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Sio Gene Therapies
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Sio intends to return the global rights for AXO-Lenti-PD and that they intend to cease work on this gene therapy programme in Parkinson’s Disease. Sio is currently conducting a Phase 2 SUNRISE-PD trial with AXO-Lenti-PD.


Lead Product(s): AXO-Lenti-PD

Therapeutic Area: Neurology Product Name: OXB-102

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Recipient: Oxford Biomedica

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement January 31, 2022

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Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to increase β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.


Lead Product(s): AXO-AAV-GM1,Rituximab,Sirolimus

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

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 21, 2021

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AXO-AAV-GM1, the company’s adeno-associated viral vector (AAV)9-based gene therapy for the treatment of Type I (early infantile onset) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.


Lead Product(s): AXO-AAV-GM1,Rituximab,Sirolimus

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

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 04, 2021

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Initial data from the ongoing Phase 1/2 study in five patients in the low-dose cohort showed that AXO-AAV-GM1 was generally well tolerated with a favorable safety profile and provide early indications of clinical disease stability.


Lead Product(s): AXO-AAV-GM1,Rituximab,Sirolimus

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: National Human Genome Research Institute

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 15, 2020

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AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases. The Company received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold.


Lead Product(s): AXO-AAV-GM2

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM2

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 09, 2020

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AXO-Lenti-PD gene therapy demonstrate consistent treatment outcomes including Favorable safety and tolerability profile, with no serious adverse events attributable to gene therapy; Improvement in Hauser diary “Good ON time” and “OFF time” and other outcomes.


Lead Product(s): AXO-Lenti-PD

Therapeutic Area: Neurology Product Name: OXB-102

Highest Development Status: Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Oxford Biomedica

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 29, 2020

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AXO-AAV-GM1 is the first gene therapy to enter clinical trials for GM1 gangliosidosis and the company believes that it has the potential to provide meaningful clinical benefit to both Type I and Type II patients.


Lead Product(s): AXO-AAV-GM1

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

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 09, 2020

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AXO-Lenti-PD was well-tolerated with no treatment-related serious adverse events at 6 months. EXPLORE-PD, a randomized, sham-controlled study of AXO-Lenti-PDis expected to begin dosing in 2021.


Lead Product(s): AXO-Lenti-PD

Therapeutic Area: Neurology Product Name: OXB-102

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 October 06, 2020

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Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercialization.


Lead Product(s): AXO-AAV-GM1

Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Recipient: Viralgen

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership September 15, 2020

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Oxford Biomedica will manufacture GMP batches for Axovant to support the ongoing and future clinical development of AXO-Lenti-PD, a clinical-stage gene therapy product to treat moderate to severe Parkinson’s Disease based on Oxford Biomedica’s LentiVector® platform.


Lead Product(s): AXO-Lenti-PD

Therapeutic Area: Neurology Product Name: OXB-102

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Recipient: Oxford Biomedica

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement July 31, 2020

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