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The combined company will advance the development of Q32 Bio’s two clinical development candidates, ADX-914 (bempikibart) in Phase 2 for the treatment of atopic dermatitis (AD) and alopecia areata (AA), and ADX-097 entering Phase 2 for the treatment of complement disorders.
Lead Product(s): Bempikibart
Therapeutic Area: Dermatology Product Name: ADX-914
Highest Development Status: Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Q32 Bio
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Merger March 25, 2024
Details:
The combined company will advance the development of Q32 Bio’s two clinical development candidates, ADX-914 (bempikibart) in Phase 2 for the treatment of atopic dermatitis (AD) and alopecia areata (AA), and ADX-097 entering Phase 2 for the treatment of complement disorders.
Lead Product(s): Bempikibart
Therapeutic Area: Dermatology Product Name: ADX-914
Highest Development Status: Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Q32 Bio
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Merger November 16, 2023
Details:
HMI-103 is a nuclease-free gene editing candidate for PKU which harness the body's natural DNA repair process of homologous recombination replacing disease-causing gene with functional gene and liver-specific promoter to maximize PAH expression in all transduced liver cells.
Lead Product(s): HMI-103
Therapeutic Area: Genetic Disease Product Name: HMI-103
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 27, 2023
Details:
HMI-103 is a nuclease-free gene editing candidate for PKU which harness the body's natural DNA repair process of homologous recombination replacing disease-causing gene with functional gene and liver-specific promoter to maximize PAH expression in all transduced liver cells.
Lead Product(s): HMI-103
Therapeutic Area: Genetic Disease Product Name: HMI-103
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 04, 2023
Details:
HMI-103 has the potential to treat adult and pediatric PKU with its unique dual mechanism of action designed to integrate the PAH gene and liver-specific promoter into the genome and to maximize PAH expression in all transduced liver cells.
Lead Product(s): HMI-103
Therapeutic Area: Genetic Disease Product Name: HMI-103
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 27, 2022
Details:
HMI-103 is an AAVHSC15 vector containing a functional copy of the human PAH gene. HMI-103 is designed as a one-time, in vivo product candidate incorporating nuclease-free gene editing for PKU.
Lead Product(s): HMI-103
Therapeutic Area: Genetic Disease Product Name: HMI-103
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 06, 2022
Details:
Following a single I.V. administration of HMI-204 in the MLD murine model, the candidate crossed the blood-brain-barrier to the central nervous system (CNS) and reached key peripheral organs involved in MLD.
Lead Product(s): HMI-204
Therapeutic Area: Genetic Disease Product Name: HMI-204
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 2022
Details:
AAVHSC16, a type of glycan) binding feature of other AAVHSCs and Clade F AAVs in vitro, did not show improved binding or a difference in number of vector genomes (vgs) or eGFP expression in cells with terminally exposed galactose, while other AAVHSCs tested did.
Lead Product(s): AAVHSC16
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 05, 2022
Details:
The pheNIX trial is evaluating HMI-102, a one-time gene therapy product candidate that is designed to deliver working copies of the PAH gene to liver cells.
Lead Product(s): HMI-102
Therapeutic Area: Genetic Disease Product Name: HMI-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2022
Details:
HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, and delivered via the liver-tropic AAVHSC15 vector.
Lead Product(s): HMI-102
Therapeutic Area: Genetic Disease Product Name: HMI-102
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 13, 2022
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