Drugs in Dev.
Genetic Disease
Preclinical
Canada 
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Lead Product(s) : LX-96
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Symbiosis Pharmaceutical Services
Deal Size : $10.0 million
Deal Type : Financing
Paradox Gets $10M from SymBiosis to Advance Protein Misfolding Disease Therapies
Details : The financing will be used to advance Paradox’s therapeutic pipeline, which includes LX-96, a monoclonal antibody designed for the treatment of late-stage immunoglobulin light chain (AL) amyloidosis.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Undisclosed
February 25, 2025
Lead Product(s) : LX-96
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Symbiosis Pharmaceutical Services
Deal Size : $10.0 million
Deal Type : Financing

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Lead Product(s) : SAT-3247
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Satellos Announces Promising Preliminary Data in Facioscapulohumeral Muscular Dystrophy
Details : SAT-3247 is a small molecule designed to inhibit AAK1, a protein kinase member of the notch pathway. It is being developed for the treatment of facioscapulohumeral muscular dystrophy.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
February 13, 2024
Lead Product(s) : SAT-3247
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Psilocybine
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Nova Mentis Life Science
Deal Size : Undisclosed
Deal Type : Agreement
Details : The pioneering Phase II clinical trial is the first human research investigating the potential of a microdose of psilocybin to improve behavioural and cognitive symptoms associated with Fragile X syndrome.
Product Name : Undisclosed
Product Type : Controlled Substance
Upfront Cash : Undisclosed
November 17, 2022
Lead Product(s) : Psilocybine
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Nova Mentis Life Science
Deal Size : Undisclosed
Deal Type : Agreement

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Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Altasciences Company Inc
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Company to conduct a pharmacokinetic bridging study in support of the XRx-008 program for autosomal dominant polycystic kidney disease (ADPKD), and XRx-101 for acute kidney injury associated with COVID-19.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 09, 2021
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Altasciences Company Inc
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Nova Mentis Files Genetic Neuroinflammatory Disease Patent
Details : Nova Mentis has filed a genetic neuroinflammatory disease patent which describes a diagnostic/therapeutic combination of mRNA molecules that encode proteins involved in the development of neurogenerative diseases, including fragile X syndrome and autism ...
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
November 09, 2021
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : FrontFundr
Deal Size : $1.0 million
Deal Type : Funding
Details : With no treatments currently available, Oak Bay Biosciences ‘Lead Molecule’ could be more than 90% effective in preventing the retinal cell death associated with Stargardt disease.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
September 29, 2021
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : FrontFundr
Deal Size : $1.0 million
Deal Type : Funding

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Lead Product(s) : INV-101
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Forbion
Deal Size : $35.0 million
Deal Type : Series B Financing
Inversago Pharma Closes US$35 M Series B Financing Round and Expands Board of Directors
Details : Proceeds will be used to develop Inversago’s best-in-class peripherally-acting CB1 inverse agonist candidate INV-101 to clinical proof-of-concept, and advance the research on other selected compounds.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
September 08, 2020
Lead Product(s) : INV-101
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Forbion
Deal Size : $35.0 million
Deal Type : Series B Financing

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Lead Product(s) : ECT-001
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : IRICoR
Deal Size : Undisclosed
Deal Type : Funding
IRICoR Makes Additional Investment in ExCellThera
Details : IRICoR's investment enabled ExCellThera to secure the co-funding required in connection with the grant awarded to it from the California Institute for Regenerative Medicine for a Phase 1 clinical trial using its ECT-001, for the treatment of severe sickl...
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Undisclosed
August 07, 2020
Lead Product(s) : ECT-001
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : IRICoR
Deal Size : Undisclosed
Deal Type : Funding
