Genetic Disease | Novartis

Access to Healthcare

Breaking down barriers for patients with sickle cell disease

One patient’s story reveals the hidden obstacles to better health – and opportunities to help clear them.

Access to HealthcareDisease AwarenessGenetic DiseaseLiving With DiseasePatient Voices
Discovery

Discovery gives cystic fibrosis researchers new direction

Novartis-Harvard team identifies a new type of cell that is key to the disease.

DiseasesDrug DiscoveryNovartis Institutes for BioMedical ResearchScientific ResearchGenetic Disease
From Our Labs

When tiny brains are best for brain research

Zebrafish imaging sheds light on human neurological conditions.

Biomedical ResearchDrug DiscoveryNeuroscienceNovartis Institutes for BioMedical ResearchPostdoc ProgramGenetic Disease
From Our Labs

Fixing what’s lost in translation

Drug DiscoveryNovartis Institutes for BioMedical ResearchRare DiseasesNew Scientific DiscoveriesGenetic Disease
From Our Labs

Eye disease tracked to a glitch in starburst cells

A cellular computing error wreaks havoc on vision.

Novartis Institutes for BioMedical ResearchNew Scientific DiscoveriesScientific ResearchGenetic Disease
From Our Labs

Electrical brainstorms traced to genetic mutations

Researchers from Seattle Children’s and Novartis shed light on developmental brain disorders that lead to intractable epilepsy.

Biomedical ResearchGene SequencingNovartis Institutes for BioMedical ResearchReimagine MedicineNext Generation SequencingGenetic Disease
Discovery

Small viruses could accelerate cell and gene therapy research

Novartis collaborates with Homology Medicines to adapt and refine genome editing technology.

Cell and Gene TherapyDigitalEmerging TechnologyInnovationNovartis Institutes for BioMedical ResearchGenetic Disease
Discovery

Rewriting the playbook for clinical testing in a rare disease

A Novartis team blazes new trails with an experimental treatment for spinal muscular atrophy.

Novartis Institutes for BioMedical ResearchRare DiseasesClinical TrialsGenetic Disease
Discovery

Rare speed on a rare disease

An investigational treatment for an uncommon immunodeficiency disease races into clinical studies.

Novartis Institutes for BioMedical ResearchRare DiseasesReimagine MedicineTargeted TherapyClinical ResearchClinical TrialsGenetic Disease
Discovery

Novartis researches SMA’s genetic roots

Spinal muscular atrophy (SMA) is the most common genetic casue of infant and toddler death. NIBR scientists are trying to make a back up gene take up the slack.

Novartis Institutes for BioMedical ResearchRare DiseasesClinical ResearchCareersBiomedical JobsGene TestingGenetic Disease
Discovery

Neuroscientists restore cell-to-cell signaling and sociability in autism models

Team uncovers a potential therapeutic approach for patients deficient in a protein called Shank3.

Novartis Institutes for BioMedical ResearchPostdoc ProgramNew Scientific DiscoveriesBiomedical ScienceGenetic Disease
Discovery

Making Possible: Stepping toward a future with cell and gene therapy

These stories offer a glimpse into the lives of people touched by a new era of medicine.

Cell and Gene TherapyEmerging TechnologyFuture of HealthcareReimagine MedicineGenetic Disease