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GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated in the treating of 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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 15, 2024
Details:
UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).
Lead Product(s): UX111
Therapeutic Area: Genetic Disease Product Name: UX111
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 06, 2024
Details:
GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A-AS. It is being phase 1/2 clinical trials for the treatment of 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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 05, 2024
Details:
UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter. It is being evaluated in phase 1/2 clinical trials for the treatment of Wilson Disease.
Lead Product(s): UX701
Therapeutic Area: Genetic Disease Product Name: UX701
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 25, 2024
Details:
Evkeeza (evinacumab) is the first angiopoietin-like 3 (ANGPTL3) inhibitor. ANGPTL3 is involved in controlling cholesterol levels. It is approved for the treatment of adolescents and adults aged 12 years and older with Homozygous Familial Hypercholesterolemia (HoFH).
Lead Product(s): Evinacumab-dgnb
Therapeutic Area: Genetic Disease Product Name: Evkeeza
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 04, 2024
Details:
GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS). It is under phase 1/2 clinical development for the treatment of 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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 03, 2024
Details:
Evkeeza (evinacumab) attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood.
Lead Product(s): Evinacumab-dgnb
Therapeutic Area: Genetic Disease Product Name: Evkeeza
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 18, 2023
Details:
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: UX143
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: J.P. Morgan
Deal Size: $326.1 million Upfront Cash: Undisclosed
Deal Type: Public Offering October 23, 2023
Details:
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: UX143
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: J.P. Morgan
Deal Size: $300.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering October 18, 2023
Details:
The proceeds will advance company's late-stage clinical programs, including UX143 (setrusumab), an investigational, fully human mAb that inhibits sclerostin, support commercial launch, and advance its preclinical pipeline through IND applications into clinical development.
Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: UX143
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: J.P. Morgan
Deal Size: $345.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering October 17, 2023
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