Votoplam

Votoplam

CAS 2407849-89-0

Votoplam is a gene splicing modulator, used to inhibit Huntington’s disease.

Target: DNA/RNA Synthesis
Pathway: Cell Cycle/DNA Damage

Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. Death, typically caused by pneumonia or coronary artery disease, usually occurs 13 to 15 years after the onset of symptoms. The prevalence of HD is between three and seven individuals per 100,000 in populations of western European descent. In North America, an estimated 30,000 people have HD, while an additional 200,000 people are at risk of inheriting the disease from an affected parent. The disease is caused by an expansion of uninterrupted trinucleotide CAG repeats in the “mutant” huntingtin (Htt) gene, leading to production of HTT (Htt protein) with an expanded poly-glutamine (polyQ) stretch, also known as a “CAG repeat” sequence. There are no current small molecule therapies targeting the underlying cause of the disease, leaving a high unmet need for medications that can be used for treating or ameliorating HD. Consequently, there remains a need to identify and provide small molecule compounds for treating or ameliorating HD.

SCHEME

PATENT

PTC Therapeutics Inc., WO2022104058

WO2022103980’

PATENT

WO2020005873

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2020005873&_cid=P20-M1EWD1-90833-1

Example 37

Preparation of Compound 163

• Novel rna transcriptPublication Number: US-2022162610-A1Priority Date: 2020-11-12
• Novel rna transcriptPublication Number: WO-2022103980-A1Priority Date: 2020-11-12
• Novel rna transcriptPublication Number: WO-2022103980-A9Priority Date: 2020-11-12
• Heterocyclic and heteroaryl compounds for treating Huntington’s diseasePublication Number: JP-2021528467-APriority Date: 2018-06-27
• Heterocyclic and heteroaryl compounds for treating huntington’s diseasePublication Number: US-2021238186-A1Priority Date: 2018-06-27

References

[1] Annalisa Gatto et al. Audiol Res. Otological Planning Software-OTOPLAN: A Narrative Literature Review

[2] Dimitrios Paouris et al. J Pers Med. Validation of Automatic Cochlear Measurements Using OTOPLAN® Software

[3] Andrea Lovato et al. Otol Neurotol. OTOPLAN in Cochlear Implantation for Far-advanced Otosclerosis

[4] Kranti Bhavana et al. Indian J Otolaryngol Head Neck Surg. OTOPLAN-Based Study of Intracochlear Electrode Position Through Cochleostomy and Round Window in Transcanal Veria Technique

[5] Giampietro Ricci et al. J Int Adv Otol. OTOPLAN, Cochlear Implant, and Far-Advanced Otosclerosis: Could the Use of Software Improve the Surgical Final Indication?

REFERENCES
[1]. Sydorenko, et al. Preparation of heterocyclic and heteroaryl compounds for treating Huntington’s disease. World Intellectual Property Organization, WO2020005873 A1.
2020-01-02.

20240216369THE USE OF A SPLICING MODULATOR FOR A TREATMENT SLOWING PROGRESSION OF HUNTINGTON’S DISEASE

20240132509HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR TREATING HUNTINGTON’S DISEASE

20230405000TABLET FOR USE IN TREATING HUNTINGTON’S DISEASE AND METHOD OF MAKING THE SAME

20220162610NOVEL RNA TRANSCRIPT

20210238186Heterocyclic and heteroaryl compounds for treating Huntington’s disease

3814357HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR TREATING HUNTINGTON’S DISEASE

112654625HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR TREATING HUNTINGTON’S DISEASE

WO/2020/005873HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR TREATING HUNTINGTON’S DISEASE

/////////PTC Therapeutics, Votoplam