Similar to 2020, the COVID-19 pandemic has dominated both biopharma deals with nonprofit entities and grants in 2021. Combined, pandemic deals and grants account for 87% of the total value, but only 22% of the volume, for the year.

Changing splicing lowers mutant huntingtin

Researchers at PTC Biotherapeutics Inc. have identified orally available small-molecule compounds that broadly lowered the levels of mutant huntingtin protein in both the brain and the periphery by affecting its splicing. One of those compounds, PTC-518, is currently in phase I trials as a therapy for Huntington's disease.

Sixfold raises $10.5M to advance targeted mRNA therapies

LONDON – The global pandemic has put the term mRNA on lips worldwide, but vaccines apart, the field of RNA therapeutics has failed to overcome the problem of delivery and is stuck at first base, targeting the liver. Newco Sixfold Bioscience Ltd. is the latest company working to end this constraint, applying a mixture of computation and advanced chemistry to design tissue-specific RNA delivery systems. The London-based start-up has just closed a US$10.5 million seed round to advance development of its Mergo platform, and says it already has promising data showing precise tissue targeting.

Varied players investigating Hippo pathway as Ikena sets up for phase I trial

Ikena Oncology Inc.’s plan to start the first-in-human phase I study with IK-930, a transcriptional enhanced associate domain inhibitor targeting the Hippo pathway, brought new attention to the space. The FDA accepted Ikena’s IND application early last month. Work with Hippo candidates is picking up steam, with a handful of companies pursuing early-stage prospects.

Could a new generation of drugs redefine care for schizophrenia?

Schizophrenia remains one of the toughest mental health issues to treat, as well as an indication that has created substantial challenges for drug makers. While players such as Invega Sustenna (paliperidone palmitate) developer Johnson & Johnson and Abilify (aripiprazole) developer Otsuka Pharmaceutical Co. Ltd. have seen substantial market success, both companies’ products have faced a multitude of challenges in terms of both common side effects and dosing. For those reasons and more, the pipeline of potential new schizophrenia medicines is busy, with Cortellis showing that 10 new phase III schizophrenia trials that began recruiting patients in 2021 and 59 phase III trials ongoing at the end of the year.

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Aditxt, Amylyx, Astrazeneca, Bio-Thera, Cantargia, Enbiotix, Hansa, Ionis, Janssen, Kolon Tissuegene, Lidds, Nrx, Oravax, Rational Vaccines, RDIF, Salarius, Saniona, Statera