Some people with the genetic mutation for Huntington’s disease never develop symptoms, and Alchemab Therapeutics has found that their immune systems may be producing protective antibodies that keep the disease at bay. Huntington’s disease is a fatal neurodegenerative condition caused by a single inherited gene. There are currently no treatments that can slow or stop its progression. Alchemab analysed B cells from individuals who remained healthy despite carrying the mutation, and identified antibodies present in these resilient people but absent in those who became sick. The company now believes these naturally occurring antibodies could be turned into a therapy for patients who did not develop their own protective immune response. This grant funds the pre-clinical work needed to take a lead antibody through safety, efficacy, and manufacturability testing. If successful, the antibody could become the first treatment to slow Huntington’s disease progression, offering a fundamentally different approach from traditional drug discovery. The company aims to move faster than conventional timelines by starting with a molecule that nature has already validated in resilient individuals.
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Alchemab Therapeutics has a new way of approaching drug discovery by focussing on what keeps people well, sometimes against the odds. All of us produce a vast repertoire of different antibodies as part of our usual immune responses to fend off pathogens and to help the body maintain its health status. We believe that some individuals are able to use this mechanism to protect themselves against diseases to which they are pre-disposed. Huntington's disease (HD) is a devastating neurodegenerative disease, without any current medicines to slow or stop its progress. It has a genetic cause that is screened for in members of HD high-risk families. Alchemab analysed B cells (which produce antibodies) of patients resilient to various neurodegenerative disorders and identified antibodies common to those individuals resisting disease and not present in those patients who are showing symptoms. We have identified antibodies which we believe may be of particular utility in slowing progression in Huntington's Disease. This grant will accelerate the pre-clinical development of our lead antibody to demonstrate the safety, efficacy and manufacturability profile required by regulators for first in human clinical trials, to prepare for later stages of development and to attract the funding required to take us through an innovative clinical program to the market. Our aim is to develop and test these antibodies as therapies to help individuals living with a HD diagnosis that may not have been so lucky as to develop a protective antibody response themselves, which could be transformative in slowing the course of the disease. We hope this will help us to bring much needed therapies to Huntington's Disease patients faster than traditional drug discovery.
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