Mitochondria destruction can help in Cancer Treatment 

Human cells require structures known as mitochondria for the generation of energy for their biochemical activities. Researchers at the Karolinska Institutet in Sweden thought that destroying these could be a strategy for cancer treatment. 

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). In mice, treatment with the drug led to strong anti-tumour responses, decreasing tumour growth without affecting healthy cells, as per reported in Nature by the team.

The data promote further development of the drugs as potential first-in-class mitochondria inhibitors for the cancer treatment, the researchers stated in the paper.

Cancer cells require mitochondria for energy and for producing several building blocks to maintain their wild proliferation.

There had been previous attempts in which mitochondria were targetted for treatment of cancer but often directed to severe side effects because they are also vital for maintaining normal cell functions. The Karolinska-led team focused on a unique feature of cancer cells. As part of uncontrolled cell division, cancer cells must regularly make new mitochondria. Instead of directly targeting existing mitochondria, the team went after mitochondrial DNA transcription that is vital to the formation of new mitochondria and the production of energy.

Atsena raises USD 55 Million financings for blindness gene therapy

University of Florida (UF) spinout Atsena Therapeutics has completed a USD 55 million financing that will benefit 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, which progressively damages the retina, in patients with a mutation in the GUCY2D gene. Atsena took the candidate from Sanofi earlier this year that licensed it from UF.

The Durham, North Carolina-based biotech is aspiring to follow in the footsteps of Spark Therapeutics, whose Luxturna became the first directly administered gene therapy to be sanctioned in the U.S. in 2017.

Luxturna has been authorized for LCA caused by mutations in the RPE65 gene, and another inherited retinal disease called Retinitis Pigmentosa.

Atsena’s GUCY2D therapy, known as SAR439483, emerged from the lab of Atsena co-founder and Chief Scientific Officer Shannon Boye, PhD, and co-founder and Chief Technology Officer Sanford Boye, both are working at UF’s College of Medicine.

The therapy is being administered by subretinal injection to one eye of each patient in the phase 1/2 study with an aim that the untreated eye can serve as a placebo, with a year of follow-up after dosing. Preliminary results are expected in the first half of next year.

Novartis collaborates with neuro partner Cadent for USD 210 Million upfront 

Novartis collaborated with Cadent Therapeutics on treatment-resistant depression back when the neuro-focused biotech was working under the radar. Five years after, the Swiss pharma is backing itself to the rest of Cadent’s pipeline in a contract worth USD 210 million upfront, but that could make the latter up to USD 770 million in whole. 

Novartis will take over all of Cadent’s outstanding stock in exchange for the upfront fee and up to USD 560 million in milestone payments. The duo anticipates the pact to end in the first quarter of 2021.

Under the agreement, Novartis will acquire the milestones and royalties for the depression asset, MIJ821, which is in testing phase 2 study. It also picks up CAD-9303, a schizophrenia treatment, and CAD-1883 that is in development for movement disorders.

As Novartis pushed its depression asset through the clinic, the progress of CAD-9303, Cadent’s schizophrenia program, forced it to turn the partnership into an acquisition, as said by Gopi Shanker, interim co-head of neuroscience at Novartis Institutes for BioMedical Research (NIBR).

Neuron23 reveals with USD 113.5 Million to tackle CNS disorders 

Scientists at Neuron23, since October 2018, have worked to discover their place in the world of precision medicine. That place is utilizing artificial intelligence and data science to aim neurodegenerative and neuroinflammatory diseases in patients with specific genetic mutations.

It was founded through a partnership with Germany’s Origenis, Neuron23 launches USD 113.5 million in financing. The funds comprise USD 30 million in series A funding from Westlake Village BioPartners and Kleiner Perkins and a USD 80 million Series B led by Redmile Group, will drive two to three programs into the clinic in the next two years.

Neuron23’s lead program targets Leucine-rich repeat kinase 2 (LRRK2). It is a gene, which codes for a kinase enzyme of the same name. Mutations in this gene are strongly associated with 3% of patients with Parkinson’s disease. Neuron23 CEO Nancy Stagliano, PhD, said that finding a small-molecule inhibitor for LRRK2, which has an evident genetic linkage to disease, can render a foundation of biological knowledge to treat the other 97% of Parkinson’s patients.

The second program is pursuing brain-penetrant inhibitors for tyrosine kinase 2 (TYK2), a kinase, which leads to multiple sclerosis.