The WORLDSymposium 2022 Virtual Platform is Now Closed

Thank you to all of the WORLDSymposium 2022 speakers, participants, exhibitors, and sponsors for making the 18th Annual Scientific Meeting a success. Thank you to everyone who attended, supported, and contributed to the scientific content for helping achieve the ongoing goal of providing the latest advances in lysosomal disease research and treatment to this global audience.

Information for obtaining credit can be found in the mobile app.



Sanofi Genzyme
avalglucosidase alfa-ngpt


JCR Pharmaceuticals
pabinafusp alfa

This year WORLDSymposium recognizes two important achievements in therapy for lysosomal diseases for attaining regulatory approval. The WORLDSymposium 2022 New Treatment Award will be presented to Sanofi Genzyme for avalglucosidase alfa-ngpt (Nexviazyme®) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA), and to JCR Pharmaceuticals for pabinafusp alfa (IZCARGO®), which provided clinical data meriting approval by the Ministry of Health, Labour and Welfare (MHLW) in Japan.

Congratulations to this year’s recipients!

2022 YOUNG INVESTIGATOR AWARDS presented February 8th

2022 Young Investigator Award Winners

2022 Young Investigator Award Winners

WORLDSymposium is pleased to announce the recipients of the 2022 Young Investigator Award. A total of 70 investigators-in-training, who submitted a research abstract to WORLDSymposium 2022, also applied for the 2022 Young Investigator Award. The WORLDSymposium Planning Committee reviewed all applications and 10 individuals were selected to receive the 2022 awards. The review process was difficult due to the excellent caliber of all the applications. Congratulations to this year’s recipients!

Stuart A. Kornfeld, MD received the 2022 Roscoe O. Brady Award for Innovation and Accomplishment

Stuart Kornfeld 2022 Roscoe O. Brady Award for Innovation and AccomplishmentWORLDSymposium is excited to announce Stuart A. Kornfeld, MD, as the recipient of the 2022 Roscoe O. Brady Award for Innovation and Accomplishment.

Dr. Kornfeld is the David C. and Betty Farrell Professor of Medicine at Washington University in St. Louis, Missouri. He is best known for his contributions to the field of glycoprotein research – the study of how sugars are attached to proteins and the roles these molecules play in cellular function. His lab was instrumental in elucidating the pathway for the biosynthesis of the mannose 6-phosphate moieties on lysosomal hydrolases. Defects in this pathway give rise to the lysosomal diseases mucolipidosis II and III. This glycoprotein receptor-mediated uptake of enzymes is the basis for ‘cross-correction’, the underpinning mechanism of all lysosomal enzyme replacement therapies.

The 2022 Roscoe O. Brady Award was presented on Monday, February 7, 2022 at 7:30 AM PST, followed by a scientific presentation from Dr. Kornfeld. Click here to read more.

Sue Kahn received the WORLDSymposium 2022 Patient Advocate Leader (PAL) Award

Sue Kahn: 2022 Patient Advocate Leader AwardeeCongratulations to Sue Kahn, the recipient of the WORLDSymposium 2022 Patient Advocate Leader (PAL) Award. Sue is a leader known for her business acumen and ability to build relationships, collaborate, and drive research at one of the oldest and most respected patient advocacy groups in the rare disease community. For 14 years, she has served as the Executive Director of the National Tay-Sachs & Allied Diseases Association (NTSAD) that supports families affected by Tay-Sachs, Canavan, GM1, and Sandhoff.

The 2022 Patient Advocate Leader Award was presented at 7:30 AM PST on Tuesday, February 8, 2022, at the 18th annual WORLDSymposiumin San Diego, California.

Click here to read more.


Tippi MacKenzie is a Professor of Surgery at the University of California, San Francisco and the Director of the Eli and Edythe Broad Institute for Regeneration Medicine. She is a pediatric and fetal surgeon who is focused on developing better ways to diagnose and treat genetic diseases before birth. She runs a translational research lab examining fetal immunology and maternal-fetal tolerance, with the ultimate goal of inventing new fetal therapies for patients with genetic diseases or pregnancy complications. She has moved two fetal molecular therapies from the lab to the clinic as phase 1 clinical trials after obtaining FDA approval: in utero hematopoietic stem cell transplantation to treat fetuses with alpha thalassemia and in utero enzyme replacement therapy in fetuses with lysosomal storage disorders.

Plan to attend Dr. MacKenzie’s Keynote Address Prenatal enzyme replacement therapy for lysosomal disorders: Launching a phase I clinical trial at 7:30 AM PST on Wednesday, February 9, 2022, at the 18th Annual WORLDSymposium in San Diego, California. Click here to read more.

Preliminary LIVE Schedule at A Glance

Schedule is subject to change
Download: WORLDSymposium 2022 Schedule at a Glance

Day Sunday
Feb. 6, 2022
Feb. 7, 2022
Feb. 8, 2022
Feb. 9, 2022
Feb. 10, 2022
Feb. 11, 2022
Morning Innovation Award
Basic Science
PAL & YI Awards
Translational Research
Keynote Address
Clinical Applications
New Treatment Award
Contemporary Forum (Non-CME)
Satellite Symposia
Late-breaking Abstracts
Lunch Satellite Symposia Satellite Symposia Satellite Symposia Satellite Symposia 11:15 AM Adjourn
Afternoon Robert J. Gorlin Symposium Basic Science Translational Research Clinical Applications Contemporary Forum (Non-CME)
Poster Session I Poster Session II Poster Session III Poster Session IV
Evening Satellite Symposia Satellite Symposia Satellite Symposia Satellite Symposia

We’re Organizing Research on Lysosomal Diseases

Dates to Remember

July 1, 2021
Abstract submission website open.

August 1, 2021
Registration for the conference opens. Distribution of exhibitor prospectus and support opportunities to all prior supporting companies, and also available by request.

October 1, 2021
Deadline for submission of full-length manuscripts for peer-review in the “Lysosomes” issue of Molecular Genetics & Metabolism, February, 2022. Click here to access the Guide for Authors or to Submit Your Paper.

October 1, 2021
Deadline for abstract submission to WORLDSymposium 2022.

November 1 – December 1,  2021
Late breaking abstract submission open.

November 15, 2021
Notification of abstract acceptance for poster or platform presentation at WORLDSymposium 2022.

About WORLDSymposium

The goal of WORLDSymposium is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases.

Sign up for Updates

Sign up for Updates

Click here to sign up for updates about the 18th Annual WORLDSymposium.

This symposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances.