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Through the financing, Neurona will focus on the development of NRTX-1001 for the treatment of drug-resistant mesial temporal lobe epilepsy and also has potential application in Alzheimer’s disease.


Lead Product(s): NRTX-1001

Therapeutic Area: Neurology Product Name: NRTX-1001

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

Partner/Sponsor/Collaborator: Viking Global Investors

Deal Size: $120.0 million Upfront Cash: Undisclosed

Deal Type: Financing February 08, 2024

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NRTX-1001 is a regenerative neural cell therapy candidate derived from human pluripotent stem cells. It secretes inhibitory neurotransmitter GABA to repair hyper-excitable neural networks. It is being investigated for dominant hemisphere drug-resistant focal epilepsy.


Lead Product(s): NRTX-1001

Therapeutic Area: Neurology Product Name: NRTX-1001

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 19, 2023

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NRTX-1001 is a regenerative neural cell therapy candidate derived from human pluripotent stem cells. It secretes inhibitory neurotransmitter GABA to repair hyper-excitable neural networks. It is being investigated for dominant hemisphere drug-resistant focal epilepsy.


Lead Product(s): NRTX-1001

Therapeutic Area: Neurology Product Name: NRTX-1001

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 14, 2023

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Early data from the first two patients treated with NRTX-1001 (Human Pluripotent Stem Cell Derived Neuronal Cell Therapy) report a trend toward seizure suppression and that administration of the cell treatment has been well-tolerated.


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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 21, 2022

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The patient received NRTX-1001 (human pluripotent stem cell derived neuronal cell therapy), and the treatment was well tolerated; there have been no serious adverse events. The patient has reported having four seizures during the first three months since receiving NRTX-1001.


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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 September 21, 2022

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NRTX-1001 is a regenerative neural cell therapy delivered as a single dose and designed to provide GABA, a key inhibitory neurotransmitter, to repair hyper-excitable neural networks associated with MTLE, the most common form of focal epilepsy in adults.


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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

Partner/Sponsor/Collaborator: ClearPoint Neuro

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 29, 2022

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NRTX-1001 is a regenerative neural cell therapy delivered as a single dose and designed to provide long-term secretion of gamma-aminobutyric acid (GABA), a key inhibitory neurotransmitter, to repair hyper-excitable neural networks associated with mesial temporal lobe epilepsy.


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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 June 29, 2022

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NRTX-1001 (Human Pluripotent Stem Cell Derived Neuronal Cell Therapy) is a regenerative neural cell therapy fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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 June 17, 2022

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The grant by the CIRM will support a dose-escalation Phase 1/2 clinical trial of NRTX-1001, an inhibitory cell therapy that is being evaluated for safety and efficacy in people with drug-resistant mesial temporal lobe epilepsy (MTLE).


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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

Partner/Sponsor/Collaborator: California Institute for Regenerative Medicine

Deal Size: $8.0 million Upfront Cash: Undisclosed

Deal Type: Funding May 26, 2022

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Data, generated from a single targeted administration in a preclinical model of epilepsy, demonstrate that NRTX-1001 inhibitory neurons persisted in epileptic hippocampus for duration of a 7-month study, were well-tolerated, and resulted in significant seizure suppression.


Lead Product(s): Human Pluripotent Stem Cell Derived Neuronal Cell Therapy

Therapeutic Area: Neurology Product Name: NRTX-1001

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 April 01, 2022

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