After long years of endeavor, Scarlet Therapeutics Ltd. believes it is poised to realize the potential of red blood cells (RBCs) as drug delivery vehicles. The company recently closed a seed round and is now laying the ground for its first clinical trials in which cultured and genetically engineered RBCs will be used to deliver enzyme replacement therapies in the treatment of rare inherited forms of two metabolic disorders.

Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons. The ion channels will then be selectively controlled by an activating drug, which is taken orally.

Regulatory T-cell specialist Dualyx NV has closed a €40 million (US$43.5 million) series A to progress the lead autoimmune disease program to the clinic and to take forward two other Treg-based therapies. The company brings together expertise in antibody design with understanding of the role Tregs play in supressing the immune response to maintain homeostasis and self-tolerance, preventing autoimmunity.

Nido Biosciences Inc. emerged from stealth by unveiling $109 million in series A and B equity funding and a clinical-stage program in spinal and bulbar muscular atrophy.

Swiss biopharma startup Aphaia Pharma AG is taking the concept of “location, location, location” to its extreme. The company started dosing patients in a phase II trial of its lead candidate, Aph-012, in late April, 2023. The trial is a randomized, double-blind, placebo-controlled, multicenter proof-of-concept study to evaluate Aph-012’s ability to improve glucose tolerance in individuals with prediabetes, as measured by a pathological oral glucose tolerance test. In another phase II trial, Aph-012 is being tested as a weight loss drug for individuals with a BMI between 30 and 40. Aph-012’s active ingredient? Glucose. But delivered exactly to the right place.

The idea for a new company, Ten63 Therapeutics Inc., started in 2015, when Gilda Szacher Frenkel passed away at age 62 from pancreatic cancer. After sequencing her tumor, her son Marcel discovered that her cancer was driven by mutations to key proteins that regulate cellular processes – all the “usual suspects,” he said. At the time, he remembers they were initially encouraged by the discovery and excited to search for developed drugs that could help her. “Here was this blueprint telling us what was wrong,” Marcel Frenkel said, “but, unfortunately, those mutations were unactionable. There were no drugs to modulate the main oncologic drivers.”

Integrated Biosciences Inc., an early-stage startup that is combining synthetic biology and machine learning in the hunt for drugs that tackle cell senescence, has demonstrated its capabilities in a newly published study in Nature Aging on May 4, 2023, which employed artificial intelligence to identify three novel compounds that are highly selective for Bcl-2 and that exhibit favorable medicinal chemistry profiles.

With its sights set on a series A and an IPO following a £3.5 million (US$4.4 million) investment round in 2021, Scottish biotech ILC Therapeutics Ltd. is hoping to make waves with a sublingual interferon antiviral to treat COVID-19. The USP for the company’s lead, Alfacyte, is the fact that it’s an artificial version of interferon, so it has less of a propensity to cause the flu-like symptoms that can come from treatment with natural kinds, which hike levels of cytokines and interleukins. As a hybrid interferon that is composed of interferon alpha-10 and interferon alpha-12, Alfacyte is “up to 10,000 times less likely” to cause adverse effects, according to ILC CEO Alan Walker.

Albatroz Therapeutics Pte Ltd. has secured $3 million in seed funding to develop therapeutic antibodies against a new target that degrades the extracellular matrix, a key contributor to cancer and arthritis.

Dupuytren’s disease is often referred to as “the most common crippling hand condition that people have never heard of,” but Kurt Harrington is on a mission to change that. A seasoned biotech and pharma consultant, Harrington has had the condition himself for over a decade and, acutely aware of the lack of available treatment options, has founded Ventoux Biosciences Inc. to bring additional therapies forward.