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PBFT02 utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene to patients with mutations in the gene that encodes for progranulin (PGRN).
Lead Product(s): PBFT02
Therapeutic Area: Neurology Product Name: PBFT02
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 2022
Details:
ATA188 (Allogeneic EBV T-cell), investigational off-the-shelf T-cell candidate, has the potential to target EBV-infected B cells and plasma cells in the central nervous system that may catalyze autoimmune responses and MS pathophysiology.
Lead Product(s): Allogeneic EBV T-cell
Therapeutic Area: Neurology Product Name: ATA188
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 12, 2022
Details:
ATA188 (Allogeneic EBV T-cell), Atara’s investigational off-the-shelf T-cell candidate, has the potential to target EBV-infected B cells and plasma cells in the central nervous system that may catalyze autoimmune responses and MS pathophysiology.
Lead Product(s): Allogeneic EBV T-cell
Therapeutic Area: Neurology Product Name: ATA188
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 06, 2022
Details:
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 II Product 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
Details:
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 II Product 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
Details:
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 II Product 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
Details:
LP352 is an oral, centrally acting, 5-HT2c superagonist in development for the potential treatment of seizures associated with developmental and epileptic encephalopathies.
Lead Product(s): LP352
Therapeutic Area: Neurology Product Name: LP352
Highest Development Status: Phase I/ Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 01, 2022
Details:
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 II Product 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
Details:
Full data from Phase I first-in-human study demonstrated AL001, a lithium-salicylate-L-proline engineered ionic cocrystal in plasma is bioequivalent to the marketed lithium carbonate product.
Lead Product(s): Lithium Salicylate Proline
Therapeutic Area: Neurology Product Name: AL001
Highest Development Status: Phase I/ Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 05, 2022
Details:
In the initial clinical data from low-dose cohort of the ongoing trial, we observed that LX1001 (AAVrh.10hAPOE2) expressed the protective APOE2 protein in the CNS and decreased core Alzheimer’s disease-related biomarkers.
Lead Product(s): AAVrh.10hAPOE2
Therapeutic Area: Neurology Product Name: LX1001
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 03, 2022
