WODC 2021

Complete Coverage

First Genetic Identifier for Autism Found

GeneRide: A Novel Gene Editing Approach for Rare Disease Patients

NATIONAL HARBOR, Maryland — Gene editing is one of the more recent novel approaches in the world of rare disease therapies. At the 2021 World Orphan Drug Congress, LogicBio Therapeutics presented its innovative nuclease-free approach to gene editing called GeneRideTM, which involves a synthesized viral vector to deliver the corrective transgene to a precise and…

European Regulator Outlines EMA’s Progress on Orphan Drug Approvals

NATIONAL HARBOR, Maryland — The top official for rare disease therapies at the European Medicines Agency (EMA) said her office is seeing a steadily increasing number of applications for new therapies despite the disruptions caused by the COVID-19 pandemic. Violeta Stoyanova-Beninska, MD, PhD, chairs the Committee for Orphan Medicinal Products (COMP) at EMA, the European counterpart to the…
Experimental Therapies for Hemophilia

Generation Bio Pursues Redosable Gene Therapy for Hemophilia A

NATIONAL HARBOR, Maryland — One of the biggest drawbacks of gene therapy for rare diseases, besides the price factor, is that it’s generally considered a “one-and-done” that offers no opportunity for redosing as the patient grows. But Generation Bio, a preclinical biotech company based in Cambridge, Massachusetts, thinks it may have found a way around that.…

Ensuring Access to Therapies for All Rare Disease Patients

NATIONAL HARBOR, Maryland — Access to therapies for rare disease patients varies greatly from one region of the world to another, with ultra-rare diseases generally requiring ultra-expensive drugs to treat them — if a therapy is available at all. On August 27, 2021, the final day of the World Orphan Drug Congress USA 2021, a trio…
multiple sclerosis social support

Perspectives From a Rare Disease Diversity, Equity, and Inclusion Initiative

NATIONAL HARBOR, Maryland — The “highest quality science is inclusive,” Nadia Bodkin, PharmD, founder of the Rare Advocacy Movement (RAM), told participants of a roundtable discussion at the 2021 World Orphan Drug Congress. RAM sponsors and encourages patient activism and collaboration at a grassroots level. One such collaborative effort was the Our Lives Matter (OLM)…

Genome Editing: Making a Treatment of the Future a Reality Today

NATIONAL HARBOR, Maryland — “Technology is running ahead of regulatory understanding, but science fueled by genomics will drive it and address close to 80% of rare diseases,” Mo Heidaran, PhD, vice president of technical and regulatory consulting at Parexel International Biotech, said in a session at the 2021 World Orphan Drug Congress. The majority of…
sma inheritance

The Evolution of Clinical Development for Rare Diseases

NATIONAL HARBOR, Maryland — Clinical development in the world of rare diseases is “on the verge of tremendous progress, with increased understanding of inherited and acquired genetic diseases,” Janet Woodcock, MD, acting commissioner of the US Food and Drug Administration (FDA) said at the 2021 World Orphan Drug Congress, as she answered questions posed by…

Sanofi Executive Optimistic on Future of Rare Disease Treatment

NATIONAL HARBOR, Maryland — Despite the ravages of COVID-19, the future has never looked brighter for the world’s estimated 300 million people with rare diseases, a top executive at Sanofi Genzyme suggested. Shannon Resetich, the company’s global franchise head for rare diseases, spoke on August 26, 2021, at the opening of the World Orphan Drug…

Phenopackets: Interpreting Rare Disease Phenotypes With Machine Learning

NATIONAL HARBOR, Maryland — One of the commonly used phrases in the rare disease community describes a patient’s “diagnostic odyssey” – a term that denotes the length of time, number of providers, frequent misdiagnoses, and emotional turmoil that accompanies the journey to obtaining an accurate diagnosis of a rare disease. Multiple organizations, researchers, scientists, curators,…

Rare Disease Advisor is proud to serve as an official Media Partner for the 2021 World Orphan Drug Congress, which is being held August 25-27, 2021, in National Harbor, Maryland. Stay tuned to RDA as we offer live, comprehensive coverage of the event, plus expanded coverage featuring exclusive stories and interviews with rare disease leaders and influencers.

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