Develops small molecule therapeutics that activate lysosomal calcium ion channels and enhance autophagy to improve lysosomal function, addressing the accumulation of toxic proteins and dysfunctional organelles in neurodegenerative diseases. This approach targets conditions such as amyotrophic lateral sclerosis, Alzheimer’s disease, and Parkinson’s disease, aiming to slow disease progression by restoring cellular balance and reducing neurotoxic protein production.
Funding
$29M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
BIECFounders
Product
Problem
Neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Parkinson’s disease, are characterized by the accumulation of toxic proteins and dysfunctional organelles within cells. This buildup disrupts cellular balance, leading to neuronal damage and cell death. Current treatments do not effectively address the underlying causes of this cellular dysfunction.
Solution
Libra Therapeutics is developing small molecule therapeutics designed to enhance lysosomal function and autophagy, thereby restoring cellular balance and slowing neurodegeneration. Their approach focuses on activating lysosomal calcium ion channels, such as TRPML1, to increase the breakdown and clearance of toxic proteins and dysfunctional organelles. Additionally, they are working on inhibiting the translation of nucleotide repeat expansions into toxic protein species, addressing diseases like ALS and frontotemporal dementia (FTD) caused by C9orf72 mutations. By modulating the C9orf72 function, Libra aims to re-establish healthy levels of autophagy and lysosomal function.
Target Audience
The primary target audience includes patients suffering from neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, Parkinson’s disease, and frontotemporal dementia (FTD), as well as the medical professionals treating these conditions.
Features
- Small molecule therapeutics that activate the lysosomal calcium ion channel TRPML1 to enhance autophagy and lysosomal biogenesis.
- Screening platform to identify compounds that influence autophagy and lysosomal biogenesis in vitro and in vivo.
- Programs targeting the inhibition of RAN-translation of C9orf72 repeat expansions to reduce toxic dipeptide-repeat-containing proteins (DPRs).
- Development of molecules that modulate C9orf72 function to restore healthy levels of autophagy and lysosomal function.
- Focus on restoring the balance between protein accumulation and cellular clearance to maintain healthy neurons.