GRIDS

GRIDS brings together world renowned experts from across the globe to discuss recent developments and unique challenges in the field of lysosomal storage disorders and/or rare genetic diseases.

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GRIDS 2021- Virtual Summit

Title: Lysosomal Storage Disorders – Concepts, Challenges and New Directions In the Era of Personalized Medcine

Sponsored by:

Takeda Pharmaceutical Company, Sanofi Genzyme, Spark Therapeutics, Chiesi USA, Amicus Therapeutics, Pfizer Inc, AVROBIO Inc and Ultragenyx

ABOUT

Even though there are subtle differences, the terms “personalized medicine” or “individualized medicine” express a similar vision — a reality where physicians treat based on each patient’s unique biology. Recognizing the value and need for such individualized therapies (also termed ‘Bespoke therapeutics’), US Food and Drug Administration (FDA) has also been proactively working with a range of organizations to facilitate public-private partnerships to best address the critical needs of rare disease population. Application of this concept is particularly important in rare disorders such as Lysosomal storage diseases (LSDs), which are therapeutically challenging due to phenotypic variability, complex pathology, systemic inflammation, and the need to deliver therapy to multiple organs. With the emergence of novel cellular and molecular technologies, several advanced therapeutic options including stem cell transplants, gene therapy, small molecule/nanomedicine approaches, and genome editing are being pursued across the globe, some of which are still in preclinical phases and some in clinical trials. As in case of treatment, major advances have also taken place over the years in diagnostic and prognostic testing for LSDs.

Advent of all the new and novel developments in the recent years as well as promise for emerging technologies makes it imperative to pursue precision or individualized medicine that result in patient-centric care for patients and families affected by LSDs. GRIDS 2021 will focus on latest advances, upcoming technological breakthroughs and regulatory frameworks which are changing the landscape of LSD diagnosis and treatment strategies. As in previous years, GRIDS will create a venue for clinicians, researchers, and industry and advocacy groups to exchange opinions and promote collaboration. We believe our innovative and strategic CME will provide an avenue to increase knowledge and develop healthcare providers' skills to identify better and treat patients with rare disorders. Thus, this program will ultimately enhance and improve the quality of life of rare and ultra-rare disease patients.

Global Learning Objectives

  • Get introduced to molecular and cellular aspects of clinical diversity and disease progression in LSDs

  • Gain knowledge about burden/role of genetic variation in pathological manifestation of LSDs

  • Get introduced to role for precision or individualized medicine approaches for LSDs

  • Gain insights into current treatment strategies and findings from preclinical studies and clinical trials towards novel therapies

  • Discuss the efficacy and safety in pre-clinical studies using gene therapy and promising clinical results

  • Review personalized pharmacochaperone and small molecular approaches for LSDs

  • Learn about novel tools and technologies for personalized medicine

AGENDA

Clinical Diversity and Genetic Variation in Lysosomal Disorders

  • Molecular and cellular aspects of clinical diversity and disease progression in Lysosomal disorders

  • Next-Generation molecular investigations in Lysosomal Diseases

  • Integration of patient aspects with disease mechanisms for patient-centric care

    Preclinical studies and Clinical Trials towards individualized therapies

  • Efficacy and safety in pre-clinical studies using gene therapy and promising clinical results

  • Targeting autophagy to overcome Lysosomal storage disorders

  • Pharmacological chaperones in treatment for Lysosomal storage disorders

Novel Technologies and Emerging Therapies for Precision Medicine

  • Digital Health- Digital tools and services to improve treatment of patients

  • Pharmacological small molecules for treatment of Lysosomal storage disorders

  • Gene therapy approaches to address emerging challenges

  • Guiding Treatment Decisions in Patients with Rare Diseases: Using a Patient-Centered Approach