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RP-L102 is a lentiviral (LV) vector-based investigational gene therapy, which is being evaluated for the treatment of Fanconi Anemia, complementation group A.
Lead Product(s): RP-L102
Therapeutic Area: Genetic Disease Product Name: RP-L102
Highest Development Status: 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 02, 2024
Details:
Kresladi (marnetegragene autotemcel) is an investigational gene therapy that acts as a vector to deliver a functional copy of the ITGB2 gene. It is being developed for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).
Lead Product(s): Marnetegragene Autotemcel
Therapeutic Area: Genetic Disease Product Name: Kresladi
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 13, 2024
Details:
RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy, which is investigated for the treatment of Severe Leukocyte Adhesion Deficiency-I.
Lead Product(s): Marnetegragene Autotemcel
Therapeutic Area: Rare Diseases and Disorders Product Name: RP-L201
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 02, 2023
Details:
The proceeds will fund the development of company's pipeline of gene therapies for rare diseases, including advancement of RP-A501 in Phase 2 for Danon Disease. It consists of a recombinant AAV9 capsid containing a functional version of the human LAMP2B transgene.
Lead Product(s): RP-A501
Therapeutic Area: Genetic Disease Product Name: RP-A501
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: J.P. Morgan
Deal Size: $175.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering September 12, 2023
Details:
RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon Disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies.
Lead Product(s): RP-A501
Therapeutic Area: Genetic Disease Product Name: RP-A501
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 September 12, 2023
Details:
RP-A601 is an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).
Lead Product(s): RP-A601
Therapeutic Area: Cardiology/Vascular Diseases Product Name: RP-A601
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 June 08, 2023
Details:
RP-L301 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the PKLR gene.
Lead Product(s): RP-L301
Therapeutic Area: Genetic Disease Product Name: RP-L301
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 May 23, 2023
Details:
RP-A601 is an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).
Lead Product(s): RP-A601
Therapeutic Area: Cardiology/Vascular Diseases Product Name: RP-A601
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 09, 2023
Details:
RP-A501 (rAAV9 capsid) is a gene therapy product consisting of a recombinant adeno-associated serotype 9 capsid containing a functional version of the human LAMP2B transgene. RP-A501 has the potential to restore normal cardiac function in patients with Danon Disease.
Lead Product(s): rAAV9 Capsid
Therapeutic Area: Genetic Disease Product Name: RP-A501
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 09, 2023
Details:
RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease. During the meeting, Rocket reviewed the positive Phase 1 dataset with the FDA and proposed a study design and endpoints of the investigational gene therapy.
Lead Product(s): rAAV9 Capsid
Therapeutic Area: Genetic Disease Product Name: RP-A501
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 December 21, 2022
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