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%
- 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.
- Scenic Biotech announced a license and research agreement with Ono Pharmaceutical to utilize its Cell-Seq platform for discovering novel therapeutic targets. - This collaboration represents Scenic's fourth Cell-Seq partnership with a major pharmaceutical company, joining existing agreements with Genentech, Bristol Myers Squibb, and Alnylam. - The partnership validates Scenic's modifier therapy approach, which targets secondary genes to rebalance health rather than addressing primary disease-causing mutations directly.
- Royalty Pharma acquired a 1% royalty interest in Alnylam's AMVUTTRA from Blackstone Life Sciences for $310 million, extending through March 2035. - AMVUTTRA is an FDA-approved RNAi therapeutic for ATTR amyloidosis that achieved approximately $1 billion in sales in 2024 with 74% year-over-year growth. - The drug treats both hereditary TTR amyloidosis with polyneuropathy and TTR amyloidosis with cardiomyopathy, addressing a significant unmet medical need. - Analyst consensus projects AMVUTTRA sales to exceed $6 billion by 2028, highlighting the substantial market opportunity for this breakthrough therapy.
- Irish biotech startup Aerska has secured $21 million in seed funding to develop RNA interference therapies targeting degenerative neurological diseases including Alzheimer's and Parkinson's disease. - The company's antibody-oligo conjugate platform uses "brain shuttles" to overcome the blood-brain barrier challenge that has limited RNA therapeutics in the central nervous system. - Aerska aims to systemically deliver RNA medicines that can silence harmful genes in the brain, potentially treating, delaying, and preventing the onset of neurological diseases. - The funding round was co-led by Backed VC, Age1, and Speedinvest, with the company planning to expand operations in Dublin and London while advancing its drug discovery timeline.
- Novartis has signed an up to $5.2 billion licensing and options deal with China-based Argo Biopharmaceutical for experimental cardiovascular drugs using RNA interference technology. - The agreement includes rights to BW-00112, an RNAi drug in mid-stage development for severe hypertriglyceridemia, which causes elevated blood fat levels and increases cardiovascular disease risk. - Under the deal covering sales outside China, Argo will receive $160 million upfront plus up to $5.2 billion in milestone payments and royalties on future sales. - The collaboration includes options for two additional discovery-stage candidates targeting severe hypertriglyceridemia and mixed dyslipidemia, with total downstream milestones exceeding $9 billion.
