Longeveron’s Unique Approach to Stem Cell Therapy Shows Promise in Alzheimer’s Disease
Under CEO Wael Hashad, this public biotech company – which joined StartUp Health's Alzheimer's Moonshot in 2025 – is applying their proprietary stem cell methods to Alzheimer’s disease, aging frailty, and hypoplastic left heart syndrome (HLHS). Their Regenerative Medicine Advanced Therapy Designation (RMAT) from the FDA puts them in a strong position for potential US regulatory approval.
Written by Logan Plaster
Stem cell research has had a dramatic storyline.
Act I began in the 1950s with the first bone marrow transplant, but got considerably more dramatic in the 1990s. Around this time, stem cell research became a topic of public debate because researchers were harvesting stem cells from human embryos. Enough people took moral issue with the destruction of these embryos that in 2001, then President George W. Bush banned government funding for stem cell research.
Act II has looked quite different. In their labs, and without making many headlines, scientists began discovering new, non-embryonic ways to harvest stem cells. They now can get them from bone marrow, blood, and the umbilical cord – all from willing donors. That development opened up a new, exciting phase of stem cell research, one that we’re still seeing come to maturity.
What will Act III bring? If you ask Wael Hashad, CEO of stem cell therapy company Longeveron, it’s going to be huge – a revolution in therapeutics.
“I really believe that the future of therapies is around cell and gene therapy,” says Hashad, who spent 30 years working at Eli Lilly, Boehringer Ingelheim, and Amgen before taking the helm at Longeveron. “I think 25 years from now more than half of the new medicines that will be approved will be cell and gene therapy.”
Origins
Longeveron traces its roots back to the work of researcher-physician Joshua Hare, MD. Dr. Hare studied cardiology at Harvard and then went to Johns Hopkins to dive into stem cell research, particularly around heart failure. What he discovered was much bigger than the heart and had apparent applications in aging frailty and Alzheimer’s disease.
Dr. Hare was invited to the University of Miami to establish an interdisciplinary stem cell institute. In 2015, he teamed up with an investor and spun off some of his technology into a private company called Longeveron, with an initial focus on aging frailty, which later expanded its focus to include cognitive impairment and congenital heart disease. The company went public in 2021, and in 2023 Wael Hashad joined the team as CEO.
Wael Hashad was born in Salt Lake City to Egyptian parents. He went to pharmacy school with a youthful desire to heal the world.
“My hope at that time was to discover new medicines and treat people who don't have treatments and things like that. When you are sixteen or seventeen that's all you hope for.”
After graduating, Hashad went to work for Eli Lilly, and spent the majority of his career there. Over time his focus narrowed into neuroscience, both in terms of psychiatric and neurological diseases. These conditions intrigued him because of how they hid in plain sight.
“I realized that a lot of these diseases have more devastating effects than any person walking up the street knows because they don't see what’s happening beneath the surface,” says Hashad. “And it’s not just the patients who bear this unseen burden, but their families and caregivers.” That burden, he adds, is physical, it’s emotional, and it’s financial.
Over thirty years, Hashad worked on a range of psychiatric and neurological conditions, from depression to schizophrenia to attention deficit and hyperactivity disorders. But when he shifted to Alzheimer's disease he gained a new clarity and passion, one that still guides his work today.
When Wael took on the role of CEO at Longeveron, he knew he was taking a calculated risk. Stem cell research is cutting edge and not an easy road. But the potential upside was worth the challenge.
“I ask myself what do you want to do with the things you know? Do you want to be in the forefront of innovation and discovery? I’m lucky in that I’ve had a good career and I’ve been blessed in life such that I can take this risk.”
Part of the excitement for the Longeveron team is that they’re working in clinical areas with little or no therapies available. These are patients in desperate need of hope and healing, so the upside of developing a successful cell therapy is almost incalculable.
Under the Hood
Without getting too technical, stem cells are a special class of cells that has pro-vascular and anti-inflammatory properties. Stem cells are found all over the body, but today are primarily harvested from bone marrow, the umbilical cord, and adipose tissue (found in body fat). There are pros and cons to each stem cell source.
While there are a wide array of potential use cases for stem cell therapy, the underlying principles remain basically the same, says Hashad.
As we age, the number of stem cells in our body decreases. At the same time, we generally see increased levels of inflammation throughout the body, which can lead to a wide range of health problems, from heart disease to arthritis. Research has shown a correlation between these two changes, and that stem cell therapy may actively reduce inflammation in very important ways.
Longeveron has built its research around bone marrow-derived stem cells and has developed a proprietary way to harvest and grow the cells. To oversimplify, they believe their method ensures the quality of the cells and the effectiveness of how they’re used. Then, of course, they differentiate themselves by doing the hard, time-consuming work of clinical validation.
Longeveron is one of only a limited number of companies moving into large population studies for stem cell therapy in Alzheimer’s so it will be exciting to see their results. The team recently had a positive meeting with the FDA to discuss their development pathway for regulatory filing and potential approval. This kind of research is time- and resource-intensive, so Longeveron will be seeking non-dilutive funding or partnering to pursue this exciting opportunity.
One competitive advantage in Longeveron’s corner is the fact that they’ve received a Regenerative Medicine Advanced Therapy Designation (RMAT) from the FDA.
“We believe we are the only drug that has received RMAT designation from the FDA for Alzheimer’s disease,” says Hashad. “It’s equivalent to a breakthrough designation for traditional drugs,” believes Hashad. “It doesn’t just give us bragging rights; the biggest value in my opinion is that it streamlines your pathway to the market.”
Longeveron is focused on three core areas of stem cell research: Alzheimer’s disease, aging frailty, and hypoplastic left heart syndrome (HLHS). HLHS offers Longeveron the fastest path to commercialization, so that is a priority for the company. Getting this first product to market could help finance their work in Alzheimer’s and aging frailty.
It takes patience and vision to be an entrepreneur in the world of stem cell therapy. Nothing happens quickly, and there’s plenty of room for failure. However, with research proving the ability for stem cell therapy to possibly reduce inflammation and improve vascular health, the future health implications are enormous, well beyond Longeveron’s current targets. In other words, this is audacious health moonshot work if we’ve ever seen it.
“There are a lot of other things that we know that stem cells do,” says Hashad, “and we're probably going to learn about them in the future.”
Join us in welcoming Wael Hashad and the Longeveron team to the StartUp Health community.
Connect with Longeveron via email
Call for Innovation
Want to Tell Your Story to the World?
We’re looking for founders and CEOs leading digital health, life science, or biotech companies that are solving the biggest health challenges of our time – and want to build a better network to change the world.
Exposure in StartUp Health Media to our global audience of investors and partners – including our magazine and YouTube channel – is a benefit of StartUp Health's Membership. When you join a Health Moonshot Community, you can increase brand awareness through our multi-media storytelling efforts.
Our communities include: Access to Care, Alzheimer’s Disease, Cardiometabolic Health, Children’s Health, End Cancer, Food as Medicine, Mental & Behavioral Health, Oral Health, Type 1 Diabetes, Wellness, Women's Health, and many more.
If you’re mission-driven, collaborative, and ready to contribute as much as you gain, you might be the perfect fit. Learn more and apply today.
Follow us on LinkedIn for daily updates on Health Transformers.
Published: Mar 27, 2025
