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Clene Inc. (CLNN) CEO Rob Etherington On Neurodegenerative Disease

Nicole hosts Rob Etherington, the CEO of the clinical-stage biopharmaceutical company Clene (CLNN), to discuss the company’s latest progress on its treatments for Parkinson’s and other neurodegenerative diseases.

GEN

Putting Pressure On Cells to Kill Cancer

Sotio’s cell platform, called DCVAC, starts with a patient’s peripheral blood mononuclear cells, which are grown into dendritic cells (DCs). In parallel, the company uses a proprietary device to pressurize tumor cells from cell lines, and the right amount of HHP makes the cells more immunogenic. The company picks “cell lines with expression profiles that show the most overlap with a patient’s tumor profile,” says Sotio’s CTO, Luděk Sojka, PhD. Then, adding the HHP-killed tumor cells to a patient’s DCs makes them express a variety of tumor antigens on their surface.

endpoints

‘Never been more urgent:’ Scynexis looks to tackle superbug crisis with late-stage readout for antifungal hopeful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

biospace

Pivots During Pandemic Bring Patient-Centric Options to Rare Disease Patients

X4 Pharmaceuticals is developing a class of small molecule therapeutics that work as antagonists of chemokine receptor, CXCR4, the disruption of which is implicated in a number of primary immunodeficiencies (PIs) and cancers.

endpoints

Looking to take advantage of ‘silenced’ enzymes, SalioGen emerges from stealth with eyes set on gene therapy 3.0

That’s the question a new biotech is aiming to answer, as SalioGen Therapeutics emerges from stealth Monday morning with a $20 million Series A. And they believe they’ve found a new delivery system that can more precisely deliver genes in vivo than the relatively large adeno-associated virus or CRISPR system: mammalian-derived enzymes.

The Fight Against Fungi

Cidara Therapeutics is exploring echinocandin derivatives. The company’s clinical candidate rezafungin is a derivative of anidulafungin, a compound originally discovered at Eli Lilly and Company and currently marketed by Pfizer. Chemists placed a choline group at a critical point on the molecule, a feature that stabilizes the echinocandin ring and prevents metabolic breakdown of the molecule, explains Cidara’s president and CEO, Jeff Stein.

Developing Oral Drugs for Neurodegenerative Diseases Based on Bioenergetic Nanotherapeutics with Mark Mortenson Clene Nanomedicine

Mark Mortenson, Chief Science Officer, Clene Nanomedicine is combining physics with biology to develop its bioenergetic nanocatalyst drug candidates that accelerate neurorepair for patients with neurodegenerative disease. Using gold nanocrystals, the company is able to amplify bioenergetic reactions in patients that ultimately drive intracellular biological reactions. The company’s lead candidate, CNM-Au8, is a bioenergetic nanocatalyst powered to support critical intracellular biological reactions that repair and reverse neuronal damage resulting from neurodegenerative disease such as chronic optic neuropathy in patients with multiple sclerosis and the disease progression in patients with ALS.

A three way battle beckons in GM1 gangliosidosis

Sio Gene Therapies’ AXO-AAV-GM1 and Passage Bio’s PBGM01 – are due to yield data in mid-2021, and Lysogene is not too far behind with LYS-GM101. Within the next year there will be a chance to see how the projects stack up against each other in the clinic.

Sio, formerly known as Axovant, is in the lead for now. The group has already reported promising six-month data from the low-dose cohort of its phase I/II trial; notably, clinically meaningful increases have been seen in the enzyme beta-galactosidase, which is deficient in GM1 gangliosidosis.