Gene Therapy

The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.

This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized

The primary purpose of the Research Database is to have a comprehensive source of observational data that can be used to study HSC transplantation and cellular therapies. A secondary purpose of the Research Database is to have a comprehensive source of data to study marrow toxic injuries. Objectives: To learn more about what makes stem cell transplants and cellular therapies work well such as: * Determine how well recipients recover from their transplants or cellular therapy; * Determine how recovery after a transplant or cellular therapy can be improved; * Determine how a donor's or recipient's genetics impact recipient recovery after a transplant or cellular therapy; * Determine how access to transplant or cellular therapy for different groups of patients can be improved; * Determine how well donors recover from the collection procedures.

IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.

The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.

This study aims to establish a novel multimodal evaluation and precision rehabilitation system for patients with congenital hearing loss who have received hearing aids, cochlear implantation, or emerging gene therapy. The proposed system will integrate age-stratified difficulty levels, combined with a gamified interaction platform or software designed to assess and train three core perceptual domains: auditory speech perception, music perception, and spatial hearing capabilities.

The study adopts a prospective, single-center, open-label clinical study to evaluate the safety and efficacy of inhaled BMD003(CFTR mRNA) in Chinese adult patients with cystic fibrosis This study is a single dose escalation study of BMD003 nebulized inhalation in CF patients. The study is divided into four cohorts, they are 2mg, 4mg, 8mg, and 16mg dose groups. The first dose group (2mg) is planned to enroll one patient. During the study, if there are ≥ 2 grade treatment-related adverse events (TRAEs) related to the investigational product that occur during the study period, 2 patients will be added at the current dose level. Each of the remaining groups will include 2-3 subjects, regardless of gender. Based on the safety data review of subjects in the previous dose group 4 weeks after administration, subjects in the next dose group will be allowed to receive the next higher dose, while patients in the previous dose group will complete the study. The study includes three stages: screening period (2 weeks), administration period (1 day), and follow-up period (approximately 13 months). After passing the screening of the subjects, D1 was admitted to the hospital, D1 received a single nebulized inhalation investigational product (BMD003), and D2 completed safety observation before being discharged. During the study, PK sample collection and safety assessment will be conducted at the corresponding visit points. After the treatment is completed, each patient will be followed up according to the schedule specified in the protocol.This study will conduct multiple dosing studies after the end of a single dose escalation. Multiple dosing doses, dosing intervals, etc. will be revised based on preclinical data and single dose data, and will be implemented after ethical approval.

Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are: * Is the gene therapy safe and well tolerated? * Does the gene therapy improve motor function and developmental outcomes? Participants will: * Undergo screening assessments to confirm eligibility * Receive a single dose of the gene therapy vector * Attend follow-up visits for safety monitoring and developmental assessments over the course of five years

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.