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Aug 06, 2026
Whole genome sequencing is improving rare disease diagnosis, with studies from Scotland, Sweden, and the Netherlands reporting diagnostic yields around 23% and long-read sequencing matching standard care with 96.4% concordance.
Jul 23, 2026
AI-driven drug discovery attracts major investment and partnerships: Galux closes $29M Series B, LG Chem partners with LabGenius on cancer antibodies, Cheiron raises $8M seed, and Peptris secures $7.7M. Galux also teams with AimedBio on BBB-crossing proteins.
Jul 03, 2026
Anthropic announced it will run its own preclinical drug-discovery programs for neglected and rare diseases and launched Claude Science, an AI workbench for researchers. The company has not named specific diseases or detailed how it would bring candidates to market.
Jun 09, 2026
Global drugmakers are expanding rare disease research investment in Brazil, attracted by new regulations, genetic diversity, and the public healthcare system's data capabilities. Major companies including AstraZeneca, Biogen, and Roche have committed significant clinical research budgets to the country. Despite this progress, advocates warn that a significant global funding gap persists, with 95% of rare diseases still lacking an FDA-approved treatment.
May 20, 2026
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.
May 19, 2026
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.
May 18, 2026
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.
May 05, 2026
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.
May 05, 2026
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.
May 04, 2026
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.
May 04, 2026
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.
May 02, 2026
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.
Apr 22, 2026
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.
Apr 19, 2026
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.
Apr 18, 2026
India's rare disease treatment funding fell to Rs 32.73 crore in 2025-26 from Rs 82.87 crore a year earlier, while several Centres of Excellence reported unspent balances. In Madhya Pradesh, concerns were also raised over the absence of dedicated rare disease centres in most state-run medical colleges.
Apr 17, 2026
Digital drug design tools and AI are gaining ground as drug development timelines run 10 to 14 years and approval rates remain low. Recent work describes broader AI adoption and an information-theoretic predictive model for drug design.
Apr 14, 2026
The Consolidated Appropriations Act, 2026 extends the FDA’s rare pediatric disease priority review voucher authority through Sept. 30, 2029. The law also requires a GAO report on the program’s effectiveness.
Apr 07, 2026
CRISPR Therapeutics reported a $581.6 million net loss in 2025 with revenue of just $3.5 million, while continuing to advance its gene-editing pipeline including the approved CASGEVY therapy and next-generation CAR T cell programs. The company maintains strategic partnerships and expects current funds to support operations for at least 24 months.
Apr 03, 2026
The FDA has granted orphan drug designation to Nuformix PLC's tranilast lysate candidate for treating idiopathic pulmonary fibrosis. The designation provides regulatory incentives including potential market exclusivity in the US. Nuformix is a London-based developer focused on fibrosis and oncology treatments.
Apr 02, 2026
The FDA will drop its longtime requirement for two rigorous studies to approve new drugs, moving to a default position of requiring only one study. Commissioner Marty Makary says the change reflects modern scientific advances and aims to accelerate drug availability without compromising safety. The shift follows decades of increasing flexibility for rare and fatal diseases, with about 60% of first-of-a-kind drugs already approved based on single studies in recent years.
