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Ep. 22: Dr. Courtney Young of MyoGene Bio and Her Journey to Tackle DMD

  • davidcrean6
  • Dec 23, 2024
  • 2 min read

In our most recent episode, Bob and David invited Courtney Young, CEO and co-Founder of MyoGen Bio, into the podcast studio. MyoGene is a biotechnology company focused on developing cutting-edge genetic therapies for muscle diseases, with their lead program targeting Duchenne Muscular Dystrophy (DMD). The company was founded in 2018 and is headquartered in San Diego, California. Its team brings together over 50 years of cumulative experience in muscle disease research and treatment, led by Dr. Young, along with scientific advisors Dr. Melissa Spencer and Dr. April Pyle from UCLA.


"I got into the Duchenne field when my cousin was diagnosed with Duchenne muscular dystrophy when I was in high school." -Dr. Courtney Young

Key Aspects of MyoGene Bio


Lead Program: MyoDys45-55


MyoGene Bio’s primary focus is on MyoDys45-55, an AAV-delivered CRISPR/Cas9 gene editing therapy for DMD. This innovative approach aims to:

  • Permanently remove a mutational hotspot in the dystrophin gene

  • Restore a smaller but functional version of the dystrophin protein

  • Target up to 50% of all DMD patients with a single therapy


The therapy is designed to mimic a naturally occurring deletion associated with a milder form of muscular dystrophy, potentially transforming DMD into a less severe condition.


Funding and Support


MyoGene Bio has received significant backing from various sources:

  • $3.4 million grant from the California Institute for Regenerative Medicine (CIRM)

  • Investment from CureDuchenne Ventures

  • NIH and Department of Defense grants for preclinical development

  • Selected for accelerator programs like Creative Destruction Lab and MassChallenge


Recent Developments


MyoGene Bio has achieved several milestones and awards:

  • Granted Orphan Drug and Rare Pediatric Disease Designations by the FDA for MyoDys45-55 (see Press Release)

  • Awarded 4th place in the Early Stage competition at the Entrepreneurship World Cup in Riyadh, Saudi Arabia (see Press Release)

  • Advancing preclinical studies with additional grant funding (see Press Release)

  • Raising additional financing of $2 million in seed capital to advance its technology


Listen here to our journey with Dr. Young. Dive in and ride the innovation wave. Be informed, be inspired, be innovative!


Latest Episode of Innovation Wave Podcast: Dr. Courtney Young
Latest Episode of Innovation Wave Podcast: Dr. Courtney Young

For our listeners who want to learn more or get involved with MyoGen Bio, contact Dr. Young on LinkedIn or visit their homepage for more information.


About Innovation Wave Podcast


Innovation Wave is a life sciences and technology-dedicated podcast produced by co-hosts Bob McGriff and David H. Crean. Relevant topics within the ecosystem related to the growth and exit of businesses are discussed in candor with entrepreneurs, key executives, influencers, and thought leaders in 15-20 minute episodes.



Disclosure


This podcast and article are provided for informational purposes only and do not constitute an offer, invitation, or recommendation to buy, sell, subscribe for or issue any securities. David H. Crean, a principal at Cardiff Advisory LLC, is a registered representative of BA Securities, LLC Member FINRA SIPC, located at Four Tower Bridge, 200 Barr Harbor Drive, Suite 400 W. Conshohocken, PA 19428. Cardiff Advisory LLC and BA Securities, LLC are unaffiliated entities. All investment banking services and securities are offered through BA Securities, LLC, Member FINRA SIPC.

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