Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.
Type:
Grant
Filed:
October 22, 2019
Date of Patent:
May 31, 2022
Assignee:
VICO THERAPEUTICS B.V.
Inventors:
Peter Christian De Visser, Susan Allegonda Maria Mulders
Abstract: The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
Type:
Grant
Filed:
October 22, 2019
Date of Patent:
March 15, 2022
Assignee:
VICO THERAPEUTICS B.V.
Inventors:
Josephus Johannes De Kimpe, Gerard Johannes Platenburg, Derick Gert Wansink