Rare Genetic Conditions

Bespoke genetic medicines

Personalised gene therapies for rare genetic diseases.

At Bespoke Genetic Medicines Inc., we believe no disease is too rare and no patient group is too few to deserve a cure. We are a community-based, not-for-profit organisation pioneering individualised gene therapies for ultra-rare genetic neurodegenerative conditions.

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Our Vision

To realise the promise of individualised gene therapy so that rare disease patients can live the good life.

The Global Scale of Rare DiseaseS

7,000+ unique rare diseases worldwide
300M+ people affected globally
95% have no approved treatment
30% of rare disease children do not survive past age 5

There are up to 1,700 distinct rare genetic neurological conditions with no available treatment. Individuals with ultra-rare diseases and their families are too often left behind because there is no profitable market opportunity for conditions affecting fewer than 100 patients globally.

Learn why a new model is needed

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300M+ people affected globally

7,000+ unique rare diseases

Our Approach

Traditional pharmaceutical companies develop one drug for millions of patients. We design therapies tailored to each patient's unique genetic error, one at a time.

Our modular platform strategy uses reliable foundational elements across multiple conditions. The only component we customise is the therapeutic payload, precision-engineered to correct an individual's specific genetic error.

Discover

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Develop

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Deliver

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Read about our approach

Joseph's Mission

CureSPG75

Born in 1996, Joseph spent 27 years navigating misdiagnosis, developmental challenges, and uncertainty before whole exome sequencing finally delivered his genetic diagnosis: SPG75. His story drives our first mission — developing a rescue gene replacement therapy to correct the root cause of this devastating disease.

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Read Joseph's full story

Our Leadership Team

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Dr Laura Issa

Chairperson, Scientific

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Michelle Sadek

Patient Community Engagement

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Dr Kimberley Lilischkis

Clinical Development, Regulatory

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Anjelica Issa

PR and Fundraising

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Joseph Issa

Patient Affairs

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Jenny Saliba

Treasurer and Secretary

Learn more about the team

Our research partners

Help us bring hope from the lab to the clinic

Your support is crucial to help bring specialised, life-changing treatments to the children and families who desperately need a chance to thrive.

Become A Partner

Donate

Connect With Us

If you or someone you know has SPG75, we want to hear from you.

Every family we connect with brings us one step closer to understanding this disease, and one step closer to a cure.

Whether you're a parent navigating a new diagnosis, a researcher working in this space, or simply someone who wants to help, there is a place for you in the CureSPG mission.

Follow our journey, share our story, and together lets turn awareness into action.