The company have together with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), consisting of Imperial College London and the Universities of Oxford and Edinburgh, Imperial Innovations, and Oxford BioMedica (OXB) announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).

The new partnership brings together the academic partners’ leading expertise in developing gene therapy for CF and OXB’s leading expertise in manufacturing lentiviral vector-based therapies with Boehringer Ingelheim’s capabilities in drug discovery and the clinical development of novel breakthrough therapeutic agents, reports the collaborators.

A possible universal treatment option

The collaboration will focus on a novel approach using a replication-deficient lentiviral vector in an inhaled formulation, to introduce a healthy copy of the CFTR gene into the cells of the lung. This method has demonstrated high gene transfer efficiency and offers the possibility of repeated administration to maintain the therapeutic effect. Gene therapy is the only therapeutic approach to date that can address all CFTR gene mutations, thus potentially offering a universal treatment option.

Three-way partnership

“This novel three-way partnership brings together an unparalleled combination of clinical, scientific, manufacturing and commercial skills in an effort to develop new treatments and make a major contribution to the lives of patients affected by cystic fibrosis,” stated John Dawson, Chief Executive Officer of Oxford BioMedica. “The GTC has been working determinedly for over 15 years to get to this exciting point of forming a partnership with Boehringer Ingelheim, a global pharmaceutical company with respiratory expertise. Our contribution to this partnership reaffirms our leading position in the development and manufacturing of lentiviral vector gene therapy products at large scale. We look forward to working with our new academic and industry partners.”

“Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward,” said Clive R. Wood, Ph.D., Senior Corporate Vice President, Discovery Research at Boehringer Ingelheim. “Bringing together our existing expertise as a leader for nearly a century in the discovery and development of therapies that have advanced patient care in respiratory diseases with the gene therapy knowledge of our partners, we aim to unlock unprecedented opportunities for patients with this devastating disease, who are desperately waiting for better treatment options.”

Collaboration details

Boehringer Ingelheim has received an option to license the exclusive global rights to develop, manufacture, register, and commercialise this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. Financial terms are not disclosed. During the option period the partners will work closely together to pursue the development of this innovative approach financed by Boehringer Ingelheim. The new collaboration is a joint initiative of Boehringer Ingelheim’s Respiratory Therapeutic Area and Research Beyond Borders (RBB), which is one of the pillars of Boehringer Ingelheim’s research and development strategy and explores emerging science, disease areas and technology. It builds on Boehringer Ingelheim’s commitment to early science and its comprehensive approach to respiratory research and development to investigate new treatments that have significant clinical value in areas of high unmet need.

Source: Nordic life science

ENPICOM finalized development and released the first version of their ImmunoGenomiX (IGX) platform to support the development, patient stratification and treatment monitoring of immunotherapies.

The adaptive immune system, also known as the acquired immune system, is a subsystem of the overall immune system that is composed of T and B lymphocytes, which eliminate pathogens or prevent their growth. Moreover, the clinical relevance of T cells in the control of different human cancers and autoimmune diseases is beyond doubt. Immunotherapies that boost the ability of T cells to destroy cancer cells have proven therapeutic efficacy in a variety of human malignancies.

ENPICOM develops a T cell and B cell receptor (TCR/BCR) repertoire immunosequencing data analysis solution. This IGX platform will be modularly expanded to become a comprehensive end-to-end immunosequencing data analysis platform designed to analyze, monitor, and compare the immune repertoires in the context of immunotherapy development and at all stages of treatment and disease over time. Starting from high-throughput sequencing data, it will deliver an easy-to-read report depending on the specific application, be it research, diagnosis, patient stratification, or treatment monitoring. The IGX platform will allow customers to use their own sample preparation protocols and the next-generation sequencing technology of choice. It requires no programming skills as the interface is intuitive and flexible.

The first version is available as of today and consists of the base module, IGX Explore, to analyze TCR repertoires from raw sequencing data, report back the number of individual TCR clones and interactively visualize them in a user-friendly way. With IGX Explore (TCR) immunotherapy companies can boost their target discovery, research and drug development processes. In case they need TCR repertoire analysis for a specific application or results visualized in a different way, ENPICOM can custom-build it.

Using a recently published and independent benchmark, ENPICOM showed that IGX Explore offers superior accuracy. The independent benchmark was published in a recent paper by Afzal et al., in which the authors benchmarked ten state-of-the-art TCR repertoire analysis tools. More information about this benchmark study can be found at the company’s website.

Dr. Nicola Bonzanni, Chief Scientific Officer, explains: “An important step in repertoire analysis is the correction of sequencing errors. If sequencing errors were left uncorrected, the richness of a sample would be vastly overestimated; correcting too many errors would lead to under-estimating richness. A critical challenge in immune repertoire analysis is to correct the sequencing errors in the data without losing repertoire-specific information in the process. IGX Explore uses an Adaptive Error Correction algorithm that learns sample-specific mutation rates from the data. By using sample-specific error estimations, erroneous sequences can then be corrected to provide accurate clone sequences, sizes, and frequencies” and adds “These results are very comforting, as highly accurate clonality analysis is the stepping stone towards successful application of this technology in the clinic at later stage”.

MDxHealth meldt de publicatie in The Journal of Urology van een studie die de kosteneffectiviteit van SelectMDx® voor prostaatkanker valideert. SelectMDx® is een niet-invasieve ‘vloeibare biopsie’ test die helpt bij het identificeren van patiënten met een verhoogd risico op agressieve prostaatkanker.

De studie toonde aan dat het opnemen van SelectMDx in het Amerikaanse gezondheidszorgsysteem kan resulteren in een gemiddelde volledige gezondheidswinst van 0,045 levensjaren, met een kostenbesparing van $1,694 per patiënt. Extrapolatie van deze gegevens naar een voorzichtige schatting van 311.879 patiënten per jaar die een biopsie ondergingen, resulteerde in 14.035 levensjaren in volledige gezondheidswinst met een jaarlijkse kostenbesparing van meer dan $500 miljoen voor ieder cohort.

De studie, ontworpen voor het verkrijgen van inzicht in de kosteneffectiviteit van SelectMDx in een populatie van Amerikaanse mannen met een verhoogd PSA-gehalte, laat een model zien met de impact van het gebruik van de niet-invasieve diagnostische test om mogelijk onnodige prostaatbiopsie uit te sparen. Het primaire doel was om veranderingen in gezondheidsresultaten in kaart te brengen, gemeten in voor kwaliteit gecorrigeerde levensjaren. De secundaire doelstelling was het evalueren van de zorgkosten vanuit het perspectief van de Medicare verzekeraar.

Dr. Matthew Resnick van de afdeling urologische chirurgie en gezondheidsbeleid van het Vanderbilt University Medical Center, Nashville, TN zegt: “Deze studie toont aan dat routinematig gebruik van SelectMDx als leidraad voor biopsiebeslissingen de gezondheidsresultaten verbetert en de kosten voor gezondheidszorg in verband met prostaatkanker-risicobeoordeling in een populatie van Amerikaanse mannen verlaagt. Wij zijn van mening dat deze strategie de waarde van conventionele risicobeoordelingsstrategieën vergroot door verbeteringen in zowel kwaliteit als kosten, de teller en noemer van de gezondheidswaarde-vergelijking.”

“Deze overtuigende studieresultaten zijn een verdere validatie van de positieve impact van SelectMDx voor het verlagen van de kosten voor de gezondheidszorg door ondersteuning in de begeleiding van behandelbeslissingen voorafgaand aan prostaatbiopsie,” zegt Dr. Jan Groen, Chief Executive Officer van MDxHealth. “Deze gezondheids- en economische waarde van vroegtijdige toepassing van SelectMDx tijdens het behandelingsproces, zullen onze onderhandelingen met Amerikaanse ziektekostenverzekeraars belangrijk ondersteunen.”

Dr. Groen vervolgt: “Dit is een belangrijke mijlpaal voor onze SelectMDx-test en versterkt onze strategie om verder voet aan de grond te krijgen in de markt voor eerstelijnszorg. Wij zijn van mening dat met deze uitbreiding de markt voor SelectMDx in de VS en Europa kan verviervoudigen tot meer dan twee miljoen patiënten per jaar”.

Arthrogen, a clinical stage gene therapy company together with the Centre for Human Drug Research (CHDR), announced that the first three arthritis patients have been enrolled in a phase Ib gene therapy trial for treatment of arthritis with ART-I02.

ART-I02 is an adeno-associated virus (AAV5) vector encoding the human IFN-β gene under control of an inflammation-responsive promoter. ART-I02 is designed to produce the anti-inflammatory protein IFNβ in the synovial cells in the joint. The aim is to achieve a sustained clinical remission with a single treatment. This current clinical phase Ib trial, evaluates the safety, tolerability, pharmacokinetics, immunogenicity and anti-inflammatory activity of ART-I02 treatment in patients with rheumatoid arthritis (RA) or osteoarthritis (OA). In total 12 patients with RA or OA in their hand joints will be included. Parallel to this study, a similar phase Ib trial has started in Canada treating 15 patients with RA in their wrist.

“Patients with rheumatoid arthritis and osteoarthritis face chronic pain and immobility, even despite the availability of new, mostly systemic treatments.”, explains principal investigator Prof. dr. Koos Burggraaf of CHDR and he continuous: “In this trial we evaluate the safety of ART-I02 and we assess effect measures, hoping that this first step may prove to be an important milestone marking a new era in the treatment of arthritis, a chronic debilitating disease.”.

Robert Jan Lamers, Chief Executive Officer of Arthrogen comments ‘We would like to thank our investors, collaborators and participating patients in helping us achieve this pivotal milestone. Based on anticipated enrollment and follow-up timelines, the first set of data is expected to be available in the fourth quarter of 2018’.

Source: Arthrogen