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Articles by Sharon Hesterlee
Steven W. Pipe, MD, on the Implications of the Final Data From Hemgenix’s HOPE-B Trial
“I think for patients who are interested in a genetic therapy like this, who want to be liberated from prophylaxis, this is really the only approach that really delivers that.
Rabi Hanna, MD, on Practical Considerations for Integrating Gene Therapy
“I cannot emphasize how much misinformation there is on gene therapy.
End-of-Study Results From Hemgenix’s HOPE-B Trial
At the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, end-of-study data from the pivotal phase 3 HOPE-B clinical trial (NCT03569891), which evaluated CSL Behring/uniQure’s etranacogene dezaparvovec (marketed as Hemgenix) for the treatment of severe or moderately severe hemophilia B, were reported.
Evaluating Gene Editing Therapy Reni-Cel for Severe Sickle Cell Disease
There are currently 2 FDA-approved gene therapy treatments available for sickle cell disease (SCD)—Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia)—but development of new genomic medicine options for SCD remains ongoing with the aim of filling in gaps in need unserved by the current options.
CGTLive®’s Weekly Rewind – December 12, 2025 | CGTlive®
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. AZD0120, a dual-targeted CAR T-cell therapy, shows high efficacy in treating relapsed multiple myeloma, achieving a 96% response rate.
Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration
This interview originally appeared on our sister site, NeurologyLive®. "Our goal is really about community—bringing together all these different facets, from clinical care to research, to create incredible momentum." The Muscular Dystrophy Association (MDA) will hold the annual MDA Clinical and Scientific Conference for 2025 from March 16 to 19 in Dallas, Texas.
Sharon Hesterlee, PhD, on What Attendees Can Look Forward to at MDA’s 2024 Conference
“I think it's a really exciting time right now in the world of neuromuscular disease.
Sharon Hesterlee, PhD, on Collaborating to Expand Therapy Development to More Dystrophies
“There are so many rare diseases that don't have big commercial markets. We see a lot of activity [with muscular dystrophies], it's been growing exponentially around Duchenne and some of the more common ones. But we have this big portfolio of dystrophies. So we've launched an internal program that we call MDA Kickstart, and we basically are operating it like a gene therapy company.
Sharon Hesterlee, PhD, on Further Research Needs With Muscular Dystrophies
“I think we're going to have to develop some new strategies for people who progress past the therapeutic window. There are also plenty of other diseases, muscle diseases and other types of neuromuscular disease, for which therapies aren't as advanced. Sometimes, because it's a particularly tricky thing to tackle, like a dominant mutation in which gain of function can be a little tricky...
Sharon Hesterlee, PhD, on Unprecedented Progress in Muscular Dystrophies
“I think the message is that we're moving into the treatment era. For so long, we were just at a stage where we could diagnose people and follow them. And now we're actually at the point where there are treatment options, and there are going to be treatment options available for many people. So it won't be a case of, if your exon matches this drug, there's a treatment, I think we're going to have enough variety of options that everyone is going to have the potential to benefit.
06.06.22 -- New Clinical Studies In ALS Show Promise
By Kamila Novak, KAN Consulting We tend to forget to talk about the knowledge and skill silos in clinical trials that we have built over the years. This article describes how the industry got to where we are with narrow specialization and recommends five strategies to break skill silos.
New Clinical Studies In ALS Show Promise
By Sharon Hesterlee, Ph.D., Muscular Dystrophy Association There are approximately 17,000 people in the United States living with the progressive neuromuscular disease amyotrophic lateral sclerosis (ALS). The exact cause for ALS is still unknown; about 10%-15% of cases have known genetic causes (familial ALS), while the remaining cases have no clear cause (sporadic ALS).
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