Regenerative Myo-Bionics Therapeutic Pipeline

We are developing next generation regenerative medicine treatment strategies for neuromuscular & neurological disorders

Therapeutic Pipeline






FSHD is caused by a loss of DNA on Chromosome 4 which leads to the Dux4 gene being switched on.

BioRegenex is working to develop a bio-engineered muscle transplant construct for FSHD that aims to deliver therapeutic myoblasts into skeletal muscle, address fibrotic muscle damage caused by transient exposure to the Dux4 protein and establish a myoregenative niche of genetically corrected healthy muscle cells.


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Duchenne muscular dystrophy (DMD) is caused by alterations of a protein called dystrophin that helps keep muscle cells intact.

Using its Myo-Regenerative synthetic construct to deliver genetically corrected muscle cells with functional dystrophin to patients with Duchenne Muscular Dystrophy BioRegenex aims to restore muscle function and normal muscle biology in DMD patients.



Cancer cachexia is a wasting syndrome characterized by weight loss, anorexia, asthenia and anemia. The pathogenicity of this syndrome is multi-factorial, due to a complex interaction of tumor and host factors.

Oncology associated cachexia is a devastating condition that affects many cancers but is especially prevalent in pancreatic cancer from which 93% of patients die within 5 years of diagnosis. Cachexia often makes patients too weak to undergo surgery or tolerate chemotherapy.

BioRegenex is working to restore muscle in patients with severe Cachexia in order to maximize effective delivery of anti cancer treatment. 





Facioscapulohumeral muscular dystrophy or FSHD is a highly complex, progressive muscle wasting disease.


FSHD affects the lives of an estimated 1 million people but despite being considered one of the most common forms of muscular dystrophy in adults and children there are no treatments and no cure.

FSHD is commonly associated with progressive weakening of facial, shoulder and upper arm muscles. 

The regression often comes in bursts with sudden deterioration followed by periods of no change.

FSHD is arguably the most complex genetic condition currently known. FSHD involves a convoluted and complicated interplay between genes and proteins in the muscle cell. Unlike most genetic conditions where a mutation causes pathological changes in a particular gene and protein FSHD is caused by mutations that actually increase the expression of a toxic protein.

Nearly all cases of FSHD are associated with a mutation on chromosome 4. Chromosome 4 contains a series of repeated pieces of DNA, so called D4Z4 units. People without FSHD1 have 11 – 100 D4Z4 units. In people with FSHD 1 (95% of cases) the D4Z4 array is shortened to 1 – 10 units.

The D4Z4 units act like a lock for this region of the genome. With fewer repeats a gene embedded in this region called DUX4 is expressed. DUX4 is a transcriptional protein & when expressed in skeletal muscle leads to a cascade of events that eventuates in muscle cell death. 


The exact mechanisms are unknown & are still being studied, however it appears that DUX4 expression causes downstream muscle cell death. This loss of cells causes muscles to waste away. DUX4 may also damage the normal muscle regeneration pathways preventing muscles from repairing any damage experienced through normal daily activities.


DUX4 is also thought to invoke an immune response further inflicting damage on the muscles. Oxidative stress is also thought to play a role. As we learn more about the role of DUX4 the exact mechanisms for FSHD will become clearer.

Source: FSHD Global Research Foundation


Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.


DMD is one of four conditions known as dystrophinopathies. The other three diseases that belong to this group are Becker Muscular dystrophy (BMD, a mild form of DMD); an intermediate clinical presentation between DMD and BMD; and DMD-associated dilated cardiomyopathy (heart-disease) with little or no clinical skeletal, or voluntary, muscle disease.

DMD symptom onset is in early childhood, usually between ages 2 and 3. The disease primarily affects boys, but in rare cases it can affect girls.

In Europe and North America, the prevalence of DMD is approximately 6 per 100,000 individuals.


Muscle weakness is the principal symptom of DMD. It can begin as early as age 2 or 3, first affecting the proximal muscles (those close to the core of the body) and later affecting the distal limb muscles (those close to the extremities). Usually, the lower external muscles are affected before the upper external muscles. The affected child might have difficulty jumping, running, and walking. Other symptoms include enlargement of the calves, a waddling gait, and lumbar lordosis (an inward curve of the spine). Later on, the heart and respiratory muscles are affected as well. Progressive weakness and scoliosis result in impaired pulmonary function, which can eventually cause acute respiratory failure.

In 1986, MDA-supported researchers identified a particular gene on the X chromosome that, when flawed (mutated), leads to DMD. In 1987, the protein associated with this gene was identified and named dystrophin. Lack of the dystrophin protein in muscle cells causes them to be fragile and easily damaged.

Until relatively recently, boys with DMD usually did not survive much beyond their teen years. Thanks to advances in cardiac and respiratory care, life expectancy is increasing and many young adults with DMD attend college, have careers, get married, and have children. Survival into the early 30s is becoming more common than before.

MDA-supported researchers are actively pursuing several exciting strategies in DMD, such as gene therapy, exon skipping, stop codon read-through and gene repair.

Source: MDA


Oncology Associated Cachexia

Cancer-associated cachexia is a disorder characterized by loss of body weight with specific losses of skeletal muscle and adipose tissue. Cachexia is driven by a variable combination of reduced food intake and metabolic changes, including elevated energy expenditure, excess catabolism and inflammation.


Cachexia is highly associated with cancers of the pancreas, oesophagus, stomach, lung, liver and bowel; this group of malignancies is responsible for half of all cancer deaths worldwide.


Cachexia involves diverse mediators derived from the cancer cells and cells within the tumour microenvironment, including inflammatory and immune cells. In addition, endocrine, metabolic and central nervous system perturbations combine with these mediators to elicit catabolic changes in skeletal and cardiac muscle and adipose tissue.


At the tissue level, mechanisms include activation of inflammation, proteolysis, autophagy and lipolysis.


Cachexia associates with a multitude of morbidities encompassing functional, metabolic and immune disorders as well as aggravated toxicity and complications of cancer therapy.


Patients experience impaired quality of life, reduced physical, emotional and social well-being and increased use of healthcare resources.


To date, no effective medical intervention completely reverses cachexia and there are no approved drug therapies.


Adequate nutritional support remains a mainstay of cachexia therapy, whereas drugs that target overactivation of catabolic processes, cell injury and inflammation are currently under investigation.


Source:  Cancer-associated cachexia

Baracos VE1, Martin L2, Korc M3, Guttridge DC4, Fearon KCH5.