About AlphaRose Therapeutics

AlphaRose makes genetic medicines faster and more affordably, giving potential accessible to millions of patients that are unaddressed by pharma. Using their all in one platform, AlphaRose plans to make hundreds of treatments for less time and money than pharma spends to make one. The company is co-founded by executives from the most successful rare disease company to date, Genzyme Corporation, and collectively the team has commercialized over 20 rare disease products globally. The companies' lead product, "Rosiphersen," is slated for clinical trials in early 2026.

For the first time in history, families no longer have to rely on the pharmaceutical industry to decide whether their loved ones receive treatment.

Tagged with

BiotechnologyGenetics & GenomicsArtificial Intelligence

Team

Casey McPherson

Founder

Problem statement

400 million people suffer from 10,000+ rare genetic diseases, and most are ultra rare. Currently, we have the technology to treat thousand of these diseases, but we don't have the business models to do it sustainably. Personalized genetic medicine is coming, but the business model, AI, robotics, and scalable systems must be built to open up this multi trillion dollar market.

AlphaRose is building this system.

Traction information

- Alpha Rose has raised $2.3m
- AlphaRose in-licensed Rosiphersen which is slated for clinical trials in 2026, with an expected peak annual revenue of $700m.
- The AI platform they are developing has already identified 300+ diseases that are potentially amenable to their technology platform.
- AlphaRose has acquired a new ASO technology company, "Alpha Anomeric", giving it the ability to out license, and co-develop treatments with big pharma.
- AlphaRose filed a patent for a new ASO design, targeting epigenetic expression.

Product video

Milestones

June 2025

Non Glp Tox Study for Rosiphersen Complete

Our animal tox study we just completed showed no adverse events. This study is a smaller version of the IND enabling study we will be performing next. This gives AlphaRose confidence that Rosiphersen will be safe in human trials.

April 2025

Acquired Alpha Anomeric (proprietary ASO Chemistry)

AUSTIN, Texas, April 29, 2025 /PRNewswire/ – AlphaRose Therapeutics, an advanced preclinical-stage biotechnology company focused on developing and commercializing therapeutics for neurogenetic developmental diseases, today announces the acquisition of Alpha Anomeric SA, a French company with a novel proprietary oligonucleotide chemistry platform (abcDNA). AlphaRose is currently advancing its lead antisense oligonucleotide product, Rosiphersen, through IND-enabling studies with a goal of initiating clinical studies in early 2026. AlphaRose is also developing a novel gene control technology platform and the Alpha Anomeric chemistry platform has the potential to become an integral part of the AlphaRose platform and development programs. Terms of the transaction were not disclosed.

Alpha Anomeric is a French company founded to develop a novel oligonucleotide chemistry platform (“abcDNA”) invented at the University of Bern (CH) by Professor Christian Leumann and Doctor Damien Evoquez. Alpha Anomeric was founded by Doctor Wolfgang Renner together with lead investor Advent Life Sciences and has validated the potential for the technology to be applied broadly in oligonucleotide therapeutic constructs. The novel chemistry platform is designed to enhance the safety, efficacy and applicability of oligonucleotide therapeutics in a variety of disease settings.

February 2025

Established all Partners for Clinical Trial of Rosiphersen

All contracts, quotes, and plans assembled for manufacturing, fill and finish, regulatory and clinical partners for lead product, “Rosiphersen.”

January 2025

Started Reg Cf Round

We completed due diligence and began launch of our Reg CF Round for 5m. www.startengine.com/alpharose

August 2024

Completed Stage 1 of Argus Ai

We completed our genetic graph database and initial algorithms to identify amenable genetic mutations.

July 2024

In-Licensed Rosiphersen

We succesfuly in licensed our lead product, “Rosiphersen” into AlphaRose Therpauetics from our partner “The To Cure A Rose Foundation.”

March 2024

Capitalized Company

Our Company launch with initial pre-seed capital from the Termeer Family Office.

Updates

A few updates from the AlphaRose team:

Science: We accomplished a major milestone, as our safety study for Rosiphersen come back with no adverse events, which gives us great confidence for success in our first in-human study. Our next steps will be looking at biodistribution from that study, GMP manufacturing, and setting up our IND enabling studies.

Fundraising: We have extended our Seed Round till August 28th. We're excited about this opportunity for investors to come in at the ground floor on this round, the momentum we're building, and the incredible team of investors that are coming together to support our vision.

The People Magazine full page story has now just hit the shelves today. Pick up a copy the next time you get a haircut! :)

Onward!

Casey McPherson
Ceo/Founder
512.940.4164

PS: We are doing an "Intro to AlphaRose Live" for interested investors June 25th 11:00CST - click here if you'd like to join! https://app.livestorm.co/p/eeacbc44-8a04-492c-bd74-1f107c032627

Intro to AlphaRose Therapeutics LIVE | AlphaRose Therapeutics

If you are considering a potential investment in children's health or biotechnology, we would love for you join. Intro to AlphaRose is a live session to provide you with a deeper understanding of A...

Attachments:

Added by Casey McPherson, about 8 hours ago

Emily Hyde has visited this profile using a private link.

Added 10 days ago

People Magazine just released a story on our founder, Casey McPherson. It will be in the next print edition.

https://people.com/dad-gives-up-career-find-cure-daughter-rare-disorder-exclusive-11725305

Dad Gives Up Career to Find Cure for Daughter Whose Rare Disorder Took Away Her Ability to Speak (Exclusive)

Musician Casey McPherson gave up his career to find a cure for his 9-year-old daughter Rose, who has an ultra-rare genetic disorder. Now, he says that his company is hopefully six months away from being able to give Rose her first treatment.

Added by Casey McPherson, about 1 month ago

Funding

Currently raising capital

$1,300,000

committed

$5,000,000

round goal

Total raised to date:$2,300,000

Pitch deck

Download current pitch deck