Rare and Orphan Diseases

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Our commitment to supporting healthcare till the last patient

Rare diseases are not rare^*. Around 7000 diseases are identified as rare worldwide, affecting about 300 million people. Many rare diseases are severe or life-threatening and often without treatment or therapy. Hence, there is an increased focus by the FDA and other regulatory bodies across the world on rare diseases, which reflects in over 50% of the drugs approved by the FDA in the past two years being orphan drugs.

How Syngene helped develop the world’s first drug for a rare disease affecting children

Syngene’s contribution to Orphan Drugs

Although the overall process for orphan drug development is similar to that of drugs with a wider patient base, some issues are much more evident with orphan drugs. These include a lack of natural history and early biomarkers, the non-availability of suitable in vitro/in vivo models, and the lack of suitable clinical outcomes or surrogate endpoints for clinical trials.

Syngene remains committed to delivering cutting-edge science toward orphan drug development and advancing the product pipeline. We have a proven track record in rare disease and orphan drug development, where we supported our clients in taking their orphan drug molecules from early discovery to manufacturing.

We offer an end-to-end spectrum of services from early discovery to manufacturing. This includes our computational and data sciences services, discovery biology services, preclinical safety assessment, process development, and manufacturing.

Orphan Drugs: Discovery, Development and Manufacturing services from Syngene

Computational & Data Sciences

Syngene’s computational and data sciences solutions enable informed decisions for research and development for rare diseases. Our interdisciplinary group of data scientists provides expertise in areas such as integrative data analysis, drug repurposing, systems modeling, and artificial intelligence (AI). This enables comprehensive data analysis, biomarker identification, patient stratification, and building in silico disease-progression models. We also help you to repurpose existing drugs and simulate development and manufacturing processes for efficiency and cost optimization.

Integrative Data Analysis	Target Assessment	Repurposing	Systems Modeling	Molecular Modeling
Analysis of multi omics big data Build analysis pipelines Derive biological, translational insights Identify biomarkers, targets, stratify patients	Target dossiers Target safety assessment reports Knowledgebases in area of interest Custom target related services	New indication for a molecule New therapeutic uses for a target Rare, neglected diseases Repositioning human to animal health	Disease models Efficacy, toxicity simulations Clinical trial simulations Manufacturing processes	Drug design Cheminformatics Antibody engineering Protein engineering

Discovery Biology

A common limitation in drug discovery for rare diseases is the lack of suitable in-vitro / in-vivo disease models for preclinical testing. The Discovery Biology team comprises highly experienced scientists who have established in vitro and in vivo models for rare diseases and can custom-build them to suit specific requirements. We enable you to establish suitable models and surrogate endpoints for use in clinical trials.

Preclinical Safety

Due to limitations in patient recruitment in clinical trials, preclinical safety studies have become paramount for rare diseases. Our Safety Assessment services provide a complete range of in vitro/in vivo tests to assess the safety of orphan drugs. We have a proven track record for on-time completion of projects leading to timely IND filing. This is critical to avail benefits of accelerated drug discovery approvals by regulatory bodies like the FDA for rare diseases.

Development

Syngene has a team with strong expertise in process development for developing the preclinical and clinical supplies of the drug substance. Recently, we helped one of our clients successfully develop the manufacturing process for a drug to treat a rare disease affecting children. We also supplied the required quantity of product for preclinical and clinical supply and later for commercial manufacturing. As a result, the product has become the first drug in the world to be approved for children suffering from the rare disease.