Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Bempikibart
Therapeutic Area : Dermatology
Study Phase : Phase II
Sponsor : Q32 Bio
Deal Size : Undisclosed
Deal Type : Merger
Q32 Bio Closes Merger with Homology Medicines and Raises $42 Million
Details : The combined company will develop Q32 Bio’s clinical candidates, ADX-914 for atopic dermatitis and alopecia areata, and ADX-097 for complement disorders, both in Phase 2.
Brand Name : ADX-914
Molecule Type : Large molecule
Upfront Cash : Undisclosed
March 25, 2024
Lead Product(s) : Bempikibart
Therapeutic Area : Dermatology
Highest Development Status : Phase II
Sponsor : Q32 Bio
Deal Size : Undisclosed
Deal Type : Merger
Lead Product(s) : Bempikibart
Therapeutic Area : Dermatology
Study Phase : Phase II
Sponsor : Q32 Bio
Deal Size : Undisclosed
Deal Type : Merger
Q32 Bio and Homology Medicines Announce Merger Agreement
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...
Brand Name : ADX-914
Molecule Type : Large molecule
Upfront Cash : Undisclosed
November 16, 2023
Lead Product(s) : Bempikibart
Therapeutic Area : Dermatology
Highest Development Status : Phase II
Sponsor : Q32 Bio
Deal Size : Undisclosed
Deal Type : Merger
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 tr...
Brand Name : HMI-103
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
July 27, 2023
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 tr...
Brand Name : HMI-103
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 04, 2023
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : HMI-103
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 27, 2022
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Homology Medicines to Participate at Upcoming Conferences
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.
Brand Name : HMI-103
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 06, 2022
Lead Product(s) : HMI-103
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-204
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : HMI-204
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 11, 2022
Lead Product(s) : HMI-204
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : AAVHSC16
Therapeutic Area : Neurology
Study Phase : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 tes...
Brand Name : Undisclosed
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
July 05, 2022
Lead Product(s) : AAVHSC16
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-102
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Homology Medicines to Present on its Gene Editing and Therapy Programs for PKU
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.
Brand Name : HMI-102
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 29, 2022
Lead Product(s) : HMI-102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : HMI-102
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Homology Medicines Announces FDA Lifted Clinical Hold on pheNIX Gene Therapy Trial for PKU
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.
Brand Name : HMI-102
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 13, 2022
Lead Product(s) : HMI-102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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