
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : PPL-002
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Papillon Gets Rare Pediatric Designation for PPL-002 in Danon Disease
Details : PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, being evaluated for the treatment of Danon disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
October 21, 2024
Lead Product(s) : PPL-002
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : PPL-002
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Papillon Therapeutics Receives Orphan Drug Designation For PPL-002 in Danon Disease
Details : PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, being evaluated for the treatment of Danon disease.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
October 01, 2024
Lead Product(s) : PPL-002
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : PPL-001
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Papillon Receives Orphan Drug Designation for PPL-001 in Friedreich's Ataxia
Details : PPL-001 is an experimental gene-corrected CD34+ HSPC therapy. Currently, it is being investigated for the treatment of Friedreich's ataxia.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
September 17, 2024
Lead Product(s) : PPL-001
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
