Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in regulating a number of pathways involved in the control of neuronal viability and function.

In an ideal world, when a patient takes a medicine, it acts only at the specific site of disease in the human body whilst sparing healthy tissues. But it almost goes without saying that with many drug regimens, side effects or complications are part of the package. Working behind the scenes to address this limitation over the past couple of years, Cambridge, Mass.-based venture capital company Flagship Pioneering is now publicizing its platform of “programmable medicines” that directly and precisely target diseased tissue, funnelling $50 million of investment in the technology and recognising its achievements formally through the launch of Ampersand Biomedicines.

Rapport Therapeutics Inc. launched with $100 million in series A funding and ambitious plans to bring a hitherto unprecedented level of precision to therapies for neurological disease. Although the Boston-based company is new to the world, its underlying platform has been a decade in the making, and it already has one clinical-stage asset, which is in development for seizure disorders. It is currently undergoing a phase I trial.

Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the non-coding genome.

Armed with $193 million in combined series A and B rounds, Aera Therapeutics aims to address current challenges with delivering gene-based therapies via a self-assembling human-derived protein platform it has in-licensed from a well-known Harvard scientist’s lab.

For Aqemia SA, the year got off to a good start, as one of its pharma partners, Les Laboratoires Servier SAS, extended an existing collaboration to drug a supposedly undruggable immuno-oncology target, using its Launchpad artificial intelligence platform.

Transcend Therapeutics Inc. has closed on a series A funding of $40 million for its next-generation compound, methylone, an MDMA analogue, to be developed as a rapid-acting, disease-modifying, non-hallucinogenic treatment for neuropsychiatric conditions, including post-traumatic stress disorder.

Resalis Therapeutics Srl closed a €10 million (US$10.6 million) seed round to progress toward the clinic a micro-RNA (miR) inhibitor in development for metabolic disease indications. Riccardo Panella, chief scientific officer and founder, identified the potential of a particular miR species, miR-22, as a target for metabolic syndrome while conducting studies on its potential role in oncology while at Harvard Medical School and Beth Israel Deaconess Medical Center.

One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said.

Fresh off an end-of-year IPO, Coya Therapeutics Inc. is gearing up for clinical testing with its lead Treg-enhancing biologic in neurodegenerative disease, aiming to build on a wealth of academic-generated data highlighting the potential of Treg therapy to attack the neuroinflammation underlying diseases such as amyotrophic lateral sclerosis and Alzheimer’s disease.