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ISIS 678354 (olezarsen) is a APOC3 inhibitor, antisense oligonucleotide, which is being evaluated for the treatment of familial chylomicronemia syndrome.
Lead Product(s): Olezarsen
Therapeutic Area: Genetic Disease Product Name: ISIS 678354
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 07, 2024
Details:
ION224 is a phase 2 investigational LIgand-Conjugated Antisense (LICA) medicine designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) to treat patients with metabolic dysfunction-associated steatohepatitis (MASH).
Lead Product(s): ION224
Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: ION224
Highest Development Status: Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 13, 2024
Details:
AKCEA -APOCIII-LRx (olezarsen) is an RNA-targeted investigational ligand conjugated antisense oligonucleotide medicine, that acts as inhibitor of Apolipoprotein C-III. It is being evaluated for the treatment of familial chylomicronemia syndrome.
Lead Product(s): Olezarsen
Therapeutic Area: Genetic Disease Product Name: AKCEA -APOCIII-LRx
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 15, 2024
Details:
Wainua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is being developed for the treatment of transthyretin-mediated amyloid cardiomyopathy.
Lead Product(s): Eplontersen Sodium
Therapeutic Area: Genetic Disease Product Name: Wainua
Highest Development Status: Phase IIIProduct Type: Large molecule
Recipient: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 08, 2024
Details:
IONIS-PKK-LRx (donidalorsen) is an investigational ligand-conjugated antisense (LICA) medicine designed to target the prekallikrein (PKK) pathway. It is under phase 3 clinical development for the treatment of hereditary angioedema.
Lead Product(s): Donidalorsen
Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 22, 2024
Details:
IONIS-PKK-LRx (donidalorsen) is an RNA investigational LIgand-Conjugated Antisense (LICA) medicine designed to precisely target and silence the production of prekallikrein (PKK). It is being evaluated in phase 3 clinical trials for the treatment of hereditary angioedema.
Lead Product(s): Donidalorsen
Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 22, 2024
Details:
Wainua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is approved for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Lead Product(s): Eplontersen Sodium
Therapeutic Area: Neurology Product Name: Wainua
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 21, 2023
Details:
Under the agreement, Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target the prekallikrein, or PKK, pathway, for hereditary angioedema (HAE).
Lead Product(s): Donidalorsen
Therapeutic Area: Genetic Disease Product Name: IONIS-PKK-LRx
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Otsuka Pharmaceutical
Deal Size: Undisclosed Upfront Cash: $65.0 million
Deal Type: Licensing Agreement December 18, 2023
Details:
ION582 is designed to unsilence the paternal UBE3A allele in order to increase production of the UBE3A protein in the brain, which is investigated for the treatment of Angelman syndrome.
Lead Product(s): ION582
Therapeutic Area: Genetic Disease Product Name: ION582
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 November 11, 2023
Details:
Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization for two undisclosed early-stage programs for RNA-targeting investigational medicines for the treatment of Alzheimer's disease and Huntington's disease.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Large molecule
Partner/Sponsor/Collaborator: F. Hoffmann-La Roche
Deal Size: Undisclosed Upfront Cash: $60.0 million
Deal Type: Licensing Agreement September 27, 2023
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