A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

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Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication…

Continue ReadingA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

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Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic…

Continue ReadingA Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant

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Therapeutic Candidate or Device COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host Indication Older (>40 yrs) Severe Aplastic Anemia…

Continue ReadingTreatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant

Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

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Therapeutic Candidate or Device NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells. Indication Focal epilepsy; drug-resistant mesial temporal lobe epilepsy. Therapeutic Mechanism NRTX-1001 is an…

Continue ReadingPhase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas

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Therapeutic Candidate or Device Neural stem cells that are genetically engineered to express a cancer-killing virus that specifically targets brain tumor cells Indication Recurrent brain tumors in adults: high grade…

Continue ReadingA Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas