Biotech Firm Neuralgene Begins Human Testing For New ALD Therapy

Neuralgene announced the enrollment of the first human patient in its new genetic therapy PRCN-323 testing for rare disease adrenoleukodystrophy (ALD).

Adrenoleukodystrophy is an inherited, rare, genetic disease linked to the X chromosome, in particular a mutation of the ABCD-1 gene. ALD causes build-up of very long-chain fatty acids in the brain and adrenal cortex and disrupts normal organ functions. The disease effects the male population more severely because males only have one X chromosome.

The disease is characterized by a number of symptoms, including adeficiency in adrenalin (known as Addison’s disease), mood changes, cognitive decline, and dementia. Adults diagnosed with ALD may experience decades of downward progression. But in children, onset of ALD typically evolves rapidly to a vegetative state and eventual death. A child diagnosed with ALD has a life expectancy of 1 to 10 years.

Founder and CEO Jason Williams, M.D., said that he believes his team is on the path towards discovering a cure for currently incurable maladies such as ALD. “We are very excited to closely monitor the results of this first patient. We plan to expand our trial to more patients right away. This phase of the study will be performed in Playa del Carmen, Mexico. In the meantime, we continue to study gene therapy options for other neurodegenerative disorders. We feel that we are close to having treatment options for several of these, including ALS and Parkinson's. At this point, the biggest hurdle is building the infrastructure to manufacture enough gene therapy for these patients.”

Current therapies for ALD address symptoms rather than the cause. The company’s approach to gene therapy is different in that it uses adeno-associated virus (AAV) to deliver corrective gene material. In PRCN-323, the viral payload is injected directly into the spinal fluid. AAV then preferentially targets neural tissue. Expression of the corrected genes is then expected to begin in a few weeks. Neuralgene said PRCN-323 is its fourth novel technique developed for neural-based gene therapy.