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Last Updated: April 25, 2024

Claims for Patent: 9,655,979

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Summary for Patent: 9,655,979

Title:	RNA trans-splicing molecule (RTM) for use in the treatment of cancer
Abstract:	The present invention pertains to a novel pre-mRNA trans-splicing molecule (RTM) comprising a binding region which is complementary to a pre mRNA of a tumor associated gene, and a coding domain which encodes for a suicide gene. Specific embodiments of the present invention relate to RTMs which mediate trans-splicing of a suicide gene, such as thymidine kinase from herpes simplex virus (HSV-tk), with the pre mRNA of the squamous cell carcinoma associated gene solute carrier organic anion transporter family member 1B3 (SLCO1B3). The invention provides RTMs which selectively kill cells expressing SLCO1B3 and which are thus useful in the treatment of cancer, specifically squamous cell carcinoma associated with epidermolysis bullosa. Also provided are methods, kits and pharmaceutical compositions relating to the RTMs in accordance to the invention.
Inventor(s):	Bauer; Johann (Salzburg, AT), Gruber; Christina (Bad Vigaun, AT), Koller; Ulrich (Neumarkt am Wallersee, AT)
Assignee:	Bauer; Johann (Salzburg, AT) Gruber; Christina (Bad Vigaun, AT) Koller; Ulrich (Neumarkt am Wallersee, AT)
Application Number:	14/438,625
Patent Claims:	1. A method for treating a disease, wherein said method comprises administering, to a subject in need of such treatment, an RNA trans-splicing molecule (RTM) comprising a splicing domain, a coding domain, and a binding domain, characterized in that (a) the splicing domain comprises a strong conserved branch point (BP) sequence, a polypyrimidine (PPT), and a 3' acceptor splice site (ss); (b) the coding domain comprises a suicide gene sequence thymidine kinase from herpes simplex virus (HSV-tk), and (c) the binding domain comprises a sequence complementary to intron 3 of the pre-mRNA of solute carrier organic anion transporter family member 1B3 (SLCO1B3). 2. The method, according to claim 1, wherein said disease is a proliferative disease. 3. The method, according to claim 2, wherein said proliferative disease is selected from squamous cell carcinoma, epidermolysis bullosa, cystic fibrosis, pachyonychia congenita, psoriasis and neurodermitis.

Details for Patent 9,655,979

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Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986	⤷ Try a Trial	2032-11-02
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132		⤷ Try a Trial	2032-11-02
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132		⤷ Try a Trial	2032-11-02
>Applicant	>Tradename	>Biologic Ingredient	>Dosage Form	>BLA	>Approval Date	>Patent No.	>Expiredate

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