2023 TANGO2 Research Symposium

Tango2 Research Foundation

SHARING THE LATEST TANGO2 RESEARCH & BROADENING OUR RESEARCH NETWORK.
*Families, fellows & students FREE with code SPECIALGUEST

Thank you for registering for the 2023 TANGO2 Research Symposium!

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Baylor College of Medicine and Tango2 Research Foundation. Baylor College of Medicine is accredited by the ACCME to provide continuing medical education for physicians.

Baylor College of Medicine designates this live activity for a maximum of 3.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Baylor College of Medicine and Tango2 Research Foundation. Baylor College of Medicine is accredited by the ACCME to provide continuing medical education for physicians.Baylor College of Medicine designates this live activity for a maximum of 3.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

The TANGO2 Research Foundation is hosting a virtual TANGO2 Research Symposium on Friday, October 27, 2023 from 9:00am to 1:00pm EST. Led by leading TANGO2 deficiency disorder (TDD) researchers and healthcare professionals, this symposium provides a program of information-rich sessions spotlighting current research efforts propelling scientific advancements in TDD. This agenda will also underscore recent progress in TDD clinical care, including prospective recommendations for TDD therapeutic approaches, thereby ensuring learners are on the cutting edge of TDD treatment and management.We welcome researchers, scientists, medical professionals and families directly affected by TANGO2 deficiency disorder. At the conclusion of the symposium, participants should be able to:<ul><li>Describe the current TANGO2 deficiency disorder research landscape and its clinical relevance</li></ul><ul><li>Evaluate the clinical recommendations and pertinent published literature on TANGO2 deficiency disorder to assess its current understanding and diagnostic approaches</li></ul><ul><li>Evaluate and identify when to refer patients with TANGO2 deficiency disorder to research or clinical experts</li></ul>Don't miss this opportunity to learn more about TANGO2 deficiency disorder!

Agenda

Founder's Welcome

This talk will discuss efforts to characterize the role of TANGO2 homologs in a nematode model. Content presented will include the rationale for and process of generating a TANGO2-Deficiency Disorder (TDD) model in C. elegans, initial data showing a robust behavioral differences between wildtype and TANGO2 homolog knockout worms, as well as data potentially implicating a role for Coenzyme A, an important metabolic compound, in TDD. After participating in this session, attendees should be able to: understand the utility of C. elegans as a model system for TDD, describe a novel loss of function phenotype observed in worms lacking TANGO2 homologs and discuss recent evidence for a potential mechanism underlying TDD in the Coenzyme A salvage pathway

Investigating TDD Phenotype & Mechanism in a C. elegans Model

TANGO genes were identified for their requirement in transport & Golgi organization in metazoans. Human TANGO2 is a ~31 kDa protein that lacks a transmembrane domain. The TANGO2 protein mainly localizes to mitochondria & is enriched in mitochondrial sites that contact the endoplasmic reticulum and lipid droplets (LDs). Loss of TANGO2 function causes an increase in the size of LDs, highlighting altered neutral lipid catabolism. Lipidomic analyses of HepG2 cells also revealed quantitative changes in lysophospholipids and phospholipids levels. Moreover, the loss of TANGO2 function causes a significant increase in the levels of reactive oxygen species (ROS) and lipid peroxidation. All these features are exacerbated in cells cultured in nutrient-deprived conditions. These pleiotropic metabolic changes are consistent w/the role of TANGO2 in Acyl-CoA metabolism. Finally, we will present our ongoing studies that reinforce the physiological function of TANGO2 in controlling levels of acyl-CoA.

TANGO2 functions at the level of acyl-CoA metabolism

TANGO2 deficiency disorder has prominent and well defined cardiac phenotypes in patients. Cardiac tissues present an important model for pre-clinical screening of potential therapeutics and disease modeling. We present results on deciphering the cardiac pathophysiology of TANGO2 deficient induced pluripotent stem cell (iPSC) cardiomyocytes with systematic testing of metabolic stress conditions on electrical and mechanical functions. This study utilizes a novel, high precision platform for electrical and mechanical phenotyping of cardiac muscle bundles. We demonstrate common patient cardiac phenotypes in TANGO2 deficiency disorder are difficult to elicit in iPSC cardiomyocytes. This highlights the need to focus on methods that produce strong physiologic responses for testing potential treatments.

Modeling TDD through TANGO2 Patient Derived iPSC-CMs for Therapeutic Screening

During this virtual break, attendees are encouraged to step away from their screens, stretch their legs, and grab a refreshment of their choice. This is an opportunity to connect with fellow participants in a more casual setting, engage in discussions, and exchange insights. Use this time to network, share experiences, and build connections that can enrich your virtual conference experience. Feel free to participate in themed discussions, or simply enjoy a moment to recharge before the next session. When the break concludes, we'll reconvene for more engaging TDD content tailored for our TDD families.

BREAK

A TDD Natural History Study Update & It’s Clinical Relevance

The Therapeutic Development Branch (TDB) at the National Center for Advancing Translational Sciences (NCATS) is committed to overcoming the scientific & translational challenges facing the preclinical development of new treatments for rare diseases, including a focus on those conditions affecting small numbers of patients. TDB actively partners with the scientific & clinical biomedical community to catalyze preclinical innovations and accelerate the development of new therapies. The preclinical process can be particularly challenging for rare diseases because of limited resources & investment, lack of understanding of the natural history of disease progression to identify biomarkers & select clinical endpoints, & the deficiencies in diagnostic testing for patient identification & clinical trial design. All these factors negatively impact drug development for promising therapeutics. This talk highlights TDB’s collaborative model, driven by close interactions among researchers & patients

Paths to Treatment: Enabling Preclinical Therapeutic Development for Rare Dis

Movement disorders are a widely reported feature of TDD; however, to date, the full spectrum of movement disorders has not been systematically characterized. Accurate classification of movement disorders in TDD has the potential to improve rates of diagnosis, inform our pathophysiological understanding of the disease, & contribute to clinical trial readiness for future treatment trials. In this talk, I will review data derived from videos of 23 patients with TDD. Videos were reviewed by a group of pediatric movement disorder specialists, & group consensus was reached regarding primary & secondary movement disorders present in all patients. Dystonia & ataxia were the predominant movement disorders present. Episodes of fatigue, drooling, & decreased responsiveness (colloquially termed “TANGO2 spells”) were present in a subset of patients and were not easily characterized into any movement disorder category.

Defining Abnormal Movements in TANGO2 Deficiency Disorder

The TANGO2 protein was originally identified in a genetic screen for factors that affect transport to & organization of the Golgi apparatus, a compartment of the endomembrane system of eukaryotic cells. Since 2016, TDD has been described in numerous individuals with a variety of different mutations in the gene ranging from missense mutations to large deletions of all or some of the gene. Indeed, TANGO2 deficiency affects transport to the Golgi in human cells & a portion of the protein localizes to the mitochondria. While initially believed to be a mitochondrial disorder, TDD more closely resembles a defect in lipid metabolism. Since lipids are often conjugated to a small molecule called coenzyme A (CoA) we decided to look at the effects of changing CoA levels in a whole organism that is deficient in TANGO2. We chose the fruit fly as a model system since it shares nearly 70% of its genome with humans and can be easily manipulated through the food that they eat.

Towards an understanding of the therapeutic value of vitamin B5 in TDD

During the closing statements, our leadership team will recap key takeaways from the conference, highlighting the most impactful sessions and discussions. We will reflect on the collective wisdom shared by our esteemed speakers and the vibrant interactions among participants. Furthermore, we will share the exciting next steps planned for the foundation. Learn about upcoming events, initiatives, and opportunities to stay involved with our mission beyond this conference. Your continued support and collaboration are integral to driving our vision forward.

Closing Statements and T2RF Next Steps

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2023 TANGO2 Research Symposium

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