(Mathew Schwartz/Unsplash)(Mathew Schwartz / Unsplash)
Mereo BioPharma is teaming up with Ultragenyx to work on its rare genetic bone disease drug.
Worth $50 million upfront and with modest biobucks of up to $254 million on the table, the R&D collab and licensing deal focuses on setrusumab in osteogenesis imperfecta (OI), a rare genetic disorder marked by fragile bones and reduced bone mass resulting in bones that break easily, loose joints and weakened teeth.
Setrusumab works as a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells. It is currently in mid- to late-stage testing.
(YaroslavKryuchka/Getty Images)
University of Florida (UF) spinout Atsena Therapeutics has closed a $55 million financing that will help take its gene therapy for a common cause of blindness in children into pivotal trials.
The gene therapy is already in a phase 1/2 trial for Leber congenital amaurosis (LCA) a disease that progressively destroys the retina in patients with a mutation in the GUCY2D gene. Atsena acquired the candidate from Sanofi earlier this year, which in turn licensed it from UF.
The Durham, North Carolina-based biotech is hoping to follow in the footsteps of Spark Therapeutics (now part of Roche), whose Luxturna became the first directly administered gene therapy to be approved in the U.S. in 2017.
Dec 16, 2020 12:54pm Scientists at the Karolinska Institutet designed small-molecule inhibitors (green) that target the expression of mitochondrial DNA to cause a cellular energy crisis in cancer cells to stop their growth. (Mattias Karlén)
Human cells need structures called mitochondria to generate energy for their biochemical activities and cancer cells are no exception. Researchers at the Karolinska Institutet in Sweden figured that crippling these little power plants could be a strategy for treating cancer.
Following that hypothesis, the scientists designed oral inhibitors that could target mitochondrial DNA (mtDNA). In mice, treatment with the drug led to strong anti-tumor responses, slowing tumor growth without affecting healthy cells, the team reported in Nature.
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Britain-based biopharma services company Ergomed has snapped up U.S. contract research organization MedSource to boost its offerings and geographic footprint.
Ergomed will add the Houston-based CRO into its business, boosting its rare disease and oncology services, as well around $20 million a year in contracts and revenue, with more than $41 million in order book future revenue, and new clients.
The deal, financials of which were not made public, will also “further accelerate Ergomed’s growth in the North American market,” according to a statement, with offices in Houston, Boston and Raleigh, North Carolina, in the U.S. and Newcastle upon Tyne in the U.K.
(Bioclinica)
Clinical trial health tech firm eResearchTechnology (ERT) is trying to round off a tough year with a new deal by merging with Bioclinica.
The deal, financials of which were not made public, will integrate Bioclinica s imaging focus with ERT s expertise in eCOA, cardiac safety, respiratory and wearables.
“Leveraging a unique footprint, the combined company will deliver data analytics, insights, business intelligence, virtual patient visits and hybrid technological solutions as a strategic partner to global pharmaceutical and biotechnology companies, delivering best-in-class clinical research tools, expertise and technologies to partners and patients alike across all therapeutic areas,” the pair said in a statement.