Neuromuscular Disease*
Time: 4:15 PM to 5:45 PM
Description
The speakers in this session will cover the following areas: Gene therapy in pediatric neuromuscular disorders, the future of ALS, and advances in inclusion body myositis. Gene therapy in neuromuscular disorders, especially the FDA-approval of gene therapy for Duchenne Muscular Dystrophy and other gene therapies in the pipeline, represents exciting developments in the field; however, practitioners must manage therapy risks and potential adverse effects. There has been continued progress in ALS diagnosis, genetics, and therapeutic development. Inclusion body myositis is the most common myopathy in adults over the age of 50 years; a new IBM xenograft model has been developed and is a platform for testing new therapeutic targets.
Objectives
Discuss the basics of gene therapy and managing the risk of gene therapy in pediatric neuromuscular disorders.
Outline the future of ALS diagnosis, biomarkers, and therapeutic development.
Describe the new IBM xenograft model and other advances in IBM.
Speakers
Advances in Inclusion Body Myositis
DescriptionThis presentation will provide an overview on recent advances in the field of inclusion body myositis, including pathogenesis, animal and cell culture models, and clinical trials.
Learnings from Gene Therapy in Pediatric Neuromuscular Disorders
DescriptionThe focus will be on AAV-mediated gene therapy for pediatric neuromuscular disease, including nerve directed for spinal muscular atrophy (SMA) and for giant axonal neuropathy (GAN), as well as muscle directed for Duchenne muscular dystrophy (DMD) and for X-linked myotubular myopathy (XLMTM). I will highlight the opportunities and challenges for this therapeutic modality, and discuss milestones reached as well as ongoing initiatives to understand and mitigate various emerging safety signals associated with AAV-mediated gene therapy for pediatric neuromuscular disorders.
Preclinical Overview of CB03-154 (KCNQ2/3 Channel Opener) for Amyotrophic Lateral Sclerosis (ALS)
DescriptionShanghai Zhimeng Biopharma, Inc. is developing CB03-154, a potent and highly selective voltage-gated potassium channel-2/3 (Kv7.2/Kv7.3) activator for the treatment of patients with amyotrophic lateral sclerosis (ALS) and other neuropsychiatric disorders. KCNQ channel activators act as a positive allosteric modulator of KCNQ potassium (K+) channels leading to an inhibition of high-frequency action potential firing at the initial axon segment. Preclinical efficacy data suggested that CB03-154 is a promising new investigational drug for ALS and warrant further clinical studies in ALS patients.
SpeakersWhat's on the Horizon for ALS
DescriptionThe current treatment landscape in ALS has changed in the past year and the clinical trials leading to treatments will be reviewed. Additionally, the current clinical trial landscape will be reviewed and innovations in clinical trials and recent initiatives will be discussed.