Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : BEAM-101,Busulfan
Therapeutic Area : Hematology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Patient Death Taints Otherwise Positive Data for Beam’s Sickle Cell Gene Therapy
Details : BEAM-101 is a cell therapy which increases the production of fetal hemoglobin (HbF), was being investigated in patients with sickle cell disease.
Brand Name : BEAM-101
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 05, 2024
Lead Product(s) : BEAM-101,Busulfan
Therapeutic Area : Hematology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Cyclophosphamide,Busulfan,Fludarabine Phosphate
Therapeutic Area : Oncology
Study Phase : Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
NMDP Announces Enrollment of First Patient in OPTIMIZE Clinical Trial
Details : PTCy (post-transplant cyclophosphamide) combined with Busulfan, Fludarabine, and Tacrolimus is being evaluated for blood cancer patients receiving stem cells from partially matched unrelated donors.
Brand Name : PTCy
Molecule Type : Small molecule
Upfront Cash : Not Applicable
February 20, 2024
Lead Product(s) : Cyclophosphamide,Busulfan,Fludarabine Phosphate
Therapeutic Area : Oncology
Highest Development Status : Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : EDIT-301,Busulfan
Therapeutic Area : Hematology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 16, 2023
Lead Product(s) : EDIT-301,Busulfan
Therapeutic Area : Hematology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : EDIT-301,Busulfan
Therapeutic Area : Hematology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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...
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
April 27, 2023
Lead Product(s) : EDIT-301,Busulfan
Therapeutic Area : Hematology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : EDIT-301,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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).
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 06, 2022
Lead Product(s) : EDIT-301,Busulfan
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) : EDIT-301,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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).
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
July 27, 2022
Lead Product(s) : EDIT-301,Busulfan
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) : MB-107,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : MB-107
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 03, 2022
Lead Product(s) : MB-107,Busulfan
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) : MB-207,Palifermin,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : MB-207
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 24, 2022
Lead Product(s) : MB-207,Palifermin,Busulfan
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) : EDIT-301,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Editas Medicine Reports on Recent Progress and Outlook at J.P. Morgan Healthcare Conference
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.
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 10, 2022
Lead Product(s) : EDIT-301,Busulfan
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) : EDIT-301,Busulfan
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : EDIT-301
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 11, 2021
Lead Product(s) : EDIT-301,Busulfan
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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