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Details:
PTCy (post-transplant cyclophosphamide) in combination with Busulfan, Fludarabine & Tacrolimus is being evaluated for the treatment of patients with blood cancers who have received peripheral blood stem cells from partially matched unrelated donors.
Lead Product(s): Cyclophosphamide,Busulfan,Fludarabine Phosphate
Therapeutic Area: Oncology Product Name: PTCy
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 20, 2024
Details:
EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT.
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Hematology Product Name: EDIT-301
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 October 16, 2023
Details:
EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT.
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Hematology Product Name: EDIT-301
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 April 27, 2023
Details:
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Genetic Disease Product Name: EDIT-301
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 December 06, 2022
Details:
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Genetic Disease Product Name: EDIT-301
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 July 27, 2022
Details:
All 23 treated patients are alive and 20 patients with follow-up greater than 4 months recovered from pre-existing infections, are off protective isolation and prophylactic antimicrobials, and have normal growth velocity.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-107
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 May 03, 2022
Details:
MB-207 has been studied at NIAID since 2012 and continues to be assessed in a NIAID-supported Phase 1/2 clinical trial for XSCID in patients over the age of two who have received prior HSCT.
Lead Product(s): MB-207,Palifermin,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-207
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 January 24, 2022
Details:
EDIT-301 is an experimental cell therapy medicine currently being investigated in a RUBY trial, a clinical study in patients with severe SCD and TDT. After clearance from IND company will initiate the Phase 1/2 study for EDIT-301 in patients with TDT.
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Genetic Disease Product Name: EDIT-301
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 January 10, 2022
Details:
EDIT-301 is the first experimental medicine generated using CRISPR/Cas12a gene editing. The Company reported the data in an oral presentation at the 26th Congress of the European Hematology Association (EHA) being held virtually.
Lead Product(s): EDIT-301,Busulfan
Therapeutic Area: Genetic Disease Product Name: EDIT-301
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 11, 2021
Details:
FDA removes clinical hold for pivotal Phase 2 MB-107 clinical trial. Mustang will proceed with its plans to initiate the pivotal Phase 2 trial in newly diagnosed XSCID patients.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-107
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 February 02, 2021
Details:
The companies have signed an agreement to enable technology transfer and GMP clinical manufacturing of Mustang’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, in Europe.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-107
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Minaris Regenerative Medicine
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement November 23, 2020
Details:
U.S. FDA has granted Orphan Drug Designation to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, in newly diagnosed infants under the age of two.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-107
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 September 02, 2020
Details:
U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease Designation to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, in newly-diagnosed infants.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: MB-107
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 August 17, 2020
Details:
European Medicines Agency has granted Advanced Therapy Medicinal Product classification to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency.
Lead Product(s): MB-107,Busulfan
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 April 20, 2020