X
Upload your Marketing & Sales content on your company Virtual Booth, click HERE.
Details:
GTX-102 an antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces levels of UBE3A-AS and reactivates expression of paternal UBE3A allele in neurons of CNS.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Recipient: Ultragenyx Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 18, 2022
Details:
GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Undisclosed Upfront Cash: $75.0 million
Deal Type: Acquisition July 18, 2022
Details:
Phase 1/2 Clinical Study of GTX-102 that inhibit expression of UBE3A-AS, showed no treatment-related serious adverse events to lower extremity weakness observed in the patients, and initial assessments have shown early signs of clinical activity.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 05, 2022
Details:
GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS used in pediatric patients with Angelman syndrome.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 27, 2021
Details:
The phase 1/2 study evaluates the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 22, 2021
Details:
At the highest doses, all five patients experienced a serious adverse event (SAE) of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: GTX-102
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 26, 2020
Details:
GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of Angelman syndrome.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 04, 2020
Details:
First investigational study testing an antisense oligonucleotide as a potential therapy to treat AS.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 16, 2020
Details:
The Phase 1/2 open-label, multiple-dose, dose-escalating study will evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 14, 2020
Details:
GeneTx’s Phase 1/2 study, named KIK-AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020.
Lead Product(s): GTX-102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: IND EnablingProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 15, 2020
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results] 
Market Place
