium-177 edotreotide (177Lu-edotreotide)), in gastroenteropancreatic-neuroendocrine tumors (GEP-NETs) and to take forward several more preclinical pipeline projects.

Intellia TTR amyloidosis phase I findings ahead in busy genome-editing sector

The genome-editing space continues to heat up, with Beam Therapeutics Inc. publishing in The CRISPR Journal details of its work with inlaid base editors (IBEs). Beam is applying IBEs in the BEAM-102 program for sickle cell disease. Meanwhile, Wall Street eagerly awaits data from another company in a separate indication: Intellia Therapeutics Inc. is due to roll out first-in-human data soon with a systemic CRISPR-based genome editing therapy, NTLA-2001, in ATTR amyloidosis.in transthyretin amyloidosis. Intellia, too, recently offered the first preclinical data set on its cytosine deaminase base editor technology at the Cold Spring Harbor Laboratory virtual scientific meeting on nucleic acid therapies.

Anavo Therapeutics closes $24M seed round to drug the phosphatome

DUBLIN – Anavo Therapeutics BV raised €20 million (US$24 million) in seed financing to fund the build-out of a platform that aims to drug the human phosphatome, or the ensemble of phosphatase enzymes that plays a crucial role in maintaining cells’ phosphorylation balance by removing phosphate groups from their target substrates. Phosphatases have, for the most part, been neglected as drug targets, even though they represent, in terms of cell signaling, the obverse of kinases, which have proven to be an enormously rich source of drug targets in the last two decades.

Newco news: Stuart Therapeutics raises $11M to back topical dry eye disease candidate

Stuart Therapeutics Inc., a company developing peptide therapeutics for ophthalmic disease, has completed an $11 million series A financing led by Infocus Capital Partners, an ophthalmology-focused life sciences venture capital fund, with significant participation by the Wisconsin Alumni Research Foundation, Mimo Capital and Biobrit. Proceeds from the round will fund phase II trials for the company's investigational fast-acting dry eye disease candidate, ST-100.

PCSK9 affects antitumor immunity

The enzyme proprotein convertase subtilisin/kexin type 9 (PCSK9) plays a major role in the regulation of blood LDL cholesterol levels. Now, new research shows that targeting PCSK9 may also potentiate checkpoint blockade.

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