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Details:
With the financing, CTx will continue the development and complete a Phase Ib clinical proof of concept of its lead product CTx001. CTx001 is a highly innovative AAV gene therapy for the treatment of Geograpic Atrophy (GA) secondary to dry age-related macular degeneration.
Lead Product(s): CTX001
Therapeutic Area: Ophthalmology Product Name: CTx001
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Gimv
Deal Size: $78.9 million Upfront Cash: Undisclosed
Deal Type: Series A Financing April 17, 2023
Details:
Exa‑cel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene‑edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: CRISPR Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 02, 2022
Details:
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: CRISPR Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 12, 2021
Details:
PRIME designation was granted based on clinical data from CRISPR and Vertex’s ongoing Phase 1/2 trial of CTX001 in patients with TDT.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: CRISPR Therapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 26, 2021
Details:
Vertex will lead global development, manufacturing and commercialization of CTX001 with support from CRISPR Therapeutics. Vertex will be responsible for 60% of program costs and will receive 60% of profits from future sales of CTX001 worldwide.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Vertex Pharmaceuticals
Deal Size: $1,100.0 million Upfront Cash: $900.0 million
Deal Type: Collaboration April 20, 2021
Details:
Data in seven patients from two ongoing Phase 1/2 trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
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 November 04, 2020
Details:
European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Vertex Pharmaceuticals
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 22, 2020
Details:
CRISPR Therapeutics continues to make substantial progress driving its multiple, ongoing clinical development programs. Enrollment in its immuno-oncology trials is ongoing, the compay has re-initiated dosing in its CTX001 trials.
Lead Product(s): CTX001
Therapeutic Area: Genetic Disease Product Name: CTX001
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, 2020
Details:
CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the EMA for sickle cell disease and transfusion-dependent beta thalassemia .
Lead Product(s): CTX001
Therapeutic Area: Hematology 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 May 11, 2020
Details:
Enrollment ongoing in clinical trials of CTX001™ for patients with severe hemoglobinopathies
Lead Product(s): CTX001
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 February 12, 2020
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