We believe a healthy life is a basic human right.

Many beatable diseases are left behind because they are too small. At the Institute for Life Changing Medicines, we fight to beat these diseases.

 

OUR GOAL

Change the lives of 1 million people suffering from life threatening and devastating diseases around the world with no barriers to access.

 

OUR MISSION

A world free from devastating genetic diseases.

Improve the lives of those impacted by genetic diseases through detection, prevention, treatment and advocacy.

 

Our Strategy

We are determined to see the world differently for people with diseases left behind.  We act by bringing them medicines, education and advocacy.

Our Advantage

Three unique forms of advantage come together to enable us to complement the work of our for-profit colleagues completing the continuum of care for all.

Our Medicines

Diseases that affect very few people are often not investable and left behind. We are on a crusade against these diseases. 

OUR STRATEGY

No disease left behind.

Experts within the Institute for Life Changing Medicines target lethal genetic diseases not currently investable because they are not profitable enough. 

 

Detection & Prevention

We teach aspiring parents about preconception genetic carrier testing to lessen the risk of facing a newborn tragedy.

Therapeutic Development

Bringing life changing medicines to patients within two years and for between $5 million and $30 million.  We will focus on diseases for which a transformative effect is likely and enabled by donations at multiple levels, including: capital, intellectual property, manufacturing, supply chain and more.  We will distribute our medicines with no barriers to access for anyone.

Advocacy

We will join forces with for-profit businesses to collectively take action against an even broader range of diseases. We will advocate for a world where medicines are accelerated forward and access barriers are eliminated.

 

OUR ADVANTAGE

Genetic disease is beatable.

We will bring transformative medicines to people suffering and eliminate all barriers to access.

Low Cost Provider

We will bring transformative medicines to people suffering and eliminate all barriers to access.

We have access to manufacturing through the Center for Breakthrough Medicines at reduced cost. 

Specialization

Dr. Wilson is one of the greatest minds in therapeutic development and genetic disease—combined with a full team and research lab to target tractable diseases.

Deep insights into rare diseases and the optimal approach to treatment is powered by the Orphan Disease Center at the University of Pennsylvania.

Solution-Driven Product

Very few non-profits have a margin producing strategy that is reinvested in future programs. This allows us to have a model of self-sustainability that is real, lasting and differentiated.

 

OUR MEDICINES

Targeting these rare diseases.

We are committed to changing the lives of 1 million people suffering from life threatening and devastating diseases around the world with no barriers to access.

Lesch-Nyhan Syndrome

10 babies are born each year in the US.

  • Cerebral palsy, huntington like movements, self-mutilation and gout

  • Neurological aspects have no treatments

  • Gene therapy in research phase with animal models

AADC Deficiency

10 babies are born each year in the US.

  • Severe congenital Parkinsons; never develop the ability to move their muscles

  • Experimental gene therapy restores virtually all muscle and CNS function

  • Phase 3 clinical trial underway; ready to file for registration

Crigler Najjar Type I

10 babies are born each year in the US.

  • Very high serum bilirubin creates risk of brain damage

  • Moderate control with total body blue light therapy 14 hrs/day or liver transplant

  • mRNA therapy works in animals; ready for clinical trial in 1 year

GENE THERAPY

Identification of a therapeutic gene

Cloning into a vector

Assembly into a vehicle

Injection

Expression of the therapeutic gene and disease cure

We believe a healthy life is a basic human right.

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