Written by Julie Penfold, RARE Revolution insider
Interview by Nicola Miller, RARE Revolution insider, featuring Casey McPherson, CEO, AlphaRose Therapeutics

Estimated reading time: 7 minutes

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Casey McPherson is an accomplished singer and songwriter who has toured the world and celebrated US Billboard Top 10 hits. Yet, he turned his back on a record deal with Sony and today has largely left his passion for music behind—all to pursue an inner drive to find a treatment for his beloved younger daughter, Rose.

“Rose was born different and as a baby—she would choke on her food, and fall over on her face,” Casey explains. “But when she lost her ability to talk, that’s when we knew something was really very wrong.

“As a father, you want to protect your children. Yet, there was no monster for me to slay. There was no car I could throw myself in front of either. Instead, it was this single nucleotide change and a random gene called HNRNPH2.”

After having a whole exome sequencing test, a paediatric neurologist confirmed Rose had HNRNPH2-related neurodevelopmental disorder (HNRNPH2-NDD). It’s a rare genetic condition that is characterised by developmental delay and motor and language problems.

Upon hearing his daughter’s diagnosis and being advised there was nothing that could be done with currently no cure available, everything changed for Casey.

“For the first time in my life, I wanted to do something different than music. I wanted to create a cure for my daughter,” he explains. “I started by searching for other parents who had children with a rare disease, and I was amazed. Some had very successful companies and some had also created treatments—such as Julia Vitarello [founder and CEO of Mila’s Miracle Foundation].”

Creating the To Cure a Rose Foundation

“From that point, I started theTo Cure a Rose Foundationwith the aim of one day developing a cure for my daughter and also for the next parent who has a child with a rare disease where there is no treatment.”

But Casey wanted to do this differently and help more parents to participate in developing rare disease treatments. “I wanted the foundation to be something others could participate in—so they wouldn’t need to start their own foundation or become a scientist, or source their own scientific advisory board, or have to raise millions of dollars.”

As the foundation began work on creating a treatment for Rose, Casey and his expert team of scientists and rare disease pioneers realised that a laboratory for drug development was needed. The foundation launched their own laboratory, now calledTCAR Rare Labs, and it is something he is very proud of.

“We employ highly skilled scientists at our lab that have developed many drugs before, so they know what productising an idea looks like,” Casey explains. “We began to work with oligonucleotides, gene therapies and repurposed drugs, and parents can use our lab and own all their intellectual property. They pay for using the laboratory but we keep the costs to a minimum. We also try to raise donations to even supplement some of the costs for parents. But they own everything they do at the lab, so they stay in the driver’s seat.

“As a foundation, we love antisense oligonucleotides (ASOs) as there’s a path to treating a child safely and quickly with them as Julia Vitarello has shown when she developed Milasen for her daughter, Mila, who had a rare form of Batten’s Disease, as well as N-Lorem. Those treatments are making a huge impact on neurodevelopmental and neurodegenerative disease.”

Becoming AlphaRose Therapeutics

At the end of 2023, To Cure a Rose Foundation led the way to the formation of a public benefit biotech company calledAlphaRose Therapeutics. The idea was the company would be fiduciary responsible to its investors and its customers—patients with rare disease. Casey then launched full steam ahead into pitching to potential cofounders and investors.

“The goal of AlphaRose is, can we take Rosipherson, the drug we have developed for Rose, and help others,” he explains. “Could we license that from the Foundation, which we did, and could we pioneer and scale the clinical development and commercialisation of these types of treatments, starting with neurodevelopmental disease in children?” he explains.

After doing some number crunching and financial modelling, Casey and his team realised they could do this. “It’s actually less risk than larger drugs as it targets the underlying cause of the disease, and it takes less money to make,” he adds. “These are not billion-dollar drugs, but they are financially successful drugs that make a real difference to children with small population rare diseases.”

Their next step after Rosiphersen and other ASOs are successfully through the clinical trial will be to get their process approved by regulators. “At that point, a drug that might previously have cost $500,000 could come down to a fraction of that because of this “process approval.” Added to this, we could make drugs in days, not years. That’s the vision of AlphaRose and I think that’s where genetic medicine is heading.”

Casey says he’s been especially inspired by Henri Termeer who used his non-medical background as a powerful vehicle for change as the CEO of leading biotech company, Genzyme.

“Henri would bring patients into investor meetings and ask his commercial team how many patients had been treated that day. He would also go into countries who were unwilling to pay for his drug and treat patients who needed it, then negotiate with those governments. He would not put the burden of the high costs of rare disease treatments on patients. That really inspired me, and I want to take that inspiration to find my own new ways to do things differently too.”

As part of this bold mission, Casey started equity crowdfunding, rather than going down the venture capital-seeking route. This method of fundraising enables patients and rare disease advocates to buy shares in companies and was an important part of retaining the values of his foundation and early work.

“My thought was can we create a company where the majority of ownership is rare disease patients, families, scientists and people who care about making a change. That’s what I’m in the middle of right now.”

Next steps for Rosipherson (named after Rose)

At the time of chatting with Casey, AlphaRose had already raised over $1M via equity crowdfunding. The company’s chief scientist has also identified a number of other genes that AlphaRose could target.

“My goal with this seed round is to raise $5M and then we want to take Rosipherson into a clinical trial so we can move closer to treating the first children with our drug.

“It could be as early as the end of the year, or it could be as late as next year (depending on funding). That’s what gets me really excited because I continue to see the light at the end of the tunnel.”

Casey says he is in this journey solely to usher in new treatments so the next parent like him or the next child like Rose, that has a gene mutation that no-one has seen before, has a path forward.

“Companies don’t usually start with patients, they just pick some disease, and patients often have zero voice within endpoint development and that’s something I want to change,” he says. “I’m trying a different model where the patient community stays part of the development throughout the entire process. They are the ones that we’re trying to treat and that the clinical trial needs to be right for them.”

His experiences of working in the highly competitive music industry, where knock-backs are part of everyday life, are proving useful with fundraising and pitching to investors as part of his CEO role at AlphaRose.

“I’m used to fighting and what better muse to succeed for than your own daughter,” he adds.

Connect with Casey

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