The rapid migration of gene editing technologies from the bench to the clinic has opened up new therapeutic possibilities for patients with previously intractable genetic diseases and difficult-to-treat cancers. But mobilizing gene editing components into a target cell or organ remains a critical step for the field. Integra Therapeutics SL, an early stage Spanish firm, is now engaged in that process with a novel gene writing platform.

Although preclinical studies of genetically engineered interleukin-18 (IL-18) historically indicated its potential in cancer treatment, interest in the cytokine languished after GSK plc shelved its IL-18 therapeutic on lack of responses in a phase II melanoma trial. But in 2020, Yale University-based professor of immunobiology Aaron Ring set about reviving IL-18 as a cancer therapeutic. He went on to engineer a decoy-resistant form of IL-18 and then founded Simcha Therapeutics Inc. to develop the drug – named ST-067.

Galimedix Therapeutics Inc. plans to move its lead compound, GAL-101, into a phase IIa proof-of-concept trial next year, in order to test an intriguing hypothesis associated with certain retinal degeneration conditions. GAL-101 selectively binds misfolded amyloid beta species, and the company believes that this mechanism may benefit patients with the dry form of age-related macular degeneration or with glaucoma.

“From one to many” is how Actio Biosciences Inc. describes its approach to drug development. The firm emerged with a $55 million series A financing and an eye for biological targets found in both rare and common diseases, starting with TRPV4, a target associated with Charcot-Marie-Tooth disease type 2C and other bone diseases.

Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own. “Nucleic acid therapeutics can be incredibly fragile,” Thomas Madden, CEO of Acuitas Therapeutics, told BioWorld. “When injected into the body without a delivery system, messenger RNA, for example, is rapidly destroyed.”

As its name suggests, Superluminal Medicines Inc. is aiming for speed. The startup, which closed a $33 million seed round led by RA Capital Management, is combining a biology-focused approach with a generative AI platform it says has the potential to create candidate-ready compounds in a matter of months, with its initial sights set on G protein-coupled receptor targets.

As a cytokine used in the field of immuno-oncology, interleukin-2 (IL-2) can produce durable and even complete responses in some patients, as well as induce immune memory against tumors. However, its rapid metabolism within the body means it has a short serum half-life, so it needs to be given in high doses, which can trigger severe side effects.

The old Viralytics Ltd. team is back together again in a new company called Immvirx Pty Ltd. that is developing oncolytic viruses to tackle the four most deadly cancers, Immvirx CEO and co-founder Malcolm McColl told BioWorld.

Biocity Biopharmaceutics Co. Ltd. is advancing its pipeline of differentiated, modality-independent therapeutics for oncology that include small molecules, monoclonal and bispecific antibodies, as well as antibody-drug conjugates (ADCs). The Wuxi, China-headquartered company is focused on first-in-class molecules for different modalities that can be combined with other treatments, Biocity Co-founder and Executive Vice President Ivy Wang told BioWorld.

Critically shortened telomeres are known to be a key limiting factor in successful CAR T-cell immunotherapy, with cells from older patients tending to have shorter telomeres with reduced dividing capacity. As such, these cells are unable to fully eliminate malignancies and provide durable and persistent protection against cancer. So, what if you could lengthen these DNA-protein structures found at the end of chromosomes during the ex-vivo manufacturing of CAR T cells, i.e., before they are put back into the body, so increasing their potency against disease? Newly-launched firm Telos Biotech – a subsidiary company of Cambrian Bio – believes you can, with its patent-protected recombinant protein, Telovance, promising to shake up the cell therapy field.