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Neurodegenerative Diseases

Avant-garde RNA Therapeutics for Neurodegenerative Diseases

Just as "Avant-garde" signifies the pursuit of groundbreaking values that go beyond conventional methods, BIORCHESTRA is challenging the traditional approach to treating neurodegenerative diseases and pushing the limits with innovative RNA treatment methods.

Neurodegenerative diseases are disorders that affect the central nervous system and gradually impair brain function and regulation. These conditions lead to damage and degeneration of brain tissue, as well as reduced neuronal function and abnormalities in neurotransmitters. Examples of neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, and Amyotrophic lateral sclerosis.

Alzheimer's Disease

Alzheimer’s disease is a progressive degenerative brain disorder characterized by gradual memory loss and cognitive decline. It is characterized by the presence of beta-amyloid plaques and neurofibrillary tangles in the brain, eventually leading to significant impairment in daily functioning.

Parkinson's Disease

Parkinson’s disease is a degenerative brain disorder caused by the progressive dysfunction of the gamma-aminobutyric acid (GABA) system, which controls motor functions. Key symptoms include progressive muscle rigidity, tremors, and bradykinesia (slowness of movement).

Amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a degenerative neurological disorder that affects the motor neurons, leading to progressive muscle weakness. It is characterized by symptoms such as muscle stiffness, muscle atrophy, motor impairment, and muscle weakness.

Huntington's Disease

Huntington’s disease is an inherited degenerative brain disorder that primarily affects the basal ganglia and cerebral cortex. It is characterized by a triad of symptoms, including dementia, motor abnormalities, and psychiatric disturbances. The disease worsens over time, leading to severe impairments.

Addressing the Challenges of Neurodegenerative Diseases with Innovative RNA Therapies

There remains a substantial unmet medical need in the treatment of neurodegenerative diseases.
BIORCHESTRA addresses several key areas of unmet medical needs and provides solutions in the following ways:

Disease modifying therapies

Currently, there are no therapies available that can provide a complete cure for most neurodegenerative diseases. Most treatments focus on symptom management and slowing disease progression. Therefore, the demand for a complete cure remains unmet.

At BIORCHESTRA, we are developing therapeutics for mechanisms that regulate RNAs involved in the production of pathological proteins in neurodegenerative diseases.

Minimization of Side Effects

At BIORCHESTRA, we use BDDS™ (brain-targeting drug delivery system) to deliver RNA drugs with high efficiency, enabling medicinal effects with fewer doses.  And RNA is a relatively safe molecule compared to DNA. It naturally occurs in host cells, and many RNA-based therapies focus on transient existence or ex vivo applications, minimizing potential side effects.

Passing the Blood-brain barrier

The Blood-brain barrier is a protective barrier between the brain and the bloodstream that prevents the entry of unnecessary substances and protects the brain. This barrier poses challenges for the development of brain disorder treatments.

At BIORCHESTRA, we developed BDDS™ platform technology to safely deliver a range of nucleic acid cargos, such as microRNA, siRNA, mRNA, and more, to all regions of the CNS. The proprietary platform fuses the core technologies of brain-targeted chemistry and RNA chemistry and is specifically formulated for IV administration.

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