AlphaRose Therapeutics

AlphaRose unlocks precision medicines for patients without a cure
Austin

About AlphaRose Therapeutics

AlphaRose makes genetic medicines affordable and accessible for 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.

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Problem statement

The current drug development model is based on one drug for many people, but genetic sequencing has shown us that most large diseases are actually many small diseases. We call these "rare diseases" once discovered. 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, and scalable systems must be built to open up this multi trillion dollar market.

Product video

Milestones

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.

Read more in the press release here: https://www.prnewswire.com/news-releases/alpharose-therapeutics-announces-acquisition-of-alpha-anomeric-sa-a-french-company-with-industry-leading-oligonucleotide-technology-302440678.html#:~:text=AlphaRose%20will%20maintain%20Alpha%20Anomeric,global%20basis%2C%20including%20throughout%20Europe.

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

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

Funding

Currently raising capital

$1,300,000
committed
$5,000,000
round goal
Total raised to date:$2,300,000
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