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The funding will support SpliSense to continue the clinical development of an inhaled antisense oligonucleotide drug SPL84 for treating patients suffering from cystic fibrosis.
Lead Product(s): SPL84
Therapeutic Area: Genetic Disease Product Name: SPL84
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: CF Foundation
Deal Size: $8.5 million Upfront Cash: Undisclosed
Deal Type: Funding April 03, 2024
Details:
SPL84 is an antisense oligonucleotide product that binds specifically to the mutated CFTR RNA in the targeted sequence. It is being evaluated for the treatment of cystic fibrosis.
Lead Product(s): SPL84
Therapeutic Area: Genetic Disease Product Name: SPL84
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 03, 2024
Details:
SPL84, the Company's lead inhaled anti sense oligonucleotide (ASO) product for the treatment of patients with CF, carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
Lead Product(s): SPL84
Therapeutic Area: Genetic Disease Product Name: SPL84
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 06, 2023
Details:
SPL84, has shown to have completely restored CFTR activity in the CF gold standard pharmacological model, suggesting potential cure for these patients.
Lead Product(s): SPL84
Therapeutic Area: Genetic Disease Product Name: SPL84
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 December 14, 2022
Details:
Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein.
Lead Product(s): SPL23-ASO
Therapeutic Area: Genetic Disease Product Name: SPL23-ASO
Highest Development Status: PreclinicalProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 11, 2021
Details:
Proceeds will be used to advance the Company’s pipeline, including its lead Anti Sense Oligonucleotide product, SPL84-23. SPL84-23 to treat the 3849+10kb C->T CFTR mutation, that has showed ability to completely restore CFTR channel function in patient-derived cell cultures.
Lead Product(s): SPL84-23
Therapeutic Area: Genetic Disease Product Name: SPL84-23
Highest Development Status: IND EnablingProduct Type: Large molecule
Partner/Sponsor/Collaborator: Biotel Limited
Deal Size: $28.5 million Upfront Cash: Undisclosed
Deal Type: Series B Financing May 13, 2021
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