GENE THERAPY

Gregor Mendel

The father of genetics.

DNA or deoxyribonucleic acid

The hereditary material in humans and almost all other organisms.

Mitochondria

Structures within cells that convert food into a form that cells can use.

GENE THERAPY

Also known as “Gene Editing”. The deliberate alteration of a selected DNA sequence.

1947

An article about Gene Therapy was published in the Journal of Heredity

1960 - 1968

Studies on the modification and DNA transfers using virus.

1972

Foundational proposal and rationale for gene therapy

1980

Premature and non-approved attempt at clinical Gene therapy

1977-1986

Development of adenovirus vectors

1990

First human gene transfer study

1999

Patient dies during a trial of gene therapy

2003

First regulatory approval of a gene therapy product

2008

The first clinical trial utilizing electrotransfer (electroporation)

2012

European Commission approves Glybera

2010-2013

Discovery and utilization of CRISPR-C

2013

FDA approval of Kynamro

2014

First clinical trial of genome editing

2015

FDA approves a first of its kind of product for the treatment of melanom

2016

FDA approval of Spinraza

2017

Use of “universal donor” TALEN- engineered CAR-T cells

2017

FDA approval of Kymriah, Yescarta, and Luxturna

LEBER’S CONGENITAL AMAUROSIS (LCA)

A genetic retina disorder that causes visual impairment beginning in infancy.

DUCHENNE MUSCULAR DYSTROPHY (DMD)

• A mutation in the Dystrophin gene effects both skeletal and cardiac muscles.

• Most commonly inherited myopathy in childhood

• Typically affects thighs & pelvis followed by arms

• Trouble standing up

• Most are unable to walk by the age of 12

• Gene therapy trials on mice and dogs have shown

BLOOD CANCER: ACUTE LYMPHOBLASTIC LEUKEMIA (ALL)

• Type of cancer where the bone marrow makes too many immature lymphocytes and affects the blood chemistry

• FDA has approved two (2) CAR-T therapies by 2017

• Patients show high remission rates after treatment