Co-Founder & CEO
Casey McPherson
Rose’s father. Built To Cure A Rose Foundation, assembled the team that created Rosiphersen in under a year, and drives the company’s vision, culture, and fundraising.
|| ABOUT
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THE CRISIS NO ONE TALKS ABOUT
More than 200 million children worldwide are living with rare genetic diseases.
The vast majority — 95% — have zero approved treatments. Not because the science doesn’t exist. Because the business model doesn’t work.
Traditional drug development was built for blockbusters: big populations, big margins, long timelines. If your disease affects fewer than 500 people, you don’t get a drug. You get a shrug.
That’s not a scientific failure. It’s a systems failure. And we’re here to fix it.
- Million children
- 200
- Untreated
- 95%
|| STARTED
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WHERE THIS STARTED
A father. A daughter. A broken system.
AlphaRose has a simple premise. When Casey McPherson's daughter, Rose, was diagnosed with HNRNPH2-related neurogenetic disease with no treatment and no roadmap — he was told there was nothing to be done.
He refused to accept that.
Casey assembled a team of world-class scientists, built a nonprofit (To Cure A Rose Foundation), and created the first proof-of-concept treatment for Rose's disease in under a year. Not in a decade. Not with hundreds of millions of dollars. In months, with a small team and relentless urgency.
That experience exposed a hard truth: the science to treat these children already exists. What doesn't exist are the systems, the business models, and the regulatory pathways to deliver it at scale.
AlphaRose Therapeutics was built to change that.
|| WHAT WE BUILT
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A platform that turns genetic diagnoses into treatments.
|| THAT SCALES
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THE MODALITY THAT SCALES
Antisense oligonucleotides are the most deployable genetic medicine on the planet.
Gene therapies cost $1.5M+ per dose. They require viral vectors, complex manufacturing, and often can only be administered once. ASOs are different.
ASOs are synthetically manufactured, rationally designed, and chronically administered. They’ve already been approved by the FDA for multiple neurological diseases. Manufacturing costs are a fraction of gene therapy. And because they can be given repeatedly, they generate sustainable revenue per patient — not a one-time payment.
For ultra-rare diseases affecting 50 to 500 patients, ASOs are the only genetic modality where the economics actually work. That’s not an opinion. That’s math.
AlphaRose isn’t betting on ASOs because they’re trendy. We’re betting on them because they’re the only technology that can treat an individual child’s disease and still build a viable business around it.
|| PROGRAMS
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|| MARKET
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This isn’t charity. It’s a new category of biotech.
Ultra-rare diseases aren’t a niche — they’re an untapped market. With orphan drug designation, 7 years of U.S. market exclusivity, 10 years in the EU, and the FDA’s Pediatric Review Voucher (worth $75–100M alone), the regulatory incentives are enormous.
Our lead program, Rosiphersen, is projected at $300–600K per patient per year, with each patient generating an estimated $3.5M+ in lifetime revenue. Peak annual product revenue for Rosiphersen alone: $100–700M.
But the real value isn’t one drug. It’s the platform. Every treatment we develop compresses our timelines, enriches our datasets, and proves out a repeatable model. We’re building toward a future where a new ultra-rare medicine goes from genetic diagnosis to IND in six months — and where the platform itself becomes licensable.
AlphaRose is a Public Benefit Corporation. That’s not a compromise. It’s a competitive advantage. It aligns every stakeholder — patients, investors, partners — around the same outcome: treatments that work, delivered fast, at scale.
|| THE PEOPLE
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THE PEOPLE BEHIND THIS
Built by operators, not observers.
COO & President
Masako Nakamura
Oversees clinical development, commercialization, and pipeline operations. Leads execution of Rosiphersen’s path to market and manages grant funding strategy.
Chief Scientific Officer
Robert Cabrera, Ph.D.
Baylor College of Medicine. Leads all preclinical work across indications and oversees new technology platform development, including biomarker and toxicity programs.
Head of R&D
Jeffrey Brown, Ph.D., MBA
20+ years in preclinical drug development across Pfizer, BMS, Alexion, Voyager, and Wave Life Sciences. Has advanced multiple programs from concept to clinical candidate across RNA, AAV, and small molecule modalities.
Co-Founder & Executive Chairman
Alan Walts, Ph.D.
Former head of Genzyme Ventures (now Sanofi Ventures). MIT Ph.D. in Chemistry. Harvard Business School. Co-founded multiple life sciences companies. Advisor to the Termeer family office.
|| WHERE THIS GOES
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WHERE THIS GOES
We’re not building a drug company. We’re building the operating system for genetic medicine.
The endgame is a world where a child gets a genetic diagnosis and a personalized treatment in the same conversation. Where genetic medicine is a procedure, not a decade-long development program. Where cures are manufactured at the point of care.
Every piece of this technology exists today. No one has put it together. We are.
Phase 1
Rosiphersen — proving the platform works from target to clinic.
Phase 2
Commercialization and pipeline expansion.
Phase 3
RNAR-1: a fully automated robotics lab that takes drug development from design to GMP manufacturing.
Phase 4
Global scale — licensing our AI platform (RINAE.AI) and our process to hospitals, biotechs, and pharma partners worldwide.