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Developing Gene Therapies for Neuromuscular & Metabolic Diseases
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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 patient communities to accelerate progress responsibly and efficiently.
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Gene Therapy

Building Platform Technologies to Deliver Tomorrow’s Gene Therapies

HaliGene Therapeutics Inc is developing a gene therapy framework designed for diseases that impact skeletal muscle, cardiac function, and central nervous system integrity. Our approach combines vector engineering with deep rare-disease biology to deliver precise, durable therapeutic solutions — including our lead program targeting CHKB congenital megaconial muscular dystrophy.

Via a public-health lens, we focus on patient populations that sit between bespoke academic solutions and commercially tractable pharma programs.

Understanding CHKB Congenital Megaconial Muscular Dystrophy

25+ Years Of Experience
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CHKB congenital megaconial muscular dystrophy (MDCMC) is a progressive, autosomal recessive neuromuscular disorder caused by rare variants in the CHKB gene, which encodes the choline kinase beta enzyme — essential for phospholipid biosynthesis. Loss of CHKB activity disrupts muscle-cell structure, leading to weakness, impaired mobility, and megaconial mitochondrial enlargement.
Key Clinical Characteristics:
Progressive muscular dystrophy (100% of known patients) Intellectual disability and speech delay (96%) Cardiomyopathy in a subset (30%) Onset between 38 days and 16 years, most often ages 2–4 Muscle weakness, hypotonia, delayed motor milestones Fatty replacement and megaconial phenotype in advanced disease Supportive care only; no curative therapies available
1+
Documented CHKB
cases worldwide
1%
of patients experience speech
or cognitive challenges
1%
experience cardiac involvement
1000%
disease-modifying
therapies currently available
Our Therapeutic Focus

CHKB Muscular Dystrophy
(Congenital Megaconial Muscular Dystrophy)

CHKB muscular dystrophy is an ultra-rare, progressive neuromuscular disorder caused by loss of CHKB enzyme activity, leading to impaired mitochondrial function and muscle-cell integrity. Individuals commonly experience early hypotonia, delayed motor development, intellectual disability, and, in many cases, cardiomyopathy. A defining feature is the megaconial mitochondrial phenotype, reflecting severe cellular energy disruption. As the disease advances, respiratory decline becomes the primary life-limiting complication. Together, these features make CHKB muscular dystrophy a complex, multi-system disorder with urgent unmet therapeutic needs.

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