AlphaRose’s RareLabs emerges from stealth with AI-robotics platform targeting untreatable genetic diseases
RareLabs, a division of AlphaRose Therapeutics, has made its public debut with a modality-agnostic drug discovery platform that uses artificial intelligence and robotics to compress rare-disease treatment timelines from years to weeks. The Austin, Texas-based lab simultaneously announced the appointment of Dr Rob Freishtat as Chief Executive Officer.
Platform design and therapeutic modalities
Operating out of the ACC Bioscience Incubator in Austin, RareLabs has built an integrated system capable of designing, screening, and validating candidate therapies across four modalities under a single roof: antisense oligonucleotides (ASOs) for splicing and gene-silencing interventions; small interfering RNAs (siRNAs) for targeted mRNA knockdown; AAV-based and non-viral gene therapies; and repurposed-drug screens that mine approved compounds for rare-disease applications. The modality-agnostic architecture allows the platform to match each patient’s specific disease mechanism to the most appropriate therapeutic approach, rather than applying a fixed discovery methodology regardless of target biology.
Early-stage results and patient engagement
Since its founding, the laboratory has been directed by Dr Rodney Bowling Jr., whose prior record includes the discovery of more than 20 therapeutics and participation in dozens of clinical trials. Under his direction, RareLabs reports the identification of eight novel candidate treatments for conditions that previously had no therapeutic options, alongside partnerships with 30 disease communities and families spanning four continents. The programme has progressed candidates through the full discovery-to-validation arc, from target selection to in vitro efficacy demonstration.
A case study highlighted in the launch concerns a patient identified as Sasha, who carries an ultra-rare mutation in SLC6A1 – a gene associated with severe neurodevelop-mental and epileptic phenotypes. RareLabs designed a custom ASO therapy for this patient, which subsequently demonstrated efficacy in patient-derived cell lines, representing a critical preclinical milestone on the path to compassionate-use consideration.
“Sasha’s story is why we built RareLabs,” said Casey McPherson, CEO of AlphaRose Therapeutics. “When a person is facing an ultra-rare mutation, there is no pipeline waiting for them. We build the pipeline – fast, rigorous, and tailored to that single person. Cell-line efficacy is how you turn ‘impossible’ into ‘let’s keep going.’”
Leadership and strategic direction
Dr Freishtat, who retains his role as Chief Medical Officer of AlphaRose Therapeutics, brings more than two decades of experience at the intersection of paediatric medicine, genomics, and therapeutics development. “For patients and families facing a rare genetic diagnosis, every month of delay is potentially a month of lost function – often, lost irreversibly,” he said. “RareLabs exists to collapse the drug discovery timeline. Time is life for patients facing the impacts of rare conditions.”
International expansion
RareLabs leadership has recently concluded discussions in the Middle East aimed at establishing regional partnerships, with the stated aim of extending access to genetic medicine discovery for families historically underserved by Western rare-disease infrastructure.
The company is actively seeking collaborations with biopharma organisations, patient foundations, advocacy groups, and academic investigators.
For more information, visit: www.hopeatrarelabs.com
