R&D Pipeline

Bgx 1: Celiac Disease

A chronic autoimmune disorder

Celiac disease is a chronic autoimmune disorder that requires a strict, lifelong gluten-free diet as the only effective treatment. Compliance with the gluten-free diet leads to resolution of symptoms and mucosal healing of the small intestine in the majority of patients.[1] The standard of care success rate varies based on disease severity, duration, patient age, and adherence to the diet. Studies show that 82% to 95% of patients adhering to a gluten-free diet experience significant improvement in symptoms and small intestinal histology.[2] Additional therapies such as immunosuppressive drugs or nutritional supplements may be necessary for refractory celiac disease cases.[3] While the standard of care has a high success rate, there is a need for ongoing research to improve efficacy and better understand the disease mechanisms.[4]

Bgx 2: Mitochondrial disease

Various types of mitochondrial dysfunction

Mitochondrial disease is a generic term for diseases that are a result of mitochondrial dysfunction. These diseases are mainly characterized by a decrease in energy that is normally delivered by the mitochondrion and are generally caused by acquired mutations in the mitochondrial DNA, that is originally always derived from the mother.

The symptoms of mitochondrial disease can vary. It depends on how many mitochondria are defective, and where they are in the body. Each person can have a different mixture of healthy and defective mitochondria, with a unique distribution in the body, which is why each instance of mitochondrial disorder is characterized by a spectrum of abnormalities.

In many cases, mitochondrial disease is a multi-system disorder affecting more than one type of cell, tissue or organ. Because muscle and nerve cells have especially high energy needs, muscular and neurological problems are common features of mitochondrial disorder.

Mitochondrial diseases cause prominent muscular problems are called mitochondrial myopathies, while mitochondrial diseases that cause both prominent muscular and neurological problems are called mitochondrial encephalomyopathies. In observational studies we found evidence that Bgx2 may positively affect mitochondrial disease.

Bgx 3: Noonan

A genetic disorder that impacts multiple body systems

Noonan Syndrome is a genetic disorder that impacts multiple body systems, including facial features, growth, and cardiovascular function. It is caused by mutations in genes involved in cellular signaling pathways. The incidence rate of Noonan Syndrome is estimated to be between 1 in 1,000 to 1 in 2,500 live births[5], making it one of the most prevalent genetic disorders with an autosomal dominant inheritance pattern.

Noonan Syndrome affects individuals of all ethnicities and genders, typically diagnosed in early childhood[6]. Clinical features can vary, but common manifestations include characteristic facial features, short stature, developmental delay, cardiac abnormalities, and bleeding disorders. Hearing loss, skeletal abnormalities, and lymphatic disorders are less common but may also occur.[7]

It is essential to understand the prevalence and impact of Noonan Syndrome to develop effective treatment options for patients. With an incidence rate of up to 1 in 1,000 live births, the need for innovative and targeted therapies for this patient population is critical. Biogntx is dedicated to advancing research in this area to address the unmet medical needs of Noonan Syndrome patients.

Bgx 4: Immune-mediated neuropsychiatric disorders

Also known as PANS, PANDAS and BGE

These disorders combine neurological and/or immunological problems with psychiatric and/or behavioral symptoms and are also known as PANS, PANDAS and BGE.

PANS (Pediatric Acute-Onset Neuropsychiatric Syndrome) is viewed as an[8] autoimmune/autoinflammatory condition that can be triggered by infections, metabolic disturbances, and other inflammatory reactions. Infectious triggers include upper respiratory infections, influenza, sinus infections, mycoplasma pneumonia, tick infections (such as Lyme borreliosis), and other sources. It is not always possible to identify the trigger.

At this time, PANS is a clinical diagnosis based on a child’s medical history, a physical examination and by ruling out other possible diagnosis that might better explain the constellation of symptoms. PANS is characterized by the abrupt onset of OCD (obsessive compulsive disorder) and/or eating restrictions, with concurrent symptoms in at least 2 of 7 neuropsychiatric categories:

• Anxiety
• Emotional Lability and/or Depression
• Irritability, Aggression, and/or Severe Oppositional Behaviors
• Behavioral (Developmental) Regression
• Sudden Deterioration in School Performance
• Motor or Sensory Abnormalities
• Somatic Signs and Symptoms, including Sleep Disturbances, Enuresis, or Urinary Frequency

PANDAS (Pediatric Autoimmune Neuropsychiatric Syndrome Associated with Streptococcus) is a subset of PANS. Symptoms follow an infection with group A beta-hemolytic streptococcus (GABHS), which can occur in many parts of the body including the throat, skin, intestines and perianal area.

In PANDAS, misdirected antibodies set off an inflammatory response that interferes with basal ganglia functions, producing behavioral, psychiatric, and neurologic symptoms including OCD, motor or vocal tics (or both) or both OCD and tics.

BGE: The basal ganglia are in an area of the brain associated with regulating functions such as motor movement, cognitive and emotional response, and procedural learning.  Post-infectious basal ganglia encephalitis (BGE) disorders are a subset of Autoimmune Encephalitis (AE) that occur when anti-basal ganglia autoantibodies (ABGA) are triggered by either bacterial, viral or fungal infections.

At Biogntx, we are committed to better understanding this type of disorder to develop innovative treatments. Our team of experts is dedicated to conducting research to uncover new insights into this condition and provide hope to those affected.

In addition, we are working to develop new treatments that offer a potential solution to this condition, bringing hope to families and children affected by these disorders.