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Aug 21, 2026
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.
Jul 21, 2026
Crispr Therapeutics AG trades at $47.78 with a $4.78B market cap, while Moderna trades at $61.82 with a $25.06B market cap, about 5.2 times larger. Both face losses, but CRISPR's Casgevy approval and Moderna's pipeline advances shape divergent analyst outlooks.
Jul 17, 2026
Data presented at EHA show that sickle cell disease patients have high interest in gene therapy, with 71% eager to learn more, despite limited awareness and concerns about safety and cost. Early clinical data from approved CRISPR-based therapies demonstrate significant reductions in vaso-occlusive crises and hospital admissions.
Jul 15, 2026
Vertex Pharmaceuticals dominates cystic fibrosis therapies with TRIKAFTA/KAFTRIO generating $2.57 billion in Q4 2025 and ALYFTREK ramping to $380.1 million. The company posted strong free cash flow of $3.19 billion in 2025 and is expanding into pain, kidney disease, and gene-editing therapies.
Jun 08, 2026
Canaccord Genuity initiated Beam Therapeutics with a buy rating and $74 price target, highlighting near-term catalysts. Beam reached an agreement with the FDA on an accelerated approval pathway for its BEAM-302 AATD therapy, which showed early clinical proof-of-concept for in vivo base editing. The company plans a BLA for risto-cel and a pivotal plan for BEAM-302 in early 2026.
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 10, 2026
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
May 09, 2026
Three advances in TB diagnostics: a portable $300 device delivering results in 30 minutes, a Quick DNA kit detecting drug resistance in 5 minutes, and an ultrasensitive test finding TB DNA in 12-16% of hospitalized US patients.
May 05, 2026
Vertex completed the rolling BLA submission for povetacicept in IgA nephropathy after positive Phase 3 RAINIER interim data. The company plans to use a Priority Review Voucher to accelerate FDA review.
Apr 26, 2026
More than 30 approved cell and gene therapies carry prices from nearly $1 million to over $4 million, creating fiscal pressure for Medicaid. A federal access model launched in 2024 aims to centralize outcomes-based contracting.
Apr 02, 2026
Transformative therapies including stem cell transplant and gene therapy offer sickle cell disease patients potential freedom from the SCD phenotype with reduced morbidities. About 90% of patients successfully switch to donor blood cell production after transplant, while gene therapies work by editing the patient's own stem cells. These treatments require careful consideration of disease severity and potential risks including major adverse events.
Mar 28, 2026
CRISPR Therapeutics reported a Q4 2025 loss of $1.37 per share, missing estimates, with revenues of $0.9 million falling short of expectations. The company's partner Vertex recorded $54 million in Casgevy sales for the quarter, with regulatory submissions for pediatric label expansion planned for early 2026.
Mar 24, 2026
The cell therapy manufacturing market is projected to reach $14.01 billion by 2035, with CAR-T therapies dominating at 65% market share. Recent FDA approvals for new CAR-T indications and Japanese regulatory acceleration highlight growing clinical adoption, while research advances include new anti-aging protein platforms and CRISPR-based treatments.
Mar 12, 2026
Scientists have developed two breakthrough approaches to dramatically enhance gene editing and mRNA therapy delivery: a simple amino acid supplement that increases CRISPR efficiency to nearly 90 percent, and a self-replicating CRISPR system that spreads between cells like a virus.
Mar 03, 2026
Sanofi's oral BTK inhibitor rilzabrutinib received orphan drug designation in Japan for IgG4-related disease and warm autoimmune hemolytic anemia, while also securing U.S. FDA breakthrough therapy designation for the rare anemia indication.
Mar 02, 2026
Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to Sanofi's rilzabrutinib for IgG4-related disease, marking the third global orphan designation for this indication based on positive phase 2 study data.
Feb 24, 2026
Health systems are learning that successful cell and gene therapy programs require early pharmacy involvement, strong payer partnerships, and flexible infrastructure to navigate complex contracts, workflows, and financial realities.
Feb 26, 2026
The FDA has issued draft guidance that may speed approval pathways for rare disease therapies using genome editing technologies, potentially benefiting CRISPR Therapeutics' pipeline of six clinical-stage candidates.
Feb 26, 2026
Approved gene-editing therapies for sickle cell disease priced at $2.2 million remain out of reach in Africa, where 80% of the world's cases occur, as Uganda launches mandatory newborn screening.
Feb 24, 2026
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.
