Intellia Therapeutics, Inc. is a clinical stage genome editing company, which engages in the development of curative therapeutics using the CRISPR/Cas9 system. Its CRISPR/Cas9 system transforms medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creates engineered cells that can treat oncological and immunological diseases. The company was founded by Andrew May, Luciano Marraffini, Rodolphe Barrangou, Nessan Bermingham, Rachel Haurwitz, Erik Sontheimer, Jennifer Doudna, and Derrick Rossi in May 2014 and is headquartered in Cambridge, MA.
相关临床试验
15
7 进行中
药物批准
0
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监管机构
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监管机构数
成立时间
2014
进行中（未招募）
6
40.0%
已完成
1
6.7%
Enrolling By Invitation
1
6.7%
招募中
4
26.7%
终止
1
6.7%
Unknown
1
6.7%
撤回
1
6.7%
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- The FDA accepted Intellia Therapeutics' Biologics License Application for lonvoguran ziclumeran (lonvo-z) in hereditary angioedema and granted Priority Review with a PDUFA date of March 10, 2027. - The application is supported by global Phase 3 HAELO data showing an 87% reduction in mean monthly attacks versus placebo over the six-month efficacy evaluation period. - The FDA has indicated it does not currently plan to convene an advisory committee, and Intellia aims to launch lonvo-z commercially in the first half of 2027 if approved. - If cleared, lonvo-z would become the world's first in vivo CRISPR-based therapy and the only one-time treatment for HAE, potentially shifting care away from lifelong prophylactic regimens.
- Mana.bio has appointed Dr. Laura Sepp-Lorenzino, former CSO of Intellia Therapeutics, to its Scientific Advisory Board to guide the company's AI-driven LNP discovery platform. - Dr. Sepp-Lorenzino brings decades of leadership experience from Intellia, Vertex Pharmaceuticals, Alnylam, and Merck, spanning CRISPR, RNAi, and nucleic acid therapies. - Mana.bio's proprietary AI/ML platform leverages the world's largest curated LNP dataset to predict safety, tropism, and stability, with NHP-validated delivery solutions for extrahepatic targets. - The appointment comes at what CEO Yogev Debbi describes as a pivotal stage of growth as the company scales its discovery engine and expands its therapeutic pipeline.
- The global gene editing therapeutics market is projected to grow at a compound annual growth rate of 12.5%, fueled by expanding clinical applications and rising disease burden. - CRISPR-based technologies dominate the market due to their efficiency and cost-effectiveness, while oncology represents the largest therapeutic application segment. - High development costs, regulatory complexity, and off-target safety concerns remain significant barriers to broader adoption and commercialization. - Key players including CRISPR Therapeutics, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics are driving innovation through partnerships and investments in next-generation editing platforms.
- Intellia Therapeutics' CRISPR-based treatment lonvo-z demonstrated an 87% reduction in swelling attacks compared to placebo in an 80-patient Phase 3 trial for hereditary angioedema. - The single-dose, in vivo gene editing therapy resulted in 62% of patients being completely attack-free six months after treatment, compared to 11% in the placebo group. - If approved, lonvo-z would become the second FDA-approved CRISPR medicine and the first to edit DNA directly inside the body, with Intellia planning to complete regulatory filing in the second half of 2026.
- AvenCell Therapeutics has dosed the first patient in its Phase I QUADvance study with AVC-203, marking the world's first CRISPR-engineered allogeneic CAR-T therapy to simultaneously target CD19 and CD20 antigens. - The therapy incorporates four key innovations including dual antigen targeting, immune evasion through CRISPR engineering, improved T-cell fitness from healthy donors, and switchable targeting capabilities for future indication expansion. - The program is supported by a $40 million grant from Japan's AMED and received FDA IND clearance and EMA approval on first submission, enabling trial initiation across multiple sites in the US and Europe. - AVC-203 addresses critical limitations of current autologous CAR-T therapies by offering off-the-shelf availability, consistent product quality, and massively scalable manufacturing for patients with relapsed/refractory B-cell malignancies.
- The FDA has lifted a clinical hold on Intellia Therapeutics' late-stage trial testing nexiguran ziclumeran, a CRISPR-based gene therapy for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). - The clinical hold was imposed in October after a patient developed severe liver-related side effects, but Intellia has since agreed to stronger safety monitoring measures. - A separate trial testing the same therapy for the cardiac form of the disease remains on hold following a patient death in November. - The company has expanded the nerve disease trial enrollment from 50 to 60 patients and plans to restart patient enrollment immediately.
- AvenCell Therapeutics has received FDA IND clearance and EMA approval for the QUADvance Phase I/II trial of AVC-203, a CRISPR-engineered allogeneic CAR-T therapy targeting both CD19 and CD20 for relapsed/refractory B-cell malignancies. - AVC-203 incorporates four key innovations including dual antigen targeting, immune evasion capabilities, improved T-cell fitness from healthy donors, and switchable targeting technology for future expansion beyond CD19/CD20. - The multi-site trial will evaluate safety, tolerability, efficacy, and pharmacokinetics in adults with B-cell malignancies, addressing the scalability and cost challenges of current autologous CAR-T therapies.
- The FDA approved three new hereditary angioedema treatments in just three months during 2025: CSL's Andembry in June, KalVista's Ekterly in July, and Ionis's Dawnzera in August. - These approvals represent breakthrough innovations including the first factor XIIa inhibitor, first oral on-demand therapy, and first RNA-targeting treatment for HAE prevention. - The rapid succession of approvals brings the total number of FDA-approved HAE treatments from 9 to 12, offering unprecedented treatment options for the estimated 7,000 U.S. patients. - Despite the therapeutic advances, significant unmet needs remain, particularly for pediatric patients under 12 years old, as 85% of HAE patients develop symptoms before age 20.
- Intellia Therapeutics paused dosing and screening in its MAGNITUDE and MAGNITUDE-2 Phase 3 trials of nexiguran ziclumeran (nex-z) following a patient's hospitalization with grade 4 liver transaminase elevation and increased bilirubin levels. - The suspended trials involve over 650 patients with transthyretin amyloidosis with cardiomyopathy and 47 patients with polyneuropathy, with approximately 450 patients already having received the one-shot CRISPR gene-editing therapy. - The company's stock plummeted more than 45% in pre-market trading as investors reacted to the safety concerns affecting one of Intellia's two priority late-stage programs. - Intellia is consulting with experts and regulatory authorities to investigate risk-mitigation strategies while maintaining its commitment to patient safety and regulatory compliance.
- Cathie Wood's ARK Invest recently invested $4 million in Intellia Therapeutics, acquiring 349,930 shares as institutional confidence builds in the gene-editing company. - The company's lead therapy NTLA-2002 for hereditary angioedema demonstrates 98% reduction in attack rates over three years, with potential regulatory filing by second half of 2026. - Intellia's Phase 3 ATTR trial is expanding to approximately 1,200 participants due to accelerated patient recruitment, indicating strong clinical momentum. - Despite stock volatility with shares dropping from above $30 to $11.36, the company maintains over $630 million in cash to fund operations through first half of 2027.
