Clinical Trial, Phase 2

Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction

Post author:
Post published:July 23, 2026
Post category:

The proposed research will demonstrate both safety and efficacy of a heart-derived stem cell product in patients who have experienced a heart attack either recently or in the past by…

Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)

Post author:
Post published:July 22, 2026
Post category:

Therapeutic Candidate or Device Novel testing of a cell based therapy (Mesenchymal Stromal Cells) for respiratory failure from ARDS. Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will…

Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease

Post author:
Post published:July 22, 2026
Post category:

Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle…

Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome

Post author:
Post published:July 22, 2026
Post category:

Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

Post author:
Post published:July 22, 2026
Post category:

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects

Post author:
Post published:July 22, 2026
Post category:

A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach

Post author:
Post published:July 22, 2026
Post category:

Therapeutic Candidate or Device ALTO-100 Indication Bipolar depression (I or II) Therapeutic Mechanism ALTO-100 increased hippocampal neurogenesis and neuroplasticity. Its effects are mediated by signaling through the brain derived neurotrophic…

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

Post author:
Post published:July 22, 2026
Post category:

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…

The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

Post author:
Post published:July 22, 2026
Post category:

Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal…

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

Post author:
Post published:July 22, 2026
Post category:

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…