TRNDS 2019 Sponsors

Industry-leading TRNDS sponsors represent innovative companies and organizations that share a common vision and mission: to advance therapy development for rare neurological diseases.

TRNDS 2019 Sponsors

About Abeona Therapeutics
Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases. Developing therapies for rare disease requires new approaches and strong collaboration between researchers, industry, regulators and patient groups. With expertise in Research and Design, integrated manufacturing and novel gene and cell therapy development, Abeona is uniquely poised to bring new treatments to patients in need. Abeona strives to deliver gene and cell therapies to people affected by disease by harnessing the promise of genetic medicine.

About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company’s portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.

TRNDS 2019 Sponsors

About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare, debilitating, chronic neuromuscular and neurological diseases. Catalyst Pharmaceuticals’ mission is to improve the lives of patients with rare and ultra-rare diseases by partnering with patients to empower them to take an active role in their health care.

TRNDS 2019 Sponsors

About REGENXBIO
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

TRNDS 2019 Sponsors

About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We have successfully applied our technology in the first gene therapy approved in both the U.S. and EU for a genetic disease, and currently have four programs in clinical trials. At Spark, we see the path to a world where no life is limited by genetic disease.

TRNDS 2019 Sponsors

About University of Rochester Center for Health + Technology (CHeT)
Over the past generation, CHeT has re-shaped the conduct of clinical research and advanced knowledge to improve health for thousands, if not millions, of individuals. CHeT is leading the field in evaluating how to harness the tools and technologies of the early 21st century to extend the reach and impact of research and clinical care. CHeT seeks to leverage Technology to Advance Care + Research by applying new tools and approaches in clinical trials to transform the design, assessment, and evaluation of novel therapies for neurological disorders. CHeT has been involved in over 111 clinical trials, enrolled over 40,000 research participants and has contributed to 7 FDA-approved treatments. CHeT collaborators and partners span the globe and include leading academic institutions, industry innovators in pharma, technology, not-for-profit foundations, advocacy groups, and the federal government.

TRNDS 2019 Sponsors

About University of Rochester Clinical and Translational Science Institute (CTSI)
The University of Rochester Clinical and Translational Science Institute (CTSI) develops, demonstrates and disseminates methods and approaches to advance translational research, by providing education and training, supporting transdisciplinary teams, improving quality and efficiency, and engaging community stakeholders. Since its inception in 2006, the CTSI has provided pilot funding and training that helped researchers and students secure approximately $58 million in additional external funding to advance their studies.

TRNDS 2019 Sponsors

About University of Rochester Del Monte Institute for Neuroscience
The Del Monte Institute for Neuroscience brings together faculty across the University of Rochester who are engaged in neuroscience research. The central purpose of the Del Monte Institute is to reduce the burden of neurological and neuropsychiatric disease in our society through development of basic scientific knowledge and of novel therapeutic approaches. The Institute exists to recognize new and emerging opportunities, to prosecute an aggressive innovative basic and translational research agenda, to build team science that tackles major problems from multi-methodological and multi-scalar perspectives, to tackle fundamental neuroscientific questions that simply cannot be broached at the level of an individual laboratory or investigator, questions that demand concerted multi-investigator teams.

TRNDS 2019 Sponsors

About University of Rochester Department of Neurology
The University of Rochester Department of Neurology is committed to providing quality patient care, conducting research to generate knowledge that will alleviate human suffering through prevention and treatment of disease and educating physicians in accordance with highest professional standards. UR Neurology has an incredible team of neurologists, scientists, nurses, social workers, neuropsychologists, therapists, trainees and staff who’s focus is making a difference in the lives of people with a neurological disorder.

TRNDS 2019

University of Rochester
Saunders Research Building
Helen Wood Hall Auditorium
255 Crittenden Blvd
Rochester, NY 14642

Date: Friday, September 13
Time: 7:30 am – 4:00pm

TRNDS News

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  • New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
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  • Neurogene Offers Access to Genetic Testing for Lysosomal Storage Disease
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  • NIH launches 5-year, $10 million study on acute flaccid myelitis
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  • Study refines ALS risk among first-degree relatives of patients with disease
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  • NCATS Director Warns of 'Poorly Understood Public Health Implications of Rare Diseases'
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  • Speeding Up Drug Discovery for Brain Diseases
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  • NIH-Funded Project Aims to Build a ‘Google’ for Biomedical Data
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  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
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  • Jsyne Gershkowitz, Amicus Therapeutics, TRNDS 2019
    (Podcast) Get to know Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics
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  • PJ Brooks, NCATS, NIH, TRNDS
    Meet TRNDS 2019 Speaker PJ Brooks
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  • Rare Pediatric Disease PRVs: FDA Updates Guidance
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  • Batten Disease Study
    Neurogene Opens Natural History Study of CLN7, CLN5 Diseases at UT Southwestern
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  • C-Path and NORD Collaborate to Launch Rare Disease Data Platform
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  • TRNDS 2019 speaker Tauna Batiste, BDSRA
    Meet TRNDS Speaker Tauna Batiste
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  • HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials
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  • Evidation & Eli Lilly Study Uses Apple Devices, Apps to Predict Cognitive Impairment
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  • 'Dr. Google' Helps Some Patients Diagnose a Rare Disease
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  • Antisense Drugs for HD, ALS & Prion Could Meet the Need for Brain Treatments
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  • Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome
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  • Netflix's Medical Investigation Docuseries 'Diagnosis'
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  • NIH All of Us Project Tops 270,000 Sign-ups
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  • FDA Cancer Office Taps Syapse for Real-world Evidence Development
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  • Gene Linked to Rare Neurological Disorder May Play a Role in Alzheimer’s
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  • Gene Therapy Developers Issue Principles for Human Genome Editing
    Gene Therapy Developers Issue Principles for Human Genome Editing
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  • Forget Single Genes: CRISPR Now Cuts & Splices Whole Chromosomes
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  • Benjjamin Schlatka MC10 TRNDS
    TRNDS Speaker Benjamin Schlatka, MC10
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  • TRNDS Danielle Edwards Jett Foundation
    TRNDS Speaker Danielle Edwards, Jett Foundation
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  • His daughter has a rare disorder. He’s developing a novel gene therapy.
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  • A Doctor Tested a New Treatment on Himself, Now it's Saving Other Lives
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  • Study Presents Clinical, Genetic Landscape of Pompe Disease
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TRNDS Speakers

Room Block

Hilton Garden Inn
Rochester/University
Medical Center

30 Celebration Drive
Rochester, NY 14620

Phone: 585-424-4404

TRNDS Rate: $149/night