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biospace

Study: Drug Candidate Improves Brain Energy Metabolism in Parkinson’s, MS

CNM-Au8, Clene’s lead drug candidate, is an aqueous suspension comprising catalytically active, clean-surfaced and faceted gold nanocrystals. The company explained in a statement that the therapy’s catalytically active nanocrystals fuel “critical cellular energy” that ultimately produce brain reactions enabling neurorepair and remyelination. This is reportedly accomplished via increases in neuronal and glial resilience to stressors relevant to neurologic diseases.

Fierce-Biotech

PepGen raises $113M to challenge Sarepta in DMD, build neuromuscular and neurologic pipeline

Boston-based PepGen’s lead candidate is a therapeutic oligonucleotide that targets exon 51 to treat a subset of DMD patients, putting the biotech squarely on the turf of Sarepta. Like Exondys 51, the drug is designed to mask exon 51 and thereby enable patients to produce the functional dystrophin needed to strengthen muscle fibers.

targeted oncology

Clinical Potential of onCARlytics Under Exploration in Solid Tumors

“We believe the synergy between Celularity’s placental-derived cells and our OnCARlytic platform has the potential to shift the cellular medicine paradigm,” Leslie Chong, managing director and chief executive officer of Imugene, stated in a press release.

Arrowhead Pharmaceuticals – New Positive Patient-Level Data In AATD Liver Disease

Dr. Chris Anzalone, PhD, President and CEO of Arrowhead Pharmaceuticals discusses new positive patient-level data from their AROAAT2002 global Phase 2 study of ARO-AAT, an RNA therapeutic being co-developed by the company and Takeda, in patients with liver disease caused by alpha-1 antitrypsin deficiency (AATD).   He also talks about the company’s RNAi platform.

endpoints

Shares of BeyondSpring skyrocket on new, positive cancer drug trial results

On Wednesday, BeyondSpring, a small New York biotech with an offbeat approach to immunotherapy, announced its lead drug significantly extended non-small cell lung cancer patients’ lives in a large trial. Although the company did not release the exact survival data, it said that nearly twice as many patients were alive after two years on the drug arm than on the standard-of-care arm.

BioProcessOnline

Intranasal Prophylactics For Flu & Coronavirus With Leyden Labs’ Koenraad Wiedhaup

As Leyden Labs Founder & CEO Koenraad Wiedhaup puts it, we breathe in galaxies of viruses on a daily basis. As those viruses multiply and mutate, our approach to mitigating their risks is largely reactive.

Austin company works to launch COVID-19 antibody treatment in new inhaler form

Dale Christensen, with Austin-based drug developer TFF Pharmaceuticals, says they’ve got an easy solution to getting a monoclonal antibody treatment straight to your doorstep — rather than being hooked up to a machine and IV.

biospace

Emerging Drug-Resistant Fungi Threat Offset by Cidara’s Phase III Antifungal Therapy

The cure for this new threat may rest with Cidara Therapeutics’ new antifungal drug, rezafungin. It currently is completing two Phase III trials. One treats candidemia and invasive candidiasis. On the other, rezafungin is used as a prophylactic for the prevention of fungal infections in a high-risk hematology setting. It shows efficacy against Candida spp.Aspergillus spp.Pneumocystis spp. and dermatophytes, can be administered safely at high doses, and it can clear the body of infection quickly.