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Aug 20, 2026
The FDA has declined approval for Disc Medicine's rare disease therapy, which was previously associated with the commissioner's voucher program. The rejection marks a setback for the company's efforts to bring the treatment to market.
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.
Jun 22, 2026
Research estimates 300 million people worldwide live with rare diseases, yet only 5% of conditions have approved treatments. Patients face diagnostic delays of 4.7 to 8 years, with emerging AI and machine learning tools showing promise in accelerating diagnosis and drug development.
Jun 14, 2026
Researchers recommend a new long-read DNA test as the first choice for rare genetic disorders, showing a 3% higher diagnosis rate. Separately, a Chinese AI diagnostic system called DeepRare achieved 57.18% accuracy using only patient symptoms, with accuracy exceeding 70% when genetic data is included.
May 16, 2026
CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
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.
Apr 12, 2026
The global API market is projected to grow to USD 198.39 billion by 2030 from USD 144.20 billion in 2025, at a 6.6% CAGR. Oncology, rare disease and diabetes pipelines are key growth drivers.
Mar 31, 2026
South Korea has approved fast-track drug listing for rare disease treatments, reducing the insurance coverage process from 240 days to 100 days. The reform includes post-listing effectiveness evaluations and flexible pricing agreements, aiming to improve patient access to innovative treatments amid ongoing budget concerns.
Mar 30, 2026
The artificial intelligence in drug discovery market is projected to grow from USD 3.25 billion in 2026 to USD 10.29 billion by 2031, representing a 25.94% CAGR. AI technologies are accelerating drug discovery timelines, reducing costs, and improving success rates through advanced analytics and machine learning. North America leads market adoption while Asia-Pacific emerges as a high-growth region driven by expanding biotechnology sectors.
Mar 25, 2026
China's newly revised Implementation Regulations of the Drug Administration Law will take effect on May 15, 2026, representing the first comprehensive revision in over two decades. The regulations strengthen the marketing authorization holder system, improve drug development processes, and introduce new provisions for clinical trials, data protection, and market exclusivity for pediatric and rare disease drugs.
Mar 23, 2026
The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.
Mar 19, 2026
Global pharmaceutical R&D deal values surged 49% to $86.7 billion in 2025 as companies concentrate investments in AI-powered drug discovery platforms, with average deal sizes reaching a record $1.16 billion despite fewer total partnerships.
Mar 11, 2026
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Mar 09, 2026
Five intra-articular gene therapy platforms for knee osteoarthritis are in human trials in the US, while India prepares regulatory guidelines for mRNA, gene and cell therapies to accelerate innovation.
Mar 04, 2026
The global mRNA and next-generation vaccine platforms market is expected to grow from $39.69 billion in 2025 to $82.61 billion by 2030 at a 15.8% CAGR, driven by personalized medicine demand and infectious disease applications, while safety concerns and regulatory scrutiny continue.
Mar 04, 2026
Major pharmaceutical and biotech companies are urging the Trump administration to withdraw two Medicare pricing proposals that would align U.S. drug prices with international benchmarks, arguing the mandatory models exceed government authority and threaten innovation.
Mar 03, 2026
New research shows 73% of AI users in clinical trials report the technology met or exceeded expectations, with 70% confirming improvements in data accuracy and 93% of organizations already using or investigating AI applications.
Feb 28, 2026
Three interconnected life sciences infrastructure markets are experiencing significant growth driven by precision medicine adoption, increasing clinical trial activity, and advances in genomic diagnostics through 2035.
Feb 26, 2026
The FDA has proposed a new "plausible mechanism" pathway to approve customized treatments for rare diseases after testing in only a handful of patients, aiming to remove barriers for gene editing and other bespoke therapies that don't fit traditional approval systems.
