# haligene.com

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## Sitemaps

- [XML Sitemap](https://haligene.com/sitemap.xml): Contains all public & indexable URLs for this website.

## Posts

- [Blog Standard](https://haligene.com/blog-standard/)
- [CHKB Splicing Research: Alternative Splicing Events of Rare CHKB Variants in MDCMC](https://haligene.com/chkb-splicing-research-alternative-splicing-events-of-rare-chkb-variants-in-mdcm/): New research has uncovered how rare CHKB variants disrupt RNA splicing, directly contributing to the pathology of megaconial congenital muscular dystrophy (MDCMC). These findings offer deeper insight into how CHKB deficiency leads to mitochondrial dysfunction and severe neuromuscular symptoms.
- [Gene Therapy Strategies for Muscular Dystrophies: Current Insights and Future Directions](https://haligene.com/gene-therapy-strategies-for-muscular-dystrophies-current-insights-and-future-directions/): This review article provides an in-depth overview of gene therapy modalities — including gene replacement, gene editing, and gene inactivation — and how these strategies are shaping the future of muscular dystrophy treatment.
- [A Rare Homozygous Variant of CHKB Induced Severe Dilated Cardiomyopathy](https://haligene.com/a-rare-homozygous-variant-of-chkb-induced-severe-dilated-cardiomyopathy/): The 2024 Muscular Dystrophy Association Summit brought together international experts to discuss the scientific, operational, and ethical challenges of bringing gene therapies safely to patients. The meeting marks a major milestone in accelerating progress for neuromuscular diseases.
- [Toward an AAV Gene Therapy to Treat Muscular Dystrophies](https://haligene.com/toward-an-aav-gene-therapy-to-treat-muscular-dystrophies/): New innovations in AAV vector engineering — including dual-vector methods and enhanced tissue targeting — are opening doors for treating muscular dystrophies once considered untreatable.
- [2024 MDA Summit Highlights Safety & Innovation in Gene Therapy](https://haligene.com/2024-mda-summit-highlights-safety-and-innovation-in-gene-theraphy/): The 2024 Muscular Dystrophy Association Summit brought together international experts to discuss the scientific, operational, and ethical challenges of bringing gene therapies safely to patients. The meeting marks a major milestone in accelerating progress for neuromuscular diseases.

## Pages

- [Home Main](https://haligene.com/): Developing Gene Therapies for Neuromuscular & Metabolic Diseases ABOUT US CONTACT US Patient-Centered Focus We prioritize diseases where unmet need is greatest, especially those affecting small and overlooked patient populations. Scientific Excellence Our programs are grounded in rigorous rare-disease biology, clinical insight, and evidence-based development principles. Collaborative Model We partner with academia, health systems, and
- [Therapeutic Areas](https://haligene.com/therapeutic-areas/): Advancing Gene Therapies for Ultra-Rare Neuromuscular and Metabolic Diseases HaliGene Therapeutic Inc focuses on addressing severe, under-recognized genetic disorders that lack effective treatments. Our therapeutic programs are built on a deep understanding of disease biology, with lead efforts centred on CHKB-associated congenital megaconial muscular dystrophy and related neuromuscular conditions. CHKB Muscular Dystrophy (Congenital Megaconial Muscular
- [Mahtab Tavasoli Bio](https://haligene.com/mahtab-tavasoli-bio/): Dr. Mahtab Tavasoli is a biomedical scientist specializing in lipid metabolism, neuromuscular disease, and gene therapy development. She earned her PhD in Medicine from the University of Alberta and completed her postdoctoral fellowship at Harvard Medical School, where she advanced research in lipid biology, mitochondrial dysfunction, and neurophysiology.Dr. Tavasoli has played a central role in
- [Christopher McMaster Bio](https://haligene.com/christopher-mcmaster-bio/): Dr. Christopher McMaster, PhD, FCAHS, is the Scientific Director of the Institute of Genetics for the Canadian Institutes of Health Research (CIHR) and Professor at Dalhousie University in Halifax, Canada. Previously, he was the Head of the Department of Pharmacology, Assistant Dean for Graduate and Post-doctoral Studies, Co-director of the Cheminformatics Drug Discover Lab, and
- [Contacts](https://haligene.com/contacts/): Partnering to Advance Gene Therapies for Ultra-Rare Diseases Get in Touch Whether you’re a researcher, clinician, patient advocate, or potential collaborator, we’d love to hear from you. Connect with our team to explore partnerships, ask questions, or learn more about HaliGene’s work in developing transformative gene therapy solutions. Submit Request
- [About Us](https://haligene.com/about-us/): About Us HaliGene is dedicated to advancing transformative gene therapies for ultra-rare neuromuscular and metabolic diseases through a collaborative, science-driven, public-health approach. Meet our Leaders Who We Are About the Company Our Impact Our Promise About the Company HaliGene Therapeutics was founded with a singular purpose: to address the unmet needs of ultra-rare disease populations
- [AAV9 Delivery Technology](https://haligene.com/aav9-delivery-technology/): All Gene Therapy & Genetic Medicine Genetic & Genomic Research Metabolic Research Neuromuscular Disease Research Paediatric Rare Disease Research Neuromuscular Disease Research, Paediatric Rare Disease Research Clinical Progression & Natural History Research Neuromuscular Disease Research, Paediatric Rare Disease Research Clinical Progression & Natural History Research Metabolic Research, Neuromuscular Disease Research Muscle Biology & Pathology Metabolic
- [Eric Hoffman Bio](https://haligene.com/eric-hoffman-bio/): Dr. Hoffman has worked on DMD since the late 1980’s, first working as a post-doctoral fellow with Louis Kunkel at Boston Children’s to identify the gene and protein (1986-1990), then running his own research group at University of Pittsburgh (1990-1998), then Children’s National Medical Center in Washington DC (1998-present). He has led development of molecular
- [Laura Hagerty Bio](https://haligene.com/laura-hagerty-bio/): Dr. Hagerty has spent more than a decade pursuing therapies for muscular dystrophy. She began her career in drug development as a preclinical pharmacologist with Acceleron Pharma and later with the Skeletal Muscle Metabolism Discovery Unit at GlaxoSmithKline. She is currently the Vice President, Research of ReveraGen BioPharma Inc. In 2024, she co-founded HaliGene Therapeutics Inc.
- [Jesse Damsker Bio](https://haligene.com/jesse-damsker-bio/): Dr. Damsker has an extensive background in immunology and the regulation of inflammatory diseases. He earned his doctorate in immunology at the George Washington University School of Medicine and completed a post-doctoral fellowship at the National Institutes of Health. In 2010, Dr. Damsker brought his immunological expertise to ReveraGen and spearheaded studies that characterized vamorolone’s
- [News & Media](https://haligene.com/blog-grid/): Gene TherapyGenetic MedicineMuscular Dystrophy Gene Therapy Strategies for Muscular Dystrophies: Current Insights and Future Directions January 27, 2026 Jesse Damsker This review article provides an in-depth overview of gene therapy modalities — including gene replacement, gene editing, and gene inactivation — and how these st Read More CHKBCongenital Muscular DystrophyGenetics CHKB Splicing Research: Alternative Splicing
- [HG-001 Platform](https://haligene.com/hg-001-gene-replacement-theraphy/): All Gene Therapy & Genetic Medicine Genetic & Genomic Research Metabolic Research Neuromuscular Disease Research Paediatric Rare Disease Research Neuromuscular Disease Research, Paediatric Rare Disease Research Clinical Progression & Natural History Research Neuromuscular Disease Research, Paediatric Rare Disease Research Clinical Progression & Natural History Research Metabolic Research, Neuromuscular Disease Research Muscle Biology & Pathology Metabolic
- [Book Visit](https://haligene.com/therapeutic-areas/book-visit/): Book A Visit The information gathered can help confirm the cause of health concerns or rule out any potential concerns which are a critical tools in helping healthcare providers understand. Insurance Plans Contact Us HomeTherapeutic AreasBook Visit Book A Lab Visit Please feel welcome to contact our friendly reception staff with any general or medical
- [Provider Login](https://haligene.com/therapeutic-areas/provider-login/): Provider Login We strive to make your experience as pleasant as possible by listening to you. Email Password Remember Me!Forget Password! Login Register
- [Provider Registration](https://haligene.com/therapeutic-areas/provider-registration/): Registration The information gathered can help confirm the cause of health concerns or rule out any potential concerns which are a critical tools in helping healthcare providers understand. Tests and Services Contact Us HomeTherapeutic AreasProvider Registration Provider Registration We strive to make your experience as pleasant as possible, and the only way we know whether
- [Accreditation](https://haligene.com/therapeutic-areas/accreditation/): Accreditation The information gathered can help confirm the cause of health concerns or rule out any potential concerns which are a critical tools in helping healthcare providers understand. Values and Culture Meet our Leaders HomeTherapeutic AreasAccreditation Metabolic reactions produce these compounds from the digestion of dietary protein, fat, and carbohydrates. The resulting organic acids are
- [Shop](https://haligene.com/shop/)
- [Privacy Policy](https://haligene.com/privacy-policy/): Who we are Suggested text: Our website address is: https://haligene.com. Comments Suggested text: When visitors leave comments on the site we collect the data shown in the comments form, and also the visitor’s IP address and browser user agent string to help spam detection. An anonymized string created from your email address (also called a

## Footers

- [Default](https://haligene.com/cms-footer/default/): Quick Links About Us Therapeutic Areas Contact If you have any questions or need help, feel free to contact with our team. Email: jesse.damsker@haligene.com Copyright © 2026 Haligene Designed by Prabbis Back To Top

## Jobs

- [Chief Executive Officer](https://haligene.com/job-archives/chief-executive-officer/): A chief executive officer (CEO) is the highest-ranking executive in a company, and their primary responsibilities include making major corporate decisions.
- [Chief Financial Officer](https://haligene.com/job-archives/chief-financial-officer/): A chief executive officer (CEO) is the highest-ranking executive in a company, and their primary responsibilities include making major corporate decisions.
- [Global Sales & Marketing](https://haligene.com/job-archives/global-sales-marketing/): A chief executive officer (CEO) is the highest-ranking executive in a company, and their primary responsibilities include making major corporate decisions.

## Research

- [Muscle Biology & Pathology](https://haligene.com/research-archives/muscle-biology-pathology/): Scalable, modular gene therapy constructs engineered to accelerate programs across neuromuscular and metabolic diseases.
- [Clinical Progression & Natural History Research](https://haligene.com/research-archives/clinical-progression-natural-history-research/): Partnering with academic centres, clinicians, and patient advocates to advance therapeutic readiness and natural-history understanding.
- [Gene Replacement Technology](https://haligene.com/research-archives/gene-replacement-technology/): A first-in-class therapeutic designed to restore functional CHKB expression using rAAV9 for targeted muscle, heart, and CNS delivery.

## Services

- [Respiratory Failure in Advanced Disease](https://haligene.com/services-archives/respiratory-failure-in-advanced-disease/): Respiratory decline is the leading cause of mortality in MDCMC, typically occurring in childhood or early adulthood. As skeletal and diaphragmatic muscle weakens, individuals experience reduced lung function, increased infection susceptibility, and progressive ventilatory insufficiency.
- [Mitochondrial Megaconial Phenotype](https://haligene.com/services-archives/mitochondrai-megaconial-phenotype/): A defining feature of CHKB disease is the presence of enlarged mitochondria accumulating at the periphery of muscle fibers — the classic megaconial phenotype. This structural abnormality reflects impaired lipid metabolism and mitochondrial dysfunction, contributing directly to the degenerative nature of the condition.
- [Early-Onset Hypotonia & Motor Dysfunction](https://haligene.com/services-archives/early-onset-hypotonia-motor-dysfunction/): Infants and toddlers with CHKB-related disease often present with hypotonia, poor head control, and delayed milestones such as sitting, standing, and walking. Earliest pathology often appears in the posterior muscle compartments, with progressive involvement of anterior and medial compartments. Advanced stages show significant fatty infiltration and weakness.
- [Cardiomyopathy Associated with CHKB-Related Deficiency](https://haligene.com/services-archives/cardiomyopathy-associated-with-chkb-related-deficiency/): Around 30% of individuals with CHKB muscular dystrophy develop cardiomyopathy, as loss of CHKB disrupts cardiac muscle integrity and affects ventricular function. Some individuals progress to heart failure in adolescence or early adulthood, making targeted cardiac monitoring and supportive care essential.
- [CHKB-Related Intellectual Disability & Neurodevelopment Delay](https://haligene.com/services-archives/chkb-related-intellectual-disability-neurodevelopmental-delay/): About 96% of individuals with CHKB-related disease experience intellectual disability and delays in speech and cognitive development despite typically normal brain MRI findings. Metabolic and mitochondrial dysfunction significantly impact neurodevelopment pathways, contributing to learning challenges, delayed milestones, and functional impairment.
- [CHKB Muscular Dystrophy (Congenital Megaconial Muscular Dystrophy)](https://haligene.com/services-archives/chkb-muscular-dystrophy-congenital-megaconial-muscular-dystrophy/): Congenital megaconial muscular dystrophy is an ultra-rare autosomal recessive neuromuscular disorder caused by variants in the CHKB gene affecting phospholipid biosynthesis. Loss of CHKB activity disrupts mitochondrial structure and muscle-cell membrane composition, leading to progressive muscle weakness and developmental delay.

## Test Results

- [Patient Demo](https://haligene.com/test-results-archives/patient-demo/): This is just message to user, Administrator can edit via backend dashboard

## Categories

- [Genetics](https://haligene.com/category/genetics/)
- [CHKB](https://haligene.com/category/chkb/)
- [Congenital Muscular Dystrophy](https://haligene.com/category/congenital-muscular-dystrophy/)
- [Gene Therapy](https://haligene.com/category/gene-theraphy/)
- [Clinical Development](https://haligene.com/category/clinical-development/)
- [Neuromuscular Disease](https://haligene.com/category/neuromuscular-disease/)
- [AAV](https://haligene.com/category/aav/)
- [Cardiomyopathy](https://haligene.com/category/cardiomyopathy/)
- [Case Report](https://haligene.com/category/case-report/)
- [Muscular Dystrophy](https://haligene.com/category/muscular-dystrophy/)
- [Genetic Medicine](https://haligene.com/category/genetic-medicine/)

## Tags

- [CHKB](https://haligene.com/tag/chkb/)
- [MDCMC](https://haligene.com/tag/mdcmc/)
- [Functional Genomics](https://haligene.com/tag/functional-genomics/)
- [Safety](https://haligene.com/tag/safety/)
- [Muscular Dystrophy](https://haligene.com/tag/muscular-dystrophy/)
- [AAV](https://haligene.com/tag/aav/)
- [Alternative Splicing](https://haligene.com/tag/alternative-splicing/)
- [Clinical Strategies](https://haligene.com/tag/clinical-strategies/)
- [Duchenne Muscular Dystrophy](https://haligene.com/tag/duchenne-muscular-dystrophy/)
- [Gene Delivery](https://haligene.com/tag/gene-delivery/)
- [Therapeutic Strategy](https://haligene.com/tag/therapeutic-strategy/)
- [CHKB Mutations](https://haligene.com/tag/chkb-mutations/)
- [Cardiac Involvement](https://haligene.com/tag/cardiac-involvement/)
- [Whole Exome Sequencing](https://haligene.com/tag/whole-exome-sequencing/)
- [Gene Editing](https://haligene.com/tag/gene-editing/)
- [Viral Vectors](https://haligene.com/tag/viral-vectors/)
- [Genetic Therapeutics](https://haligene.com/tag/genetic-therapeutics/)
- [Gene Therapy](https://haligene.com/tag/gene-therapy/)

## Research Categories

- [Genetic & Genomic Research](https://haligene.com/research-category/genetic-genomic-research/)
- [Neuromuscular Disease Research](https://haligene.com/research-category/neuromuscular-disease-research-2/)
- [Metabolic Research](https://haligene.com/research-category/metabolic-research/)
- [Gene Therapy & Genetic Medicine](https://haligene.com/research-category/gene-therapy-genetic-medicine/)
- [Paediatric Rare Disease Research](https://haligene.com/research-category/paediatric-rare-disease-research/)

## Services Categories

- [Services](https://haligene.com/services-category/services/)