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Elevidys (delandistrogene moxeparvovec) is a single-dose, adeno-associated virus (AAV) based gene transfer therapy. It is being evaluated for the treatment of Duchenne muscular dystrophy patients with a confirmed mutation in the DMD gene.
Lead Product(s): Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease Product Name: Elevidys
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 16, 2024
Details:
SRP-5051 (vesleteplirsen) is a next-generation peptide phosphorodiamidate morpholino oligome. It is being evaluated in phase 2 clinical trials for the treatment of patients with duchenne muscular dystrophy amenable to skipping exon 51.
Lead Product(s): Vesleteplirsen
Therapeutic Area: Genetic Disease Product Name: SRP-5051
Highest Development Status: Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 29, 2024
Details:
SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector. It is being evaluated in phase 3 clinical studies for the treatment of limb-girdle muscular dystrophy type 2E/R4.
Lead Product(s): Bidridistrogene Xeboparvovec
Therapeutic Area: Genetic Disease Product Name: SRP-9003
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 16, 2024
Details:
Elevidys (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV) based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy
Lead Product(s): Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease Product Name: Elevidys
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 22, 2023
Details:
Elevidys (delandistrogene moxeparvovec) is a single-dose gene transfer therapy for intravenous infusion designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Lead Product(s): Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease Product Name: Elevidys
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 30, 2023
Details:
In connection with this investment, the companies will explore the development of novel gene therapy candidates, including LX2021 and LX2022 for a range of cardiovascular diseases.
Lead Product(s): LX2021
Therapeutic Area: Cardiology/Vascular Diseases Product Name: LX2021
Highest Development Status: DiscoveryProduct Type: Cell and Gene therapy
Recipient: Lexeo Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Financing August 28, 2023
Details:
Sarepta was awarded the PRV following U.S. FDA accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.
Lead Product(s): Delandistrogene Moxeparvovec-rokl
Therapeutic Area: Genetic Disease Product Name: Elevidys
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Undisclosed
Deal Size: $102.0 million Upfront Cash: Undisclosed
Deal Type: Divestment July 05, 2023
Details:
ELEVIDYS (delandistrogene moxeparvovec-rokl) is the first FDA approved gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS.
Lead Product(s): Delandistrogene Moxeparvovec-rokl
Therapeutic Area: Genetic Disease Product Name: ELEVIDYS
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 22, 2023
Details:
SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.
Lead Product(s): Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease Product Name: SRP-9001
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 24, 2023
Details:
SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.
Lead Product(s): Delandistrogene Moxeparvovec
Therapeutic Area: Genetic Disease Product Name: SRP-9001
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 12, 2023
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