A PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)

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Therapeutic Candidate or Device An allogeneic anti-CD70 CAR-T cell product will be evaluated for the treatment of advanced or metastatic clear cell renal cell carcinoma (ccRCC). Indication Advanced or metastatic…

Continue ReadingA PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy…

Continue ReadingA Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme

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Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART…

Continue ReadingPhase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme

Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

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Therapeutic Candidate or Device Immune T cells from a patient’s transplant donor engineered to express chimeric antigen receptors for targeted leukemia killing Indication Relapsed or refractory acute myeloid leukemia Therapeutic…

Continue ReadingPhase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

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Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…

Continue ReadingPersonalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors

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Therapeutic Candidate or Device Adult Stem-Like T cells engineered with chimeric antigen receptor (CAR) to target cancers expressing IL13Ra2, including melanoma. Indication Advanced cancers that express IL13Ra2, including melanoma. Therapeutic…

Continue ReadingStem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors

A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

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Therapeutic Candidate or Device A patch comprised of a layer of stem cell-derived retinal pigmented epithelial (RPE) cells on a supporting matrix that is implanted under the retina Indication Geographic…

Continue ReadingA Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy