New research shows that many people with disease-causing gene mutations never develop illness, challenging traditional assumptions. Studies of 'modifier genes' and genetic outliers are revealing how some individuals stay healthy, potentially leading to new treatments for genetic diseases.
IDT's study showed UNCOVERseq achieved 97.6% sensitivity and 78% precision for CRISPR off-target nomination. Scribe will present new data at ASGCT and EAS, including STX-1150. Watchmaker licensed CRISPR-Cas9 IP for NGS normalization.
Alnylam reported Q4 2025 adjusted EPS of $1.25, beating estimates, while revenue of $1.10 billion missed consensus despite 85% growth. Amvuttra sales surged 189% to $826.6 million. The stock fell 4.1% after the mixed results.
LIB Therapeutics launched Lerochol in the United States after FDA approval for lowering LDL-C in adults with hypercholesterolemia, including HeFH. The once-monthly PCSK9 inhibitor is available with a $199-per-month cash-pay option.
Scientists have developed multiple breakthrough delivery systems for gene editing and mRNA therapies, including a simple amino acid supplement that increases delivery 20-fold and a CRISPR gene drive that reverses antibiotic resistance in bacterial populations.
The cardiovascular biologics market is projected to reach $4.23 billion by 2033, while the cell therapy market is expected to reach $5.38 billion by 2032, driven by RNA-based therapies and regulatory approvals.
Enlicitide decanoate demonstrated efficacy in reducing LDL cholesterol levels compared to placebo in patients with atherosclerotic cardiovascular disease history or risk, according to phase 3 CORALreef Lipids trial data.
The global CRISPR and prime editing market is forecast to grow at 24.1% CAGR through 2031, driven by therapeutic advances and regulatory progress. Scribe Therapeutics achieved a second milestone with Eli Lilly, while new RNA production and gene editing technologies accelerate research.
A phase three clinical trial shows enlicitide, an oral monoclonal antibody, reduced LDL cholesterol by 60% compared to placebo in 2,909 patients with or at risk of atherosclerosis over 24 weeks.
The FDA approved 46 novel drugs in 2025, down from 50 in 2024. Small molecules accounted for 31 approvals (67%), with oncology leading at nine approvals. Large molecules contributed 15 approvals (33%), spanning ADCs, bispecifics, and subcutaneous delivery innovations.
