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Andre H. Goy, MD; Sandra M. Swain, MD, FACP, FASCO Associate Dean for Research; Carolina Bernabe Ramirez, MD; Jonathan E. Rosenberg, MD; David H. Vesole, MD, PhD, FACP Director, Myeloma Program; Isabel Preeshagul, DO, MBS  
 

Community Practice Connections™: 5th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Karen M. Winkfield, MD, PhD; Narjust Florez, MD, FASCO; Gilberto de Lima Lopes Junior, MD, MBA, FASCO; Coral Olazagasti, MD; Vonetta M. Williams, MD, PhD

 Fighting Disparities and Saving Lives: An Exploration of Challenges and Solutions in Cancer Care

 Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. It also contains up-to-date clinical news coverage in the field of gene and cell therapy for central nervous system disorders.

Articles

Supported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.

CALD Gene Therapy Granted Breakthrough Therapy Designation

Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.

First Duchenne Patient Dosed in Pfizer's Mini-Dystrophin Study

Parent Project Muscular Dystrophy announced that the first patient with Duchenne muscular dystrophy has been dosed with microdystrophin gene therapy.

Gene Therapy Trial Doses First DMD Patient

William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.

Targeting Progranulin With Gene Therapy for Frontotemporal Dementia

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. 

Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024

Furthermore, the majority of patients treated with the gene therapy showed improved myelination as measured by T-2-weighted MRI.

BridgeBio Pharma’s Gene Therapy BBP-812 Effects Progressive Improvement in Motor Function in Patients With Canavan Disease

Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.

Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy

Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

MDA 2025 as a Mixer for Strategies and Collaboration

This interview originally appeared on our sister site, 

The three experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.

R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy for APOE4 Homozygous Alzheimer Disease

"APOE4 homozygotes have no good treatment options right now, and this trial offers a potential treatment that provides hope for patients and their families.”

Risk for Alzheimer disease (AD) is known to be strongly associated with apolipoprotein E (

) polymorphisms. Research demonstrates that people homozygous for 

 have a 14.5-fold higher risk for late-onset AD than those who are homozygous for 

. In addition, those who are heterozygous for 

 are at a 2.6-fold increased risk, thus pointing to a the potential of a protective role for 

. Lexeo Therapeutics' LX1001, an investigational adeno-associated virus vector-based gene therapy, is intended to provide a copy of the 

 gene to the cells of the central nervous system in patients with AD who are homozygous for 

. As such, the approach is expected to convert the genotype of transduced cells to 

 and to in turn slow disease progression.

LX1001 is currently being evaluated in a phase 1/2, open-label, dose-finding clinical trial (NCT03634007). Data from the study presented at the 2024 Clinical Trials on Alzheimer’s Disease (CTAD) conference, held October 29 to November 1, in Madrid, Spain, showed that LX1001 was generally safe and well-tolerated, and demonstrated a dose- and time-dependent effect on expression of 

. Furthermore, a reduction in several cerebrospinal fluid (CSF) AD tau biomarkers and changes on Tau PET suggested a treatment effect of the gene therapy on tau, which researchers note is a critical component in the pathobiology of AD.

At CTAD 2024, R. Nolan Townsend, chief executive officer at Lexeo, coauthor Sandi See Tai, MD, chief development officer at Lexeo, and lead author Kim G. Johnson, MD, the division chief of memory disorders at Duke University, sat down with 

 to speak about some of the key safety findings from the trial. During the conversation, the trio also talked about how the 

 gene delivery impacted amyloid and tau biomarkers in trial participants, as observed in the study findings. Additionally, the experts spoke about the significance of targeting those homozygous for 

 with gene therapy in the AD treatment landscape.

REFERENCES
1. Johnson K, et al. Safety and Preliminary Efficacy of AAV Gene Therapy (LX1001) in Patients with APOE4 Homozygote Alzheimer’s Disease – Interim Data from a Phase 1/2, Open-Label, 52-Week, Multicenter Study. Presented at: 2024 CTAD; October 29-November 1; Madrid, Spain. Abstract 486.
2. Lexeo Therapeutics Announces Positive Interim Data for LX1001, First-Ever Gene Therapy to Impact the Underlying Genetic Cause of APOE4-Associated Alzheimer’s Disease, at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference. News Release. Lexeo Therapeutics. Published October 30, 2024. Accessed October 20, 2024.

Videos

The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease. 

The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration

"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. The 4-day event will gather scientific and medical professionals, patients, and advocacy groups to discuss and share the latest advances in the field of neuromuscular disorders. Research pertaining to the range of neuromuscular disorders, from amyotrophic lateral sclerosis (ALS), to Duchenne muscular dystrophy, to spinal muscular atrophy, to limb-girdle muscular dystrophy, to myasthenia gravis, to Charcot-Marie-Tooth disease, and more will be represented at the meeting.

Furthermore, there will be session tracks featured on topics including strategies for regenerating muscle and nerve tissue, new insights into disease mechanisms, clinical experience in gene therapy, access and reimbursement, and clinical trial readouts. Notably, MDA's 2024 Clinical and Scientific Conference was the largest global gathering of the neuromuscular disease community, with 1492 in-person and 567 virtual attendees from over 30 countries. Those who are registered with MDA may partake in the virtual meeting in 2025 at no-cost or may register in-person at the patient/caregiver rate until allotted spots are filled.

In anticipation of the upcoming meeting, 

, reached out to Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association, to discuss the primary focus areas of the 2025 conference, particularly in muscle regeneration and ALS research. Hesterlee also spoke about how the conference addresses the challenges of workforce diversity and training in neuromuscular specialties. She also talked about the roles of advocacy groups and allied health professionals in shaping the discussions at the upcoming meeting.

Click here to register for the upcoming 2025 MDA Conference

REFERENCE
1. Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX. News Release. MDA. July 30, 2024. Accessed October 24, 2024. https://www.mda.org/press-releases/2024/registration-now-open-for-the-2025-mda-clinical-and-scientific-conference-featuring-the-latest-innovations-in-neuromuscular-disease-research-and-care-with-global-leaders-march-16-19-in-dallas-tx

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the growing interest in gene therapy for attendees of the annual meeting.

Paul Melmeyer, MPP, on the Importance of Gene Therapy for MDA’s Upcoming 2025 Conference

“The field is learning at lightspeed pace about gene therapies... It's really quite an amazing time to be a clinician in neuromuscular diseases, learning more about how to administer gene therapies and how these gene therapies that are approved by the FDA actually work for those with neuromuscular conditions.”

Each year, the Muscular Dystrophy Association (MDA), an organization dedicated to serving the community of patients with muscular dystrophies and other neuromuscular disorders, holds the MDA Clinical & Scientific Conference. The event brings together practicing clinicians, medical researchers, and members of patient advocacy groups under one roof to discuss and share ongoing research in the field. Notably, this year, the event will be held on March 16-19, 2025, in Dallas, Texas.

In early anticipation of the upcoming conference, 

 has gotten in touch with Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, to learn more about the growing importance of the annual meeting to the field of gene therapy and the significance of the conference for the aforementioned interested groups. Melmeyer emphasized the importance of the conference for advocacy groups, around 40 to 50 of which are likely have a presence at the conference. Furthermore, he noted that members of these advocacy groups would convene for a meeting referred to as the Neuromuscular Advocacy Collaborative focused on public policymaking goals intended to serve their patient communities.

Melmeyer also spoke about the growing role of gene therapy in the field of neuromuscular disease, noting that investigational gene therapy candidates, Sarepta Therapeutics’ FDA approved gene therapydelandistrogene moxeparvovec-rokl (marketed as Elevidys) for Duchenne muscular dystrophy, and Novartis’ FDA approved gene therapy onasemnogene abeparvovec-xioi (marketed as Zolgensma) for spinal muscular atrophy will be major focuses at the 2025 MDA conference. He concluded by discussing what he personally is looking forward to at the upcoming conference.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed several still-investigational drugs and therapies for DMD.

John Brandsema, MD, on Potential Upcoming Treatments for Duchenne Muscular Dystrophy

“The bottom line is that we don't think that we are done at all in terms of the approach to this disease. There's much work still to be done in optimizing care and many research trials ongoing because we want to do the best we can for impacting this disease.”

A seminal moment for the treatment of Duchenne muscular dystrophy (DMD) occurred in June 2023 when the FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy, for use in the treatment of the disorder. Although an important step forward that gets at the root cause of the disease, it is not a perfect treatment nor is it curative. As such, many companies and academic institutions are continuing to develop and evaluate new investigational products for DMD that may help to provide better efficacy and safety for some patients, or otherwise address remaining needs that are unmet by Elevidys.

 recently asked John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, whether there are any noteworthy therapeutic candidates for DMD still in development now that may be promising for the future. Brandsema discussed the potential of several new exon-skipping therapies, small molecule drugs like sevasemten, mesenchymal stem cell therapies, and new gene transfer approaches that encode different versions of microdystrophin and/or different AAV capsids. In particular, he noted that these alternative gene transfer therapies may allow for the treatment of patients who would not be eligible to receive Elevidys.

In addition, Brandsema spoke about the importance of establishing standardized newborn screening practices for DMD. He stressed that such practices are useful because earlier treatment, before the disease has caused much deterioration of muscle, is more likely to provide better outcomes. Although, he also pointed out that the optimal timing for different treatments and/or combination therapies is not yet known and more observation is needed.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

John Brandsema, MD, on Navigating the New Landscape of Care for DMD

“If you receive gene transfer with Elevidys, for example, would we continue exon skipping for those who are eligible for it? There's a scientific argument for me why that might be valid, but that may not be approved by the third-party payer of that patient.”

On June 20, 2024, the FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), for an expanded indication, making it an option for ambulatory patients (via traditional approval) and nonambulatory patients (via accelerated approval) with a confirmed mutation in the DMD gene who are 4 years of age or older and who do not have any deletion in exon 8 or exon 9 in the gene. As a result of this decision, many more patients became eligible for the gene therapy than were previously, thus adding to the number of FDA-approved treatment options many individual patients can choose between.

 recently interviewed John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, to learn more about how doctors, patients, and their families are now navigating the treatment landscape for DMD in the real world setting. Brandsema discussed several aspects of the treatment landscape that continue to make the decision-making process challenging, such as the need to balance the benefits and risks of a diverse array of approaches to treatment; the lack of data on gene therapy in older patients; and the difficulties of implementing combination treatments, both from the perspective of insurance coverage and from the scientific perspective of monitoring for potential adverse outcomes. With regard to the latter, Brandsema emphasized the friction between a staggered approach to combination treatments, for the sake of scientific rigor, and the desire to give patients everything that might help them as soon as possible in the face of a disease that causes degeneration in muscles over time.

Byrne discussed the gene therapy for DMD.

Making Decisions for DMD Treatment

“The decision process for families is actually quite challenging now because while there are various studies that children can enter into for new therapeutic modalities, there are also a host of approved therapies now that are available and the treatment landscape continues to broaden. We'll see new approaches coming frequently and it's really incumbent upon every provider to remain familiar with all the options that are available to patients and help guide them as to what's best for their child and their family."

Patients with Duchenne muscular dystrophy (DMD) today have the choice between a number of disease-modifying treatment options, such as the several FDA-approved exon-skipping therapies and adeno-associated virus (AAV) vector-based gene therapy in the form of Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys). Beyond FDA-approved options, patients also have the choice to enroll in one of the many currently ongoing trials testing new investigational drugs, gene-editing approaches, and other experimental therapies. As such, the choice facing patients, families, and their doctors is more complicated than ever for DMD.

 recently interviewed Barry J Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida, to get his insight on the navigation of this process for the aforementioned groups. Byrne discussed the current landscape of care and how Elevidys has altered that landscape. He also spoke about the work of DMD advocacy groups like MDA and 

 in distributing important information to patients and families for educational purposes. Byrne additionally discussed several promising investigational treatments that are being evaluated by various companies and institutions right now and pointed out that the potential for combination treatments may expand the breadth of decision-making even further.

The chief medical advisor of Muscular Dystrophy Association (MDA) gave his view on treatment decision-making in the world of new options for DMD.

Barry J Byrne, MD, PhD, on Making Decisions in the Face of Rapidly Expanding Treatment Options

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.

John Brandsema, MD, on Balancing Risks and Rewards With Muscular Dystrophy Gene Therapy

“While the label is broad and is for everyone we don't necessarily have the data to back that up yet and that makes the conversation more nuanced. We also aren't sure about the durability of transgene expression. Muscle is a high turnover tissue, and therefore whether this is going to be a lifelong effect is still up in the air; although it does seem, from the early boys treated that there is a measurable difference in their trajectory years after the dose, up to 5 or 6 years at this point now.”

Over the past 5 years or so, the treatment landscape for Duchenne muscular dystrophy (DMD) has rapidly evolved with the arrival of a number of new disease-modifying therapies that have been approved by the FDA. Notably, just last year, Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy, was approved by the FDA, adding to other options available to patients such as exon-skipping therapies. As such, the decision-making process for treatment for DMD for patients, families, and doctors is now more complex than ever.

In the lead up to World Duchenne Awareness Day, which was held on September 7, 

 reached out to John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, to get his view on the newly emerging landscape of care for DMD and the real-world experience gained with Elevidys in the time since it’s approval a bit more than a year ago. Brandsema briefly discussed the other treatment options for DMD that have come onto the scene in the past 5 years, and then brought the focus to Elevidys, and spoke about some of the distinct advantages and unique risks that it carries as the only AAV vector-based gene therapy currently approved for DMD. He emphasized the need for more long-term data, especially for older patients, as many patients treated thus far have been on the younger end of the spectrum.

This interview originally appeared on our sister site, NeurologyLive.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, discussed the enhancements to newborn screening for the disease.

Crystal Proud, MD, on Improving Newborn Screening for Spinal Muscular Atrophy

"The goal would be to treat as soon as possible... every day that a child with SMA goes untreated is a day where we could potentially be losing very valuable motor neurons."

Spinal Muscular Atrophy (SMA) Awareness Month is observed annually in August by the patient and clinician communities with the intent of bringing more attention to the genetic disorder, which affects motor neurons in the spinal cord and leads to muscle wasting and weakness. SMA Awareness Month came into being thanks to the work of advocacy groups, with Cure SMA playing a particularly important role, and is intended to help support research and promote early diagnosis and treatment, in addition to bringing more attention to SMA in general, which is considered one of the most common rare diseases, affecting as many as 10,000 to 25,000 children and adults in the United States.

Notably, an FDA-approved gene therapy product, onasemnogene abeparvovec (Zolgensma; Novartis), is available for the treatment of SMA.  An adeno-associated virus vector (AAV)-based gene therapy, Zolgensma is 1 of several treatment options currently available for the disease, with others including nusinersen (Spinraza; Biogen), an SMN-enhancing therapy administered via lumbar puncture, and risdiplam (Evrysdi; Genentech), an orally administered SMN-enhancing medication for those aged 2 months and older with the disease. In general, treating patients as early as possible is thought to improve outcomes, and as such, routine newborn screening for the disease is critical.

® sat down with Crystal Proud, MD, the director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, to discuss aspects of awareness for the disease that have improved over the years, particularly in newborn screening. In addition to summarizing the progress made, she stressed the continued efforts needed to enhance the screening process, and pivotal research initiatives going forward. Proud also spoke on some of the inconsistencies with the post-screening process and the ways to efficiently ensure access to novel, disease-modifying treatments.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA. 

William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia 

“Every patient who gets any modality, there is going to be variability in how high their target engagement is and how high their progranulin gets. If you're on a statin, it may work better for some people, not as well for other people. If, on average, we can get to much higher levels, the variability patient to patient is going to be around a much higher level. Therefore, our odds of being over that threshold of clinical effectiveness go way up. And there is no known toxicity from having high levels of progranulin.”

Passage Bio is set to expand eligibility for its phase 1/2 upliFT-D clinical trial (NCT04747431) evaluating investigational gene therapy PBFT02 in patients with frontotemporal dementia (FTD), following positive FDA feedback in a Type C meeting. The trial will expand from including patients with FTD with GRN mutations (FTD-GRN) to also include patients with FTD with mutations in the C9orf72 gene (FTD-C9orf72).

Passage has shared data from the first 3 patients treated in upliFT-D last December. These participants experienced a 3.6 to 6.6-fold increase in cerebrospinal fluid (CSF) PGRN over baseline at 30 days posttreatment to reach 10.7 to 17.3 ng/mL and sustained their supraphysiologic PGRN levels in the CSF at 6 months after receiving the gene therapy, compared with 61 healthy adults used as a control, whose CSF PRGN was measured at 3.3 to 8.2 ng/mL.

spoke with William Chou, MD, president and chief executive officer of Passage Bio, to learn more about PBFT02, its mechanism, the role of progranulin, and how the therapy may help address unmet needs in people with FTD. He expanded on the progress of the upliFT-D study and emphasized upcoming data that will be presented later in the year.

1. Passage Bio announces positive feedback from FDA on expansion of upliFT-D trial of PBFT02 to include FTD-C9orf72 patients. News release. Passage Bio, Inc. July 16, 2024. Accessed July 19, 2024. https://www.passagebio.com/investors-and-news/press-releases-and-statements/news-details/2024/Passage-Bio-Announces-Positive-Feedback-from-FDA-on-Expansion-of-upliFT-D-Trial-of-PBFT02-to-Include-FTD-C9orf72-Patients/default.aspx
2. Passage Bio announces promising initial data from phase 1/2 clinical trial of PBFT02 in FTD-GRN and updated strategic priorities. News release. Passage Bio, Inc. December 20, 2023. Accessed July 19, 2024. https://www.passagebio.com/investors-and-news/press-releases-and-statements/news-details/2023/Passage-Bio-Announces-Promising-Initial-Data-From-Phase-12-Clinical-Trial-of-PBFT02-in-FTD-GRN-and-Updated-Strategic-Priorities/default.aspx

The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio

“The key takeaway is that we can rescue methylation on that particular locus in the genome and that correlates very well and that translates very well into a suppression of the toxic gene target that is the cause of that disease—a complete reduction of cell death or apoptosis and eventually a rescue of muscle function. Those are the key takeaways from the product that starts with the root cause, the methylation, and all of the positive effect that restoring methylation has on that disease.”

Facioscapulohumeral muscular dystrophy (FSHD) is a rare form of muscular dystrophy that currently has limited treatment options and no cure. As such, the unmet need is very great for the approximately 800,000 people worldwide with FSHD. Although aspects of the disease can be heterogeneous, FSHD is linked to muscle wasting caused by overxpression of the gene 

, which in turn is thought to be caused by lower than normal levels of DNA methylation in the D4Z4 microsatellite region of chromosome 4.

Notably, Epic Bio is currently developing EPI-321, an investigational epigenetic gene therapy product intended to treat FSHD by restoring methylation of the aforementioned DNA region to normal levels. EPI-321 is a CRISPR-based system delivered by an adeno-associated virus vector and functions by adding a methylation mark. The company recently presented preclinical results obtained in several different models at 

the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

, held May 7 to 10, 2024, in Baltimore, MD.

 spoke with Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio, to learn more about EPI-321 and the findings that were presented. Collin de l’Hortet explained how EPI-321 targets FSHD, which is epigenetic in nature, and noted that positive downstream effects were observed in patient cell lines. She also spoke about the challenges related to working in FSHD and future plans for the company’s platform.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

REFERENCE
1. Smith LA, Borrman T, Silvis MR, et al. Validating EPI-321 safety for FSHD: A comprehensive off target characterization platform for epigenetic gene therapies. Presented at: ASGCT Annual Meeting 2024, May 7-10; Baltimore, Maryland. Abstract #258

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