CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push. On the deal front, venture-backed startup Scribe Therapeutics Inc. landed a $25 million up-front payment and a chance at more than $1 billion in milestones by granting Sanofi SA nonexclusive license to genome-editing technology for multiple oncology targets. Meanwhile, Vertex Pharmaceutical Inc. said it will soon submit a rolling BLA to the U.S. FDA for exagamglogene autotemcel, a CRISPR/Cas9 gene-edited therapy in-licensed from CRISPR Therapeutics AG for the potential treatment of patients with sickle cell disease and transfusion-dependent beta thalassemia, adding to plans for an EMA filing by the end of 2022.

Biogen to pay $900M to end whistleblower suit

While it continues to deny all kickback allegations raised in a whistleblower suit filed seven years ago, Biogen Inc. agreed yesterday to pay $900 million to resolve claims that it paid doctors in the U.S. to prescribe its multiple sclerosis drugs from 2009 through March 2014. Under the terms of the settlement, the Cambridge, Mass.-based company will pay nearly $844 million to the U.S. government and more than $56 million to 15 states. About 30% of the federal part of the settlement will be awarded to former Biogen employee Michael Bawduniak, who filed the qui tam suit against the company.

Avidity searches for cause of SAE as FDA puts partial hold on dystrophy study

A serious adverse event in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s phase I/II study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1. The partial clinical hold prevents new participants being enrolled in the study. The adverse event occurred in a patient in the study’s 4-mg/kg cohort. While Avidity conducts an analysis of the event, everyone else in the study is continuing in their dosing cohort. Avidity’s stock (NASDAQ:RNA) was taking a powerful sideswipe at midday, with shares sagging 15% at $15.85 each.

FDA gives thumbs up to Santen, UBE’s Omlonti for reducing intraocular pressure 

The U.S. FDA approved Santen Pharmaceutical Co. Ltd.’s and UBE Industries Ltd.’s Omlonti (omidenepag isopropyl) ophthalmic solution for reducing elevated intraocular pressure in patients with primary open-angle glaucoma or ocular hypertension. It is the second FDA-approved product from Japan-based Santen in the last 15 months for patients in the U.S. with vision conditions.

Huisheng raises ¥500M in series A round for diabetes drugs

Jilin Huisheng Biopharmaceutical Co. Ltd., a non-wholly owned subsidiary of Sihuan Pharmaceutical Holdings Group Ltd., has raised ¥500 million (US$70 million) in a series A round. Huisheng, which has developed a pipeline of programs addressing both diabetes and its complications, plans to use the new funds to speed up its clinical trials and purchase manufacturing equipment, according to Che Fengsheng, executive director and chairman of Sihuan Pharma.

Clinical data slow during summer months

The amount of clinical news in 2022 continues to lag last year, partly due to a decline in COVID-19-related data. Through the last week of September, there have been a total of 2,495 phase I, II and III clinical entries, compared with 3,027 during the first three quarters of 2021. This represents a drop in activity of 17.6%.

The BioWorld Insider Podcast – Psychedelic evolution: Mindset Pharma looks to change mental health treatment

An analyst recently observed that mental health treatments are stuck where cancer was 50 years ago. However, there have been major advancements in developing psychedelic medicines to address that problem. BioWorld staff writer Lee Landenberger talked with James Lanthier, the CEO of Mindset Pharma, which is developing what it calls “next-generation” psychedelic medicines to treat neurological and psychiatric disorders. Lanthier discussed challenges facing companies developing psychedelic treatments and how to overcome the one-size-fits-all, trial-and-error approach to mental health treatment that often seems stuck in the past.

Also in the news

AC Immune, Amerisourcebergen, Ammax, Anebulo, Aqualung, Arcutis, Arsenal, Atamyo, Atea, Bioheng, Biolinerx, Biontech, Brii, Catalym, Cytrx, Dystrogen, Exicure, Everest, Ferring, Imunon, Jasper, Lipocine, Mannin, Moonlake, Navigo, Novavax, Outlook, Pfizer, Prime Medicine, Pulmatrix, Sanity Group, Sciwind, Seagen, Siga, Sorrento, Synerk, Tavros, Tilt, Tolerogenixx, Y-Mabs, Zai Lab