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Acuitas CEO Dr Thomas Madden and NanoVation co-founder and president Dr Dominik Witzigmann discuss how LNP engineering is expanding genetic medicine applications to traditionally undruggable targets.

Lipid nanoparticles as RNA delivery vehicles across an expanding range of targetable indications, including outside the liver.

A new generation of RNA therapies delivered by lipid nanoparticles takes aim at extrahepatic tissues.

In vivo CAR T success will depend not only on the construct itself, but on whether the delivery system can safely and efficiently reach the right cells and provide the appropriate duration and level of expression.

A confluence of technological advancements enabling targeted in vivo delivery are beginning to release the brakes on the development of CAR T-cell therapies for autoimmune disease, where the patient population’s risk-benefit calculus favors interventions that are precise, reversible, and calibrated.

In this interview, Madden shares how the company is evolving beyond COVID. He discusses the company’s preformed vesicle technology and its potential to make personalized genetic medicines shelf-ready, the clinical and infrastructure challenges facing n-of-1 therapies, and his view of where LNP technology is headed over the next five years.

Designing an in vivo cell therapy that appropriately balances efficacy and safety remains a significant challenge; however DARPin-conjugated LNP have shown promise on both counts in preclinical studies.

Combining pre-formed vesicles with nucleic acid cargo at the point of care can remove long-standing barriers to personalized nucleic acid therapeutics.

What differentiates us is our full “soup to nuts” capability—we support process development, scale-up, and provide clinical and regulatory expertise. This ability to de-risk partner programs is highly attractive.

Acuitas Therapeutics shelled out an undisclosed sum to acquire a majority stake in Montreal-based and privately held RNA Technologies & Therapeutics.