Undergrads awarded $50k funding to develop FSHD therapy

The Undergraduate Entrepreneurship Program (UEP) at Sarafan ChEM-H was established to help undergraduates from biology, chemistry, and medicine backgrounds understand how a scientific idea turns into a new medicine, and gives them the rare opportunity to pitch an idea for a new company to industry experts—all before graduation.

Now nine years since its launch, the program has funded winning teams to research novel treatments for diseases including various cancers, antibiotics for drug-resistant bacteria, HPV, and degenerative retinal disease, among others. This year, the winning team is working to develop a new therapy for facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disease that affects nearly one million people worldwide and leads to progressive muscle weakening, particularly in the face, shoulders, and upper arms.

Students craft business plans with expert guidance

To guide undergrads through the process of identifying an unmet medical need, developing a new scientific approach, and creating a business plan, the program provides mentorship from leading biotech professionals. They are coached early in their careers on target identification, intellectual property, capital markets, company formation, and the art of the pitch.

In addition to building a business plan and practicing their pitches for the annual pitch night at the end of each fall quarter, students are exposed to a network of peers, industry and academic experts, biotech entrepreneurs, and venture capitalists.

“Undergraduates at Stanford have a legacy of starting companies, but launching a biotech company involves a longer timeline, greater complexity, regulatory hurdles, and higher financial risks. Most scientists aren’t exposed to this level of entrepreneurship until after their graduate work or PhD,” said Elizabeth Ponder, PhD, Executive Director of Sarafan ChEM-H. “The UEP gives undergrads—especially those in fields like chemistry and biology—the opportunity to explore an entrepreneurial track early in their careers, which is exciting."

The winning team – Myoterra Biosciences

On December 5, 2024, five teams of Stanford undergraduates competed for a $50,000 grant from Sarafan ChEM-H. “We had five excellent teams deliver high-quality presentations addressing an unmet biomedical need,” said Mark Smith, PhD, head of the UEP and Director of Medicinal Chemistry at Sarafan ChEM-H.

Constructing a good pitch is difficult. According to Smith, having a unique idea and a good research plan are critical, but so is including a strong narrative through the story. “Each team only had 10 minutes to present and they had to convey that message very clearly. It also had to be translatable within a limited amount of funding,” said Smith. “It’s probably more challenging than what most entrepreneurs face when they pitch to traditional venture firms!”

The winning team, Myoterra Biosciences, comprises Heloise Hoffmann ‘26, Alice Finkelstein ‘27, and Michael Liu ‘27.

During the annual pitch night, Myoterra’s vision to develop a treatment for FSHD caught the attention of the judges. FSHD is caused by a mutation in the gene that contains the instructions for making a protein known as DUX4. In healthy individuals, DUX4 is only produced during embryonic development, then it is silenced. In FSHD, however, muscle tissues continue to produce DUX4, which then binds to and activates toxic genes, leading to progressive muscle weakening. 

Currently, there is no cure for FSHD. Patients typically manage the condition through pain medication, physical therapy, occupational therapy, or in certain cases, surgical intervention. For patients with FSHD, everyday activities like raising their arms, walking, chewing and swallowing, and closing their eyelids can become insurmountable tasks.

Myoterra’s new strategy involves engineering a protein that will bind to the same genes as DUX4 but repress rather than activate those target genes, without interrupting other normal cellular processes.

For the team, this grant is just the beginning. “We have initial proof of efficacy in a cell model and now want to test it on more clinically relevant models. We’re excited to have the funding to move forward with these efforts, giving us much more potential to accomplish what we outlined in the pitch,” said Finkelstein.

“I’m excited to spend this two-meter-tall check!” said Liu, who emphasized enjoying the overall experience of the program. “It’s been amazing from day one, from idea generation to now, and we finally get to do what we pitched,” he said.

“People living with FSHD have been tirelessly advocating for a treatment for a long time. Now, we are one step closer to making that hope a reality for nearly one million patients worldwide,” said Hoffman, who has a close personal connection to FSHD. “This community is dearly close to my heart, and I am honored to get to give back in such a tangible way.”

Learn more about the Undergraduate Entrepreneurship Program (UEP) and previously funded projects.