AlphaRose Therapeutics

AlphaRose unlocks precision medicines for patients without a cure
Austin

About AlphaRose Therapeutics

AlphaRose makes genetic medicines faster and more affordably, giving potential access to millions of suffering patients unaddressable by pharma. Using their vertically integrated system, AlphaRose plans to make hundreds of treatments for less time and money than pharma spends to make one. Think Star Trek of genetic medicines. 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

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 $2.6m
- AlphaRose in-licensed Rosiphersen which is slated for clinical trials in 2026, with an expected peak annual revenue of $700m.
- They have 4 additional genetic treatments in the pipeline, with $XB in AR potential.
- 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 platform called "Sot Caller," targeting epigenetic expression. Also with the ability to out license, develop, and co-develop treatments with big pharma.
- New FDA regulations as of Sept 2025 have created a favorable pathway for the diseases that AlphaRose is targeting
- AlphaRose is in multiple discussions with potential partners, and pursuing non-dilutive funding opportunities.

Product video

Milestones

October 2025

Opened Convertible Note for 2M (Oct 1st - Nov 30th)

AlphaRose opened their second convertible note with favorable terms, with internal investors priority, from Oct 1st - Nov 30th 2025.

September 2025

ART - 002 / 003

Designed and intiated studies on two new genetic treatments with our proprietary technology, “SOT Caller” These treatments are estimated to each reach 300-700m in AR once approved.

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.

June 2025

Sot Caller (epigenetic platform)

We filed a provisional patent for our new epigenetic platform, “Sot Caller.” This technology has the potential of changing gene expression throughout the human body. In addition to new therapies we create with it, similliar technologies have produced licensing deals in the xB’s. Our plan is to develop this modality with new treatments, and once reach sufficient data, we will be poised to execute partnerships with biotech and pharma.

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

AlphaRose has built significant value over this last quarter, with 5B+ in potential revenue waiting to be unlocked. Our team is very excited about the incredible opportunities in front of us.

To that end, we are announcing a $2m Convertible Note that just opened, Oct 1st - Nov 30th for select investors. The note is filling fast. Please reach out to me immediately if you have interest. ([email protected] / 512.940.4164)

Some significant markers:
- We are currently now advancing 5 genetic treatments in the pipeline. ($XB in potential annual revenue)
- We are continuing to develop our two proprietary RNA technologies (abcDNA and SOT Caller) poised for large licensing deals as early as 2026. (see Merck and Skyhawk as an example)
- We are in multiple discussions with potential partners in biotech, venture, and healthcare systems.
- We closed our Reg CF Round Sept 30th with $1.6M in additional investment.
- We have two non-dilutive grants we're working on totaling over $50M in potential capital.
- We have board approval to acquire RareLabs, a private genetic treatment lab in Austin, Tx, bringing in $1M in AR.

AlphaRose is poised to be a revenue generating company in 2026 and beyond, and we are excited about the impact we will be making for patients with genetic diseases for generations to come!

For more info on the note and a company update Click here: https://alpharose.info/introdeckcln2

Sincerely,

Casey McPherson and the AlphaRose Team

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Check out the latest article in NewsWeek featuring our founder's story. We have the technology to treat and even cure many of these diseases. The industry lacks the business models and systems to do it at scale and speed. AlphaRose aims to fix this problem. Patients are waiting!

https://www.newsweek.com/research-moves-slowly-rare-diseases-dontso-patients-arent-waiting-2090277

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

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Funding

Currently raising capital

$1,000,000
committed
$2,000,000
round goal
Total raised to date:$2,600,000

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