Also known as: rare conditions
Research and Markets added two cell and gene therapy publications, including a tools and reagents market report forecasting growth from $12 billion in 2025 to $19.8 billion by 2030. The reports track approvals, financing, deals, and manufacturing developments.
The FDA granted Orphan Drug Designation to Cullinan Therapeutics’ CLN-049 for relapsed/refractory AML. CLN-049 is in Phase 1 studies and had already received Fast Track designation.
Drug repurposing expands the therapeutic applications of existing medications, while repositioning examines drugs that never reached market. In neurodegeneration, the approach is described as a pragmatic shortcut into mid- to late-stage trials.
India said its 2021 rare disease policy expanded Centres of Excellence from 8 to 15 and raised financial assistance to Rs 50 lakh. But rare disease treatment can cost up to Rs 1 crore or more annually.
India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.
The FDA said one pivotal trial plus confirmatory evidence will become the default standard for approval of most new drugs. The shift could extend beyond rare diseases to common conditions affecting millions.
European rare disease and healthcare policy conferences are scheduled for 2026 in Prague, Brussels and Cyprus. The meetings will address orphan products, European Reference Networks, cross-border healthcare, research collaboration and patient access to treatment.
FDA released its 2026 Rare Disease Hub Strategic Agenda and a draft guidance on 3-year New Clinical Investigation Exclusivity. The agency also outlined new pathways including the plausible mechanism pathway and Rare Disease Evidence Principles to accelerate treatments for rare diseases.
Peptris has raised INR 70 crore ($7.7 million) in a Series A round to advance programs toward clinical readiness and expand its AI-led drug discovery pipeline over 24 months.
Rznomics said it will present interim AACR results for liver cancer candidate RZ-001 from a U.S. trial in patients with hepatocellular carcinoma. Tumor-shrinking responses were observed, and about 23% experienced complete disappearance of cancer cells.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07558213 |
A Multi-Dimensional Model of cAre and transItion for Patients With cOmplex RAre Diseases |
ACTIVE_NOT_RECRUITING | |
| NCT07527624 |
Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability |
RECRUITING | |
| NCT07497581 |
A Longitudinal Study on Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases |
NOT_YET_RECRUITING | |
| NCT07400913 |
Implementation of Long-read Sequencing for the Diagnosis of Rare Diseases. |
NOT_YET_RECRUITING | |
| NCT07247292 |
Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia |
SUSPENDED | |
| NCT07247279 |
Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia |
RECRUITING | |
| NCT07205861 |
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center |
RECRUITING | |
| NCT07102966 |
Genetic Study to Determine the Cause of Birth Defects in Newborns in Texas |
RECRUITING | NA |
| NCT07063719 |
Identification of Cellular Biomarkers of Rare Eye Diseases in Adults |
NOT_YET_RECRUITING | NA |
| NCT06926127 |
Genomic Profiling of Genetic and Rare Diseases |
RECRUITING | NA |
