Patents by Inventor Amal Dakka
Amal Dakka has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).
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Publication number: 20240067646Abstract: Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.Type: ApplicationFiled: July 26, 2023Publication date: February 29, 2024Inventors: Hongyan QI, Soongyu CHOI, Amal DAKKA, Gary Mitchell KARP, Jana NARASIMHAN, Nikolai NARYSHKIN, Anthony A. TURPOFF, Marla L. WEETALL, Ellen WELCH, Matthew G. WOLL, Tianle YANG, Nanjing ZHANG, Xiaoyan ZHANG, Xin ZHAO, Luke GREEN, Emmanuel PINARD, Hasane RATNI
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Publication number: 20230310651Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.Type: ApplicationFiled: February 3, 2023Publication date: October 5, 2023Applicant: PTC Therapeutics, Inc.Inventors: Nikolai Naryshkin, Amal Dakka
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Patent number: 11753407Abstract: Provided herein are compounds of Formula (I): and forms thereof, wherein w1, w2, w3, w4, w5, w6 and w7 are as defined herein, including compositions thereof and uses therewith for treating spinal muscular atrophy.Type: GrantFiled: October 5, 2020Date of Patent: September 12, 2023Assignees: PTC Therapeutics, Inc., F. Hoffmann-La Roche AGInventors: Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Yurpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
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Publication number: 20230272367Abstract: In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production.Type: ApplicationFiled: May 12, 2023Publication date: August 31, 2023Applicant: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
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Patent number: 11702646Abstract: In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production.Type: GrantFiled: November 27, 2017Date of Patent: July 18, 2023Assignee: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
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Patent number: 11608501Abstract: Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.Type: GrantFiled: June 13, 2018Date of Patent: March 21, 2023Assignee: PTC Therapeutics, Inc.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Minakshi B. Jani, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
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Patent number: 11602567Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.Type: GrantFiled: April 13, 2020Date of Patent: March 14, 2023Assignee: PTC Therapeutics, Inc.Inventors: Nikolai Naryshkin, Amal Dakka
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Publication number: 20230057568Abstract: The present description relates to compounds useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno [3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.Type: ApplicationFiled: December 7, 2020Publication date: February 23, 2023Applicant: PTC THERAPEUTICS, INC.Inventors: Nanjing ZHANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Tom Tuan LUONG, Christie MORRILL, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Anthony TURPOFF, Jiashi WANG, Xiaoyan ZHANG
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Publication number: 20230047208Abstract: One aspect described herein is use of a compound to modulate the production of one or more mature RNA isoforms from a gene transcript in a cell. Another aspect described herein is use of a compound in a method to modulate exon inclusion or exon exclusion in one or more mature mRNA isoforms from a gene transcript in a cell.Type: ApplicationFiled: January 4, 2021Publication date: February 16, 2023Inventors: Dadi Gao, Elisabetta Morini, Michael E. Talkowski, Susan A. Slaugenhaupt, Wencheng Li, Kerstin Annemarie Effenberger, Christopher Robert Trotta, Yong Yu, Vijayalakshmi Gabbeta, Amal Dakka, Nikolai A. Naryshkin
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Publication number: 20220135568Abstract: The present description relates to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect described herein relates to substituted pyrrolo[2,3-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.Type: ApplicationFiled: February 10, 2020Publication date: May 5, 2022Applicant: PTC THERAPEUTICS, INC.Inventors: Jiashi WANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Nanjing ZHANG
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Publication number: 20220135586Abstract: The present description relates' to compounds of formula (I) useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno[3,2-b]pyridine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.Type: ApplicationFiled: February 10, 2020Publication date: May 5, 2022Applicant: PTC THERAPEUTICS, INC.Inventors: Nanjing ZHANG, Michael A. ARNOLD, Amal DAKKA, Gary Mitchell KARP, Tom Tuan LUONG, Jana NARASIMHAN, Nikolai A. NARYSHKIN, Jiashi WANG, Xiaoyan ZHANG
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Publication number: 20210276999Abstract: Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.Type: ApplicationFiled: October 5, 2020Publication date: September 9, 2021Inventors: Hongyan QI, Soongyu CHOI, Amal DAKKA, Gary Mitchell KARP, Jana NARASIMHAN, Nikolai NARYSHKIN, Anthony A. TURPOFF, Marla L. WEETALL, Ellen WELCH, Matthew G. WOLL, Tianle YANG, Nanjing ZHANG, Xiaoyan ZHANG, Xin ZHAO, Luke GREEN, Emmanuel PINARD, Hasane RATNI
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Publication number: 20210069350Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.Type: ApplicationFiled: April 13, 2020Publication date: March 11, 2021Applicant: PTC Therapeutics, Inc.Inventors: Nikolai Naryshkin, Amal Dakka
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Patent number: 10851101Abstract: Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.Type: GrantFiled: August 15, 2019Date of Patent: December 1, 2020Assignees: PTC Therapeutics, Inc., F. Hoffman-La Roche AGInventors: Hongyan Qi, Soongyu Choi, Amal Dakka, Gary Mitchell Karp, Jana Narasimhan, Nikolai Naryshkin, Anthony A. Turpoff, Marla L. Weetall, Ellen Welch, Matthew G. Woll, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Luke Green, Emmanuel Pinard, Hasane Ratni
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Publication number: 20200370043Abstract: Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small en molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.Type: ApplicationFiled: June 13, 2018Publication date: November 26, 2020Applicant: PTC THERAPEUTICS, INC.Inventors: Anuradha BHATTACHARYYA, Amal DAKKA, Kerstin EFFENBERGER, Vijayalakshmi GABBETA, Minakshi B. JANI, Wencheng LI, Nikolai NARYSHKIN, Christopher TROTTA, Kari WIEDINGER
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Patent number: 10668171Abstract: In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production.Type: GrantFiled: May 27, 2016Date of Patent: June 2, 2020Assignee: PTC Therapeutics, Inc.Inventors: Nikolai Naryshkin, Amal Dakka
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Publication number: 20190375750Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.Type: ApplicationFiled: August 15, 2019Publication date: December 12, 2019Inventors: Hongyan QI, Soongyu CHOI, Amal DAKKA, Gary Mitchell KARP, Jana NARASIMHAN, Nikolai NARYSHKIN, Anthony A. TURPOFF, Marla L. WEETALL, Ellen WELCH, Matthew G. WOLL, Tianle YANG, Nanjing ZHANG, Xiaoyan ZHANG, Xin ZHAO, Luke GREEN, Emmanuel PINARD, Hasane RATNI
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Patent number: 10501482Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.Type: GrantFiled: April 9, 2018Date of Patent: December 10, 2019Assignees: Hoffmann—La Roche Inc., PTC Therapeutics Inc.Inventors: Amal Dakka, Luke Green, Gary Karp, Jana Narasimhan, Nikolai Naryshkin, Emmanuel Pinard, Hongyan Qi, Hasane Ratni, Nicole Risher, Marla Weetall, Matthew Woll
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Publication number: 20190330615Abstract: In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production.Type: ApplicationFiled: November 27, 2017Publication date: October 31, 2019Applicant: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
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Patent number: RE47689Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.Type: GrantFiled: September 5, 2017Date of Patent: November 5, 2019Assignees: PTC Therapeutics, Inc., F. Hoffmann-La RocheInventors: Matthew G. Woll, Guangming Chen, Soongyu Choi, Amal Dakka, Song Huang, Gary Mitchell Karp, Chang-Sun Lee, Chunshi Li, Jana Narasimhan, Nikolai Naryshkin, Sergey Paushkin, Hongyan Qi, Anthony A. Turpoff, Marla L. Weetall, Ellen Welch, Tianle Yang, Nanjing Zhang, Xiaoyan Zhang, Xin Zhao, Emmanuel Pinard, Hasane Ratni