PYC has filed an international patent application titled...

PYC has filed an international patent application titled COMPOSITIONS AND METHODS FOR TREATMENT OF MONOGENIC NEURODEVELOPMENTAL DISORDER (WO2023235915). The patent application, which was filed in June this year and published last week, describes “antisense oligonucleotides, vectors, and related compositions and methods for increasing endogenous expression of SHANK3 protein and uses thereof for conditions associated with SHANK3 haploinsufficiency such as Phelan-McDermid syndrome”.

Priority right is claimed on three of the Monogenic Developmental Disorder Australian provisional patent applications filed in H2 last year.


The WO patent application makes claim with respect to both the 2”MOE and PPMO RNA chemistry approaches outlined by PYC in its PMS presentation on 06/06/23.

The conditions associated with SHANK3 haploinsufficiency which are claimed are Phelan-McDermid syndrome, an autism spectrum disorder, schizophrenia, or an intellectual disability.

Use of both viral and non-viral vectors is claimed as is use of a delivery nanocarrier comprising a lipid nanoparticle encapsulating the antisense oligonucleotide.

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2023235915&_cid=P11-LQEGBY-22394-1