Apertura Gene Therapy LLC has unveiled its proprietary engineered adeno-associated virus (AAV) capsids that bind to the human transferrin receptor 1 (TfR1) for neurological gene therapy delivery.

Kynexis BV recently launched with a series A of €57 million (US$62 million) and a lead asset, Kyn-5356, that targets the kynurenine pathway. The company is preparing for clinical trials that will test the compound for the treatment of cognitive impairment associated with schizophrenia.

4D Molecular Therapeutics Inc. (4DMT) and Arbor Biotechnologies Inc. have established a strategic partnership focused on advancing new AAV-based gene-editing therapies for central nervous system (CNS) diseases with high unmet medical need in both rare and common disease populations.

In multiple sclerosis (MS), a demyelinating disorder, autoimmunity is generally considered the primary underlying pathophysiological cause. Therefore, developing treatments for MS has traditionally focused on modulating the immune system. However, an alternative hypothesis explains MS as a primarily or initially neurodegenerative disorder, in which neuronal death releases myelin, triggering a secondary autoimmune reaction.

Voyager Therapeutics Inc. has entered into a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG, to advance potential gene therapies for Huntington’s disease and spinal muscular atrophy (SMA).