Permanent DNA Corrections. Women-Led Innovation. Why How Women Invest Backs MyoGene Bio
- Julie Castro Abrams
- Nov 10
- 3 min read
Written by Julie Castro Abrams
I've spent the last decade building alternative infrastructure for women founders, women investors, and the ecosystem that funds them. What I've learned is this: the most transformative breakthroughs often emerge from capital alignment when patient advocates, founders who understand suffering, and investors who believe in their work collide. MyoGene Bio is that collision point.
How Women Invest, alongside Parent Project Muscular Dystrophy (PPMD) and Duchenne UK, co-invested $1.42 million in MyoGene, a San Diego-based biotech company pioneering permanent DNA corrections for Duchenne muscular dystrophy.
The System That Fails Rare Disease
Duchenne muscular dystrophy is a fatal genetic muscle wasting disease that affects 1 in 5,000 boys. That's approximately 50,000 patients in the U.S. and Europe. For families navigating a disease that progresses from diagnosis at age 5 to loss of ambulation by the early teens to respiratory or cardiac crisis in the 20s, it’s devastating. Current treatments are management tools, not cures. Exon skipping therapies restore 1-5% of normal dystrophin levels and require lifelong repeated administration. Gene replacement therapies deliver micro versions of the protein via temporary transgenes with unknown long-term durability. The clinical data isn't there yet. Will that borrowed mini gene be fully functional and persist for decades? Nobody knows. This is where Dr. Courtney Young, CEO and co-founder of MyoGene Bio, had an insight that changed the equation.
The Elegant Science (And Why It Matters)
Young's observation was deceptively simple: patients with a much milder form of muscular dystrophy have a naturally occurring in-frame deletion of exons 45-55, produce a large dystrophin protein approximately 90% of normal, and can remain ambulatory into their 50s and 60s. That genetic variant isn't theoretical. It's happening in real patients. MyoGene's lead program, MyoDys 45-55, uses precision gene editing to recreate that protection in Duchenne patients. One permanent DNA correction instead of a drug for life. One edit instead of lifelong management. The target population: approximately 50% of Duchenne patients: 25,000 in the U.S. and Europe, 190,000 globally. For rare diseases, that's exceptional breadth. For venture capital, that should be a bright signal.
Capital Alignment As A Competitive Advantage
This is where venture philanthropy’s role becomes critical, and where I think we're witnessing the emergence of a new funding model. Duchenne patient foundations, like PPMD and Duchenne UK, are passionate about advancing research and supporting families with muscle diseases. For 30 years, PPMD has invested $55 million in Duchenne research that has secured eight FDA approvals. They understand the Duchenne disease biology. They have direct relationships with patients and families. They have the institutional credibility to attract philanthropic capital. How Women Invest brings something different: a network of healthcare entrepreneurs, clinical operators, manufacturing specialists, and LPs who understand the rare disease opportunity. We bring venture discipline and scaling infrastructure. We bring founder visibility and institutional connectivity. Together, patient advocates, women-led venture capital, and a woman founder who understands both the science and what's at stake, we accelerate progress faster than any single entity could alone. We would like to call this capital efficiency.
The Real Question
Over the next 18 months, MyoGene will advance preclinical studies, optimize gene-editing approaches, and generate data necessary for FDA clinical trial discussions. The science is grounded. The team is exceptional. The insight is elegant. If permanent gene editing for Duchenne works, and the biological rationale suggests it can, it will serve as evidence that systems thinking in venture capital yields better outcomes than winner-take-all competition.
Where This Leaves Me
I back companies and founders because I believe capital can be a tool for systemic change. Actual, measurable impact alongside returns. MyoGene represents that belief. Permanent cures don't happen by accident. They happen because patient advocates, women founders, and impact capital align around what's actually possible.
Julie Castro Abrams is the Founder and Managing Partner of How Women Invest and CEO of How Women Lead. She has over two decades of experience championing women’s leadership, driving legislative change, and disrupting the venture landscape to create more equitable opportunities. A seasoned investor, advisor, and Forbes contributor, she’s dedicated to elevating women leaders and was instrumental in passing California’s SB 826, mandating gender diversity on corporate boards.
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