American biopharmaceutical company focused on discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases; founded 2002, headquartered in Cambridge, Massachusetts.
相关临床试验
139
127 进行中
药物批准
8
批准总数
监管机构
1
监管机构数
成立时间
2002
进行中（未招募）
127
91.4%
已完成
6
4.3%
招募中
5
3.6%
撤回
1
0.7%
- ADARx Pharmaceuticals priced an upsized IPO of 26.25 million shares at $17 each, raising about $446.3 million in gross proceeds. - AbbVie agreed to a concurrent private placement that would give it roughly 4.9% of ADARx, capped at $100 million. - Combined gross proceeds are expected to reach approximately $535.2 million before any underwriters' option exercise. - The capital funds onvuzosiran, an siRNA for hereditary angioedema now enrolling in the Phase III STOP-HAE trial.
- Roche will pay Atavistik Bio $70 million upfront and up to $1.9 billion in research, development and commercial milestones, plus royalties on approved drugs. - The partnership uses Atavistik's metabolite-protein screening platform to find cryptic binding pockets on proteins that conventional approaches struggle to reach. - Atavistik leads discovery and research while Roche takes over preclinical work, human testing, regulatory filings and commercialization in cardiovascular, renal and metabolic diseases. - Atavistik continues its own rare hematologic programs, led by oral allosteric AKT1 inhibitor ATV-1601 in a phase 1/2 trial for hereditary hemorrhagic telangiectasia.
- Alnylam outlined Phase III plans for zilebesiran, an RNAi therapy targeting angiotensinogen to deliver blood pressure control with twice-yearly 300 mg injections. - The event-driven ZENITH trial, run with Roche, began last September and aims to enroll 11,000 patients across 35 countries with at least two years of follow-up. - Phase II KARDIA data showed a placebo-adjusted systolic blood pressure reduction of 10 mmHg with monotherapy and sustained 24-hour control for one year. - Alnylam expects a potential 15% to 20% reduction in major adverse cardiovascular events, and the program holds FDA Fast Track and China Breakthrough Therapy designations.
- Roche and Genentech opened the Roche Genentech Innovation Center in Boston's Allston neighborhood at Harvard's Enterprise Research Campus on Thursday. - The site will serve as Roche's cardiovascular, renal and metabolism hub, spanning basic research, clinical development, artificial intelligence and scientific data work. - A 10-year lease covers 95,000 square meters, with capacity for up to 500 specialists in research, clinical development and data science roles. - The investment is part of Roche's $50 billion commitment to US manufacturing and research, building on sites in North Carolina and Oregon.
- RNA interference (RNAi) therapies using small interfering RNA (siRNA) are already approved by the FDA, with at least seven treatments available for genetic diseases and more in clinical trials. - The global RNAi therapeutics market is projected to grow from about $1.5 billion in 2024 to roughly $5.1 billion in 2034, reflecting rapid commercial expansion. - Delivery beyond the liver is a key frontier, with researchers developing siRNAs targeting muscles, nerves, the placenta, and other organs. - Korean firms OliX and rznomics signed technology transfer deals with Eli Lilly worth 900 billion won and 1.9 trillion won, respectively, underscoring global interest in RNA-based platforms.
- Creative Life Science reported record first-half 2026 revenue of NT$568 million, up 13.18% year-on-year, driven by regenerative medicine materials and services. - The company's 2,180-square-meter Zhubei facility is PIC/S GMP-compliant, featuring ISO Class 5 isolators and automated aseptic filling systems for cell therapy manufacturing. - Taiwan's new regenerative medicine laws, fully effective January 2026, are raising requirements for material traceability, aseptic manufacturing, and batch release testing. - Creative Life Science is expanding into Japan and Singapore, building on a late-2025 cooperation agreement with Singapore-based ESCO for Southeast Asian market access.
- The average biopharmaceutical employee earned $116,648 in 2025, roughly 30% more than the average S&P 500 worker at $89,744, according to an Endpoints News study. - Gilead topped Big Pharma with a median salary of $239,000, while biotech firms like Alnylam ($312,000) and Ionis ($305,000) far exceeded even the most generous large drugmakers. - Johnson & Johnson, the industry's perennial revenue leader, ranked only 10th in median salary at $91,000, demonstrating that company size and profitability do not directly correlate with employee pay. - The 32 large biotech companies studied collectively employ over one million people with a combined market capitalization of $3.88 trillion.
- Regeneron's investigational siRNA therapeutic cemdisiran demonstrated superior efficacy over placebo in the Phase 3 NIMBLE trial for generalized myasthenia gravis, meeting both primary and key secondary endpoints with a 2.3-point improvement in daily living activities (p<0.001). - The therapy showed rapid onset of action within two weeks and sustained benefits through 24 weeks with convenient quarterly dosing, potentially offering best-in-class efficacy compared to approved C5 inhibitors. - Cemdisiran exhibited a favorable safety profile with lower infection rates than placebo and could become the first siRNA therapy approved for myasthenia gravis treatment following U.S. regulatory submission in Q1 2026.
- Blackstone announced the final close of its Life Sciences VI fund at $6.3 billion, marking the largest private fund ever raised for life sciences and representing a nearly 40% increase from its predecessor vehicle. - The oversubscribed fund reflects Blackstone's strong track record in life sciences, with partnerships producing 34 regulatory approvals of innovative medicines and devices since launching the platform in 2018. - Blackstone Life Sciences has achieved an 86% approval success rate for Phase III assets, outperforming industry averages, and committed nearly $2 billion in new investments over the past 12 months. - The fund closure comes as the life sciences real estate market shows signs of recovery after two years of oversupply, with construction hitting 10-year lows and demand stabilizing.
- Corsera Health secured $80 million in Series A funding co-led by Forbion and Population Health Partners to advance its preventative cardiovascular disease programs. - The company's lead siRNA therapy COR-1004, targeting PCSK9 to lower LDL cholesterol, has entered Phase I clinical trials with dosing underway and proof-of-concept data expected this year. - Founded by biotech veterans John Maraganore and Clive Meanwell, Corsera aims to develop once-yearly treatments combined with AI-enabled risk prediction tools to prevent atherosclerotic cardiovascular disease. - The company was recognized as one of BioSpace's NextGen Class of 2026 startups to watch, emerging during a challenging fundraising environment for early-stage biotechs.
