Former Musician Casey McPherson Tackles Rare Disease Cures Through Biotech
By Bio-IT World Staff
October 10, 2025 | Musician-turned-biotech founder Casey McPherson is rewriting the playbook for rare disease research and drug development. After a career in music, McPherson is now the found and CEO of AlphaRose Therapeutics, a biotechnology startup born from his daughter Rose’s diagnosis of a rare debilitating genetic mutation, HNRNPH2, which encodes a protein that belongs to the heterogeneous nuclear riboprotein family and plays a role in RNA processing and transport.
Over 10,000 rare diseases affect millions worldwide, yet fewer than 5% have treatments. McPherson refused to accept this and set out to find a cure for his daughter’s condition. But early on during his journey, he learned why it was so difficult to find treatments and cures for these rare diseases. “It wasn’t a technology issue,” he said in the latest episode of the Trends from the Trenches podcast. “It was a business model problem. It was a systems problem.”
That model, according to McPherson, lies in antisense oligonucleotide (ASO) therapies, programmable medicines that can target specific genetic mutations. With guidance from rare disease veterans at Ultragenyx and mentorship from biotech pioneers, he built AlphaRose Therapeutics to prove that personalized genetic treatments can be both scientifically and economically sustainable.
Unlike the traditional pharmaceutical model, which often takes years and loads of money to produce a single drug, Alpha Rose operates as a public benefit corporation, balancing investor interests with patient access. McPherson envisions a future where families no longer have the financial and educational burden to figure out how to help their children.
The company combines AI, automation, and ASO technology and software to accelerate preclinical and clinical workflows. Their proprietary ASO design platform integrates genetic databases like OMIM and ClinVar, using machine learning to predict toxicity and therapeutic potential across mutations. “ASOs are a sequence of nucleotides, and so are genes, right?” McPherson said, elaborating on using pattern recognition to make smarter, safer drugs in a faster way.
In addition to changing the game on rare disease treatments and clinical trials, McPherson also shares that it is critical to have and retain compassion and empathy for families with children afflicted with rare diseases. “Those are the people I consider true rock stars.”
To learn more about AlphaRose’s mission, how they’re getting investors involved, and more, listen to Trends from the Trenches.