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Lab Notes: Amicus founder gets national honor; Capstan and Code Bio land on list of companies to watch


John Crowley
Amicus Executive Chairman John Crowley
Amicus

This week's life sciences news includes items about a national honor bestowed on a Philadelphia biotechnology company's chairman, two local drug developers making a national "companies to watch" list, another firm's progress on a potential cancer therapy, and more.

Here's the roundup:

Amicus Therapeutics

John F. Crowley, executive chairman and founding CEO of the Philadelphia-based biotechnology company, was named a recipient of a 2023 Horatio Alger Award.

The nonprofit Horatio Alger Association of Distinguished Americans has named Crowley and 12 other individuals, all of whom have overcome adversity to achieve professional and personal success, to its member class of 2023.

Crowley is a North Jersey native who at age 7 lost his father, a police officer, when he died in a tragic accident while on duty. He and his mother and brother then lived with his maternal grandparents in a two-bedroom, one-bath home that his grandfather had built after World War II.

A dedicated student, Crowley went on to get his undergraduate degree from Georgetown University, a law degree from the University of Notre Dame and an MBA from Harvard Business School.

In 1998, two of Mr. Crowley’s children were diagnosed with Pompe disease, a rare and often fatal neuromuscular disorder. Rather than accept the diagnosis as terminal, Mr. Crowley left his job at the age of 31, mortgaged his home, and dedicated his life to saving his children. He was able to raise $27 million in venture capital and ultimately co-founded a biotechnology company that saved his children’s lives, and the lives of thousands of others. After selling that company to a larger biotech firm, he started Amicus Therapeutics (NASDAQ: FOLD) to continue developing treatments for rare diseases. Amicus now employs over 500 people in more than 24 countries.

Crowley’s children living with Pompe disease, Megan and Patrick, are now 26 and 24 years old. Megan is a 2019 graduate of the University of Notre Dame.

“The commitment Mr. Crowley has shown not only toward his family, but to all families receiving devastating medical diagnoses, is extraordinary,” said Terrence J. Giroux, executive director of the Horatio Alger Association. “His ability to persevere and find purpose through multiple adversities demonstrates what it means to be a Horatio Alger Member."

Capstan Therapeutics and Code Biotherapeutics

The two Philadelphia-area life sciences firms made BioSpace's annual list of companies to watch.

The life sciences industry information source developed its Class of 2023 list by evaluating North American companies launched in 2021 and 2022 in areas consisting of innovation, finance, partnerships, pipelines, and growth potential.

The 25 top-scoring companies included Capstan, a University of Pennsylvania spinout with operations in Philadelphia and San Diego, and Code Bio of Hatfield.

Capstan, which ranks sixth, raised $165 million in a Series A round last year from investors that included Novartis, Pfizer, Bayer, Eli Lilly and BMS.  

Here's what BioSpace had to say about the company:

Capstan’s targeted delivery platform aims to reprogram immune cells in vivo with the payloads necessary to benefit patients with cancer, fibrosis and inflammation-related diseases and blood monogenic disorders. The platform has already demonstrated potential in preclinical studies against a fibrosis-related target.

Code Bio, which ranks 15th, raised $75 million in a Series A round led by Northpond Ventures of Bethesda, Maryland.

Brian McVeigh Headshot large
Code Biotherapeutics co-founder and CEO Brian McVeigh
Code Biotherapeutics

Here's what BioSpace had to say about the company:

Code Bio is aiming its non-viral delivery platform at therapies for some of the most prevalent genetic diseases including Duchenne Muscular Dystrophy and type 1 diabetes.  Code Bio’s 3DNA platform is made up of modular, programmable building blocks, enabling it to target cells and tissues with a high degree of specificity. The company attests the platform can enable targeted delivery of gene therapy, RNAi and other genetic medicine modalities.

In February, Code Bio and Takeda entered into a collaboration agreement to leverage Code Bio's 3DNA platform for a liver-directed rare disease program.

BriaCell Therapeutics Corp.

The clinical-stage immunotherapy company. which has operations in Philadelphia and Vancouver, said it received positive feedback from its end of Phase 2 meeting with the Food and Drug Administration regarding the company's lead clinical candidate, Bria-IMT.

Bria-IMT is being developed in combination with a checkpoint inhibitor to treat metastatic breast cancer.

Bill Williams BriaCell
BriaCell President and CEO Dr. Bill Williams
David DeBalko

BriaCell (NASDAQ: BCTX) and the FDA have agreed on the primary end point, the essential elements of the study design, and the type of patients to be enrolled in the company’s upcoming pivotal Phase 3 clinical study for Bria-IMT.

Dr. William V. Williams, BriaCell’s CEO, called the milestone "another major step towards our goal to become one of the leading immuno-oncology companies.”


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