Biopharmaceutical Design

MendAGene LLC's unique in-house patent-pending technologies produce gene therapy designs in a snap. Our favored approach to gene therapeutics is different than gene editing, because of perceived concerns about off-target risks of gene editing. Our designs improve the chance of success and are capable of reducing costs of development and approval.

In our design process, we use natural history data, identify a specific molecular abnormality, and target the underlying or proximate biological alteration. As a platform for designing gene therapeutics, we have designed for a wide variety of genetic conditions to help reduce, mute, slow, treat, or prevent types of Parkinsons, Sickle Cell, Polycystic Kidney Diseases, cancer, and more. If you or your organization have interest in bringing better therapeutics to market, email us at designs@MendAGene.com

Current opportunities for licensing and collaboration include designs to help mitigate the following genetic conditions:

Gene Therapeutic Design Licensing and Collaboration Opportunities
Parkinsons Disease Sickle Cell Disease Cystic Fibrosis Stargardt Disease
Li-Fraumeni Syndrome Huntington Disease Canavan Disease Phenylketonuria
Polycystic Kidney Disease Pendred Syndrome Familial Amyloidosis Pompe Disease
Sandhoff Disease Bloom Syndrome MCAD Deficiency Wilson Disease
Friedreich Ataxia ADA Deficiency Biotinidase Deficiency Ataxia-Telangiectasia
Glutaric Acidemia Type I Krabbe Disease Homocystinuria Alkaptonuria
Mucopolysaccharidosis Type I Pyruvate Kinase Deficiency 21-hyroxylase Deficiency Hereditary Fructose Intolerance
Tyrosinemia Type I Citrullinemia Type I A-Betalipoproteinemia Isovaleric Acidemia
Nephropathic Cystinosis Neimann-Pick Type C1 Neimann-Pick Disease Types A/B Infantile Tay-Sachs Disease
Primary Hyperoxaluria Type I Argininosuccinic Aciduria Mucopolysaccharidosis Type VI DFNB89 Hearing Loss
Carbamoyl Phosphate Synthetase I Deficiency Congenital Disorder of Glycosylation Type 1A
VLC acyl-CoA Dehydrogenase Deficiency Juvenile Neuronal Ceroid Lipofuscinosis
Infantile Neuronal Ceroid Lipofuscinosis Glycogen Storage Disease Type III