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OTL-203, an investigational hemopoietic stem cell (HSC) gene therapy in development for the potential treatment of mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH).
Lead Product(s): OTL-203
Therapeutic Area: Genetic Disease Product Name: OTL-203
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Orchard Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 26, 2022
Details:
OTL-105 is an investigational HSC gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks.
Lead Product(s): Autologous Hematopoietic Stem Cell Gene Therapy
Therapeutic Area: Genetic Disease Product Name: OTL-105
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Pharming
Deal Size: $211.5 million Upfront Cash: $17.5 million
Deal Type: Collaboration July 01, 2021
Details:
Proceeds from this financing will support the advancement of Evox’s exosome-based therapeutics pipeline, including progression of several rare disease assets into the clinic, and continued development of its world-leading DeliverEX exosome drug platform.
Lead Product(s): EVX-101
Therapeutic Area: Genetic Disease Product Name: EVX-101
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: Redmile Group
Deal Size: $95.4 million Upfront Cash: Undisclosed
Deal Type: Series C Financing February 18, 2021
Details:
Deal allows Evox to continue advancing its exosome-based targeting and delivery technology, while also leveraging Takeda’s development and clinical expertise to advance these partnered programmes.
Lead Product(s): Protein therapeutics
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: Takeda Pharmaceutical
Deal Size: $882.0 million Upfront Cash: $44.0 million
Deal Type: Collaboration March 26, 2020
Details:
Paper demonstrates first in class TMEM16A chloride channel potentiators, to accelerate mucociliary clearance.
Lead Product(s): ETX001
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 08, 2020
