Last update 01 Nov 2024

rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

Last update 01 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Gene therapy

Synonyms

CFTR gene therapy(Boehringer-Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium/Imperial College London/University of Edinburgh/University of Oxford), lentiviral vector-based

Target

CFTR

Mechanism

CFTR stimulants(Cystic fibrosis transmembrane conductance regulator stimulants)

Therapeutic Areas

Congenital DisordersDigestive System DisordersRespiratory Diseases+ [1]

Active Indication

Cystic Fibrosis

Inactive Indication-

Originator Organization

Uk Cystic Fibrosis Gene Therapy Consortium

Active Organization

Imperial College London

Boehringer Ingelheim GmbH

University of Oxford

+ [2]

Inactive Organization-

Drug Highest PhaseDiscovery

First Approval Date-

Regulation-

100 Clinical Results associated with rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

100 Translational Medicine associated with rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

100 Patents (Medical) associated with rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

Literatures (Medical) associated with rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

01 May 2024·Regulatory Toxicology and Pharmacology

False positive findings associated with adenoviral vector-based vaccine underscore the regulatory necessity to eliminate abnormal toxicity test

Article

Author: Meng, Ryan ; Zhang, Yunhai ; Li, Yali ; Xie, Jianxun ; van Ooij, Mark

Accumulating evidence has shown that the abnormal toxicity test (ATT) is not suitable as a quality control batch release test for biologics and vaccines. The purpose of the current study was to explore the optimal ATT experimental design for an adenoviral vector-based vaccine product to avoid false positive results following the standard test conditions stipulated in the Pharmacopoeias. ATT were conducted in both mice and guinea pigs based on methods in Pharmacopeias, with modifications to assess effects of dose volume and amount of virus particles (VPs). The results showed intraperitoneal (IP) dosing at human relevant dose and volume (i.e., VPs), as required by pharmacopeia study design, resulted in false positive findings not associated with extraneous contaminants of a product. Considering many gene therapy products use adeno associated virus as the platform for transgene delivery, data from this study are highly relevant in providing convincing evidence to show the ATT is inappropriate as batch release test for biologics, vaccine and gene therapy products. In conclusion, ATT, which requires unnecessary animal usage and competes for resources which otherwise can be spent on innovative medicine research, should be deleted permanently as batch release test by regulatory authorities around the world.

01 Dec 2022·ThoraxQ1 · MEDICINE

Lung directed antibody gene transfer confers protection against SARS-CoV-2 infection

Q1 · MEDICINE

Article

Author: Viegas, Mariana A ; Meyer-Berg, Helena ; Zhao, Jincun ; Du, Yue ; Temperton, Nigel J ; Hyde, Stephen C ; Gamlen, Toby P E ; Dean, Rebecca ; Habib, Omar ; Gill, Deborah R ; Satyapertiwi, Dwiantari ; Wang, Yanqun ; Conway, Catriona C ; Miah, Kamran M

BackgroundThe COVID-19 pandemic continues to be a worldwide threat and effective antiviral drugs and vaccines are being developed in a joint global effort. However, some elderly and immune-compromised populations are unable to raise an effective immune response against traditional vaccines.AimsWe hypothesised that passive immunity engineered by the in vivo expression of anti-SARS-CoV-2 monoclonal antibodies (mAbs), an approach termed vectored-immunoprophylaxis (VIP), could offer sustained protection against COVID-19 in all populations irrespective of their immune status or age.MethodsWe developed three key reagents to evaluate VIP for SARS-CoV-2: (i) we engineered standard laboratory mice to express human ACE2 via rAAV9 in vivo gene transfer, to allow in vivo assessment of SARS-CoV-2 infection, (ii) to simplify in vivo challenge studies, we generated SARS-CoV-2 Spike protein pseudotyped lentiviral vectors as a simple mimic of authentic SARS-CoV-2 that could be used under standard laboratory containment conditions and (iii) we developed in vivo gene transfer vectors to express anti-SARS-CoV-2 mAbs.ConclusionsA single intranasal dose of rAAV9 or rSIV.F/HN vectors expressing anti-SARS-CoV-2 mAbs significantly reduced SARS-CoV-2 mimic infection in the lower respiratory tract of hACE2-expressing mice. If translated, the VIP approach could potentially offer a highly effective, long-term protection against COVID-19 for highly vulnerable populations; especially immune-deficient/senescent individuals, who fail to respond to conventional SARS-CoV-2 vaccines. The in vivo expression of multiple anti-SARS-CoV-2 mAbs could enhance protection and prevent rapid mutational escape.

01 Jan 2022·Cerebrovascular Diseases

Ischemic Stroke and Vaccine-Induced Immune Thrombotic Thrombocytopenia following COVID-19 Vaccine: A Case Report with Systematic Review of the Literature

Review

Author: Agostoni, Elio Clemente ; Giussani, Giuditta ; Cascio Rizzo, Angelo

Introduction: Vaccine-induced immune thrombotic thrombocytopenia (VITT) is a prothrombotic syndrome observed after adenoviral vector-based vaccines for severe acute respiratory syndrome coronavirus 2. It is characterized by thrombocytopenia, systemic activation of coagulation, extensive venous thrombosis, and anti-platelet factor 4 antibodies. Arterial thrombosis is less common and mainly affects the aorta, peripheral arteries, heart, and brain. Several cases of ischemic stroke have been reported in VITT, often associated with large vessel occlusion (LVO). Here, we describe a case of ischemic stroke with LVO after Ad26.COV2.S vaccine, then we systematically reviewed the published cases of ischemic stroke and VITT following COVID-19 vaccination. Methods: We describe a 58-year-old woman who developed a thrombotic thrombocytopenia syndrome with extensive splanchnic vein thrombosis and ischemic stroke due to right middle cerebral artery (MCA) occlusion, 13 days after receiving Ad26.COV2.S vaccination. Then, we performed a systematic review of the literature until December 3, 2021 using PubMed and EMBASE databases. The following keywords were used: (“COVID-19 vaccine”) AND (“stroke”), (“COVID-19 vaccine”) AND (“thrombotic thrombocytopenia”). We have selected all cases of ischemic stroke in VITT. Results: Our study included 24 patients. The majority of the patients were females (79.2%) and younger than 60 years of age (median age 45.5 years). Almost all patients (96%) received the first dose of an adenoviral vector-based vaccine. Ischemic stroke was the presenting symptom in 18 patients (75%). Splanchnic venous thrombosis was found in 10 patients, and cerebral venous thrombosis in 5 patients (21%). Most patients (87.5%) had an anterior circulation stroke, mainly involving MCA. Seventeen patients (71%) had an intracranial LVO. We found a high prevalence of large intraluminal thrombi (7 patients) and free-floating thrombus (3 patients) in extracranial vessels, such as the carotid artery, in the absence of underlying atherosclerotic disease. Acute reperfusion therapy was performed in 7 of the 17 patients with LVO (41%). One patient with a normal platelet count underwent intravenous thrombolysis with alteplase, while 6 patients underwent mechanical thrombectomy. A malignant infarct occurred in 9 patients and decompressive hemicraniectomy was performed in 7 patients. Five patients died (21%). Conclusion: Our study points out that, in addition to cerebral venous thrombosis, adenoviral vector-based vaccines also appear to have a cerebral arterial thrombotic risk, and clinicians should be aware that ischemic stroke with LVO, although rare, could represent a clinical presentation of VITT.

100 Deals associated with rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	Discovery	DE	Boehringer Ingelheim GmbH	-
Cystic Fibrosis	Discovery	GB	Imperial College London	-
Cystic Fibrosis	Discovery	GB	The University of Edinburgh	-
Cystic Fibrosis	Discovery	GB	Uk Cystic Fibrosis Gene Therapy Consortium	-
Cystic Fibrosis	Discovery	GB	University of Oxford	-

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rSIV.F/HN-CFTR(Uk Cystic Fibrosis Gene Therapy Consortium)

Overview

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Translational Medicine

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