Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency
Intellia Therapeutics (NASDAQ:NTLA) has received authorization from the UK's MHRA to initiate a Phase 1/2 clinical trial for NTLA-3001, a potential one-time CRISPR-based gene editing treatment for alpha-1 antitrypsin deficiency (AATD)-associated lung disease. NTLA-3001 aims to normalize AAT protein levels by inserting a healthy copy of the SERPINA1 gene, potentially halting disease progression and eliminating the need for weekly infusions or lung transplants.
The study will enroll up to 30 adult patients and evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics. Intellia plans to dose the first patient in the second half of 2024. This trial marks Intellia's first wholly owned in vivo targeted gene insertion candidate to enter clinical stages, validating their modular gene insertion platform for addressing various protein deficiency diseases.
Intellia Therapeutics (NASDAQ:NTLA) ha ricevuto l'autorizzazione dalla MHRA del Regno Unito per avviare un trial clinico di Fase 1/2 per NTLA-3001, un potenziale trattamento di editing genico basato su CRISPR per la malattia polmonare associata alla deficienza di alfa-1 antitripsina (AATD). NTLA-3001 mira a normalizzare i livelli di proteina AAT inserendo una copia sana del gene SERPINA1, potenzialmente fermando la progressione della malattia ed eliminando la necessità di infusioni settimanali o trapianti polmonari.
Lo studio iscriverà fino a 30 pazienti adulti e valuterà sicurezza, tollerabilità, farmacocinetica e farmacodinamica. Intellia prevede di somministrare la prima dose al secondo semestre del 2024. Questo trial segna il primo candidato alla somministrazione di geni mirati in vivo, interamente di proprietà di Intellia, a entrare nelle fasi cliniche, convalidando la loro piattaforma di inserzione di geni modulare per affrontare varie malattie da carenza proteica.
Intellia Therapeutics (NASDAQ:NTLA) ha recibido autorización de la MHRA del Reino Unido para iniciar un ensayo clínico de Fase 1/2 para NTLA-3001, un posible tratamiento de edición genética basado en CRISPR para la enfermedad pulmonar asociada a la deficiencia de alfa-1 antitripsina (AATD). NTLA-3001 tiene como objetivo normalizar los niveles de proteína AAT insertando una copia sana del gen SERPINA1, deteniendo potencialmente la progresión de la enfermedad y eliminando la necesidad de infusiones semanales o trasplantes de pulmón.
El estudio incluirá hasta 30 pacientes adultos y evaluará la seguridad, tolerabilidad, farmacocinética y farmacodinámica. Intellia planea dosificar al primer paciente en la segunda mitad de 2024. Este ensayo marca el primer candidato de inserción genética dirigida in vivo, totalmente de propiedad de Intellia, que entra en las etapas clínicas, validando su plataforma de inserción de genes modular para abordar diversas enfermedades por deficiencia de proteínas.
인텔리아 테라퓨틱스(NASDAQ:NTLA)는 영국의 MHRA로부터 NTLA-3001에 대한 1/2상 임상 시험을 시작할 수 있는 승인을 받았습니다. 이는 알파-1 항트립신 결핍(AATD)와 관련된 폐 질환에 대한 잠재적인 일회성 CRISPR 기반 유전자 편집 치료입니다. NTLA-3001은 SERPINA1 유전자의 건강한 사본을 삽입하여 AAT 단백질 수치를 정상화하는 것을 목표로 하며, 이로 인해 질병 진행을 멈추고 주간 주입이나 폐 이식의 필요성을 없애는 것이 가능합니다.
이 연구는 최대 30명의 성인 환자를 등록하고 안전성, 내약성, 약동학 및 약리학적 효과를 평가할 것입니다. 인텔리아는 2024년 하반기에 첫 번째 환자에게 투약할 계획입니다. 이번 시험은 인텔리아가 소유한 전적으로 비임상 단계에 들어가는 최초의 직접적인 유전자 삽입 후보이며, 다양한 단백질 결핍 질환을 해결하기 위한 모듈형 유전자 삽입 플랫폼의 유효성을 입증합니다.
Intellia Therapeutics (NASDAQ:NTLA) a obtenu l'autorisation de l'MHRA du Royaume-Uni pour lancer un essai clinique de phase 1/2 pour NTLA-3001, un potentiel traitement d'édition génique basé sur CRISPR pour la maladie pulmonaire associée à la déficience en alpha-1 antitrypsine (AATD). NTLA-3001 vise à normaliser les niveaux de protéine AAT en insérant une copie saine du gène SERPINA1, ce qui pourrait arrêter la progression de la maladie et éliminer le besoin d'infusions hebdomadaires ou de transplantations pulmonaires.
L'étude recrute jusqu'à 30 patients adultes et évaluera la sécurité, la tolérance, la pharmacocinétique et la pharmacodynamie. Intellia prévoit de traiter le premier patient dans la deuxième moitié de 2024. Cet essai marque le premier candidat à l'insertion ciblée de gènes in vivo entièrement détenu par Intellia, validant leur plateforme modulaire d'insertion de gènes pour traiter diverses maladies dues à des carences protéiques.
Intellia Therapeutics (NASDAQ:NTLA) hat die Genehmigung der MHRA des Vereinigten Königreichs erhalten, um eine klinische Studie der Phase 1/2 für NTLA-3001 zu starten, eine potenzielle einmalige CRISPR-basierte Genbearbeitung zur Behandlung von alpha-1 Antitrypsin-Mangel (AATD)-assoziierten Lungenkrankheiten. NTLA-3001 zielt darauf ab, die AAT-Proteinwerte zu normalisieren, indem eine gesunde Kopie des SERPINA1-Gens eingesetzt wird, was möglicherweise das Fortschreiten der Krankheit stoppt und die Notwendigkeit für wöchentliche Infusionen oder Lungentransplantationen beseitigt.
Die Studie wird bis zu 30 erwachsene Patienten rekrutieren und Sicherheit, Verträglichkeit, Pharmakokinetik und Pharmakodynamik bewerten. Intellia plant, den ersten Patienten in der zweiten Hälfte von 2024 zu behandeln. Diese Studie markiert den ersten vollständig im Besitz von Intellia befindlichen Kandidaten zur gezielten Geneinschubtherapie, der in klinische Phasen eintritt, was die Modularität ihrer Genbearbeitungsplattform zur Behandlung verschiedener Proteinmangelkrankheiten validiert.
- Authorization received to initiate Phase 1/2 clinical trial for NTLA-3001
- NTLA-3001 is Intellia's first wholly owned in vivo targeted gene insertion candidate to advance to clinical stage
- Potential to eliminate need for weekly infusions or lung transplants in AATD patients
- Validates Intellia's modular gene insertion platform for addressing multiple diseases
- Clinical trial results and efficacy of NTLA-3001 are yet to be determined
- Regulatory approvals in other countries still pending
Insights
The authorization of Intellia Therapeutics' Clinical Trial Application (CTA) for NTLA-3001 marks a significant milestone in the field of gene editing therapeutics. This development is particularly noteworthy for several reasons:
- NTLA-3001 represents Intellia's first wholly-owned CRISPR-based in vivo targeted gene insertion candidate to enter clinical trials, showcasing the company's progress in translating its technology from bench to bedside.
- The potential one-time treatment approach for alpha-1 antitrypsin deficiency (AATD) could revolutionize patient care, potentially eliminating the need for weekly protein infusions or lung transplants in severe cases.
- The use of systemic administration for in vivo gene editing is a bold step forward, demonstrating confidence in the safety and precision of Intellia's CRISPR/Cas9 platform.
From an investor's perspective, this authorization strengthens Intellia's position in the competitive gene editing landscape. The company's ability to advance a complex therapeutic candidate like NTLA-3001 into clinical trials validates its technological capabilities and could attract increased attention from both the scientific community and potential partners.
However, it's important to note that this is still an early-stage trial and success is not guaranteed. The Phase 1/2 study's results, particularly regarding safety and efficacy, will be critical in determining the long-term potential of NTLA-3001 and Intellia's gene insertion platform as a whole.
Investors should closely monitor the trial's progress, expected to begin dosing in the second half of 2024, as positive outcomes could significantly impact Intellia's market valuation and future prospects in the gene editing therapeutics space.
The advancement of NTLA-3001 into clinical trials represents a potentially groundbreaking approach to treating alpha-1 antitrypsin deficiency (AATD). This development is significant for several reasons:
- NTLA-3001 aims to address the root cause of AATD by inserting a functional copy of the SERPINA1 gene, which could lead to sustained production of the alpha-1 antitrypsin (AAT) protein at therapeutic levels.
- If successful, this one-time treatment could eliminate the need for lifelong weekly protein infusions, significantly improving patients' quality of life and potentially reducing healthcare costs.
- The systemic administration of the CRISPR/Cas9 gene editing components demonstrates progress in overcoming delivery challenges associated with in vivo gene editing.
The design of the Phase 1/2 trial, with up to 30 patients and including both dose-escalation and dose-expansion phases, is robust and should provide valuable data on safety, tolerability and initial efficacy signals. The open-label nature of the trial may allow for early insights into the treatment's potential.
It's important to note that while this approach shows promise, it also carries risks. The long-term effects of in vivo gene editing are still unknown and careful monitoring for off-target effects and immune responses will be crucial. Additionally, the complexity of AATD, which can affect both the lungs and liver, may present challenges in fully addressing the disease with this approach.
As the trial progresses, key metrics to watch will include changes in serum AAT levels, improvements in lung function and any signs of adverse events related to the gene editing process. The success of NTLA-3001 could have far-reaching implications, not just for AATD treatment, but for the broader field of genetic medicine.
The authorization of Intellia Therapeutics' CTA for NTLA-3001 is a positive development for the company's financial outlook and market position. Here's why:
- Expansion of Clinical Pipeline: NTLA-3001 adds depth to Intellia's clinical-stage portfolio, potentially diversifying revenue streams in the future.
- First-Mover Advantage: As the first wholly-owned in vivo CRISPR-based gene insertion candidate, NTLA-3001 could position Intellia as a leader in this specific application of gene editing technology.
- Market Opportunity: While AATD is a rare disease, successful treatment could command premium pricing, typical of orphan drug designations.
Financially, this development could impact Intellia in several ways:
Short-term considerations: Clinical trials are expensive and advancing NTLA-3001 will increase R&D expenses. Investors should expect higher cash burn rates in the coming quarters.
Long-term potential: If successful, NTLA-3001 could become a significant revenue driver. The one-time treatment model could lead to high per-patient revenues, although the total addressable market for AATD is relatively small.
Valuation metrics: Biotech companies are often valued based on their pipeline potential. The addition of NTLA-3001 to clinical-stage assets may positively influence Intellia's valuation multiples.
Partnership opportunities: Success with NTLA-3001 could attract potential partners for Intellia's gene insertion platform, potentially leading to lucrative collaboration deals.
However, it's important to remember that drug development is inherently risky. Many candidates fail in clinical trials and Intellia's stock price may be volatile as it releases updates on NTLA-3001's progress. Investors should carefully consider their risk tolerance and the speculative nature of early-stage biotech investments.
- NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD)
- NTLA-3001 is Intellia’s first wholly owned CRISPR-based in vivo targeted gene insertion candidate to advance into the clinic
- On track to dose the first patient in 2H 2024
CAMBRIDGE, Mass., July 30, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. AATD is a rare, genetic disease that most commonly manifests in lung dysfunction due to insufficient levels of alpha-1 antitrypsin (AAT) protein. NTLA-3001 is a systemically administered in vivo CRISPR/Cas9-based targeted gene insertion candidate. It is designed to precisely insert a healthy copy of the SERPINA1 gene, which encodes the AAT protein, with the potential to restore permanent expression of functional AAT protein to therapeutic levels after a single dose. This approach seeks to improve patient outcomes, including eliminating the need for weekly intravenous infusions of AAT augmentation therapy or lung transplant in severe cases.
“NTLA-3001 is a groundbreaking in vivo CRISPR-based gene insertion candidate designed to durably produce functional AAT protein at normal levels after a one-time treatment. We are excited to receive regulatory authorization to begin this important first-in-human study of NTLA-3001 for people living with AATD,” said Intellia President and Chief Executive Officer John Leonard, M.D. “In addition, this study serves to validate our modular gene insertion platform, which we plan to leverage to address numerous diseases caused by a missing or defective protein.”
The Phase 1/2 study will be an international, multicenter, single-arm, open-label study of NTLA-3001 in adults with AATD-associated lung disease. The study will enroll up to 30 patients and consist of a dose-escalation phase, followed by a dose-expansion phase to confirm the recommended dose. The study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-3001. More information about the study may be found on clinicaltrials.gov when available.
Beyond its first application in the United Kingdom, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development strategy for NTLA-3001.
About NTLA-3001
NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert a copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (AAT) protein, with the potential to restore permanent expression of functional AAT protein to therapeutic levels after a single dose. This approach seeks to improve patient outcomes, including eliminating the need for weekly intravenous infusions of AAT augmentation therapy or lung transplant in severe cases.
About Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition that increases the risk of liver and lung disease. AATD is caused by changes in the SERPINA1 gene that normally provides instructions for making alpha-1 antitrypsin (AAT) protein in the liver that is then secreted to protect the lungs. Mutations to the SERPINA1 gene lead to the production of abnormal AAT protein that then accumulates in the liver. As a result, AAT protein levels in the blood and lungs are very low. The shortage of AAT in the blood and lungs places the lungs at risk for emphysema, a type of chronic obstructive pulmonary disease (COPD). AATD occurs in greater than 60,000 people in the U.S. and around 250,000 worldwide.
About Intellia Therapeutics
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. The company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. Intellia’s ex vivo programs use CRISPR to engineer human cells outside the body for the treatment of cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.
Forward-Looking Statements
This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding: Intellia’s beliefs and expectations the safety, efficacy, success and advancement of NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (“AATD”)-associated lung disease pursuant to its clinical trial applications (“CTA”), including its ability to dose the first patient in its Phase 1/2 study in the second half of 2024 and the submission of regulatory applications in other countries; the ability of NTLA-3001 to durably normalize AAT protein levels after a single dose, halt the progression of lung disease associated with AATD, and improve patient outcomes; and the modularity of its gene insertion platform, including its ability to address numerous diseases caused by a missing or defective protein.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that clinical study results will not be positive. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
Intellia Contacts:
Investors:
Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
ian.karp@intelliatx.com
Lina Li
Senior Director, Investor Relations and Corporate Communications
lina.li@intelliatx.com
Media:
Matt Crenson
Ten Bridge Communications
media@intelliatx.com
mcrenson@tenbridgecommunications.com
FAQ
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