On April 6, 2022 ViGeneron GmbH, a next-generation gene therapy company, reported a target-specific strategic collaboration and option agreement with Regeneron Pharmaceuticals Inc. (Regeneron) to develop and commercialize a gene therapy product based on ViGeneron’s novel engineered recombinant adeno-associated virus vectors (vgAAVs) to treat an inherited retinal disease (IRD) (Press release, Regeneron, APR 6, 2022, View Source [SID1234611498]).

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Under the terms of the research collaboration, Regeneron and ViGeneron will create and validate vgAAV-based therapeutic candidates for one undisclosed IRD target. ViGeneron receives an upfront payment and research funding. Regeneron has an option for an exclusive license to develop, commercialize and manufacture the vgAAV-based product for the specific target. ViGeneron is eligible to receive an option exercise fee, development and commercial milestone payments, plus royalties on net sales.

ViGeneron’s vgAAV vector platform is designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies. To date, therapeutically impactful targeting of photoreceptors relies on subretinal vector delivery, which harbors substantial risks of retinal detachment and collateral damage, often without achieving widespread photoreceptor transduction. vgAAV vectors could potentially enable the efficient transduction of target cells via intravitreal injection that allows lateral spreading and minimizes the risk of retinal detachment caused by conventional subretinal injection.

"We are delighted to work with Regeneron to potentially provide an intravitreally delivered gene therapy for patients suffering from an inherited eye disease," said Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron. "This agreement with Regeneron further validates the potential of our vgAAV platform, which is excellent for us and also delivers a deal value that contributes financing for our platform and proprietary program development activities. Furthermore, it fits into our strategy of developing proprietary programs for selected retinal targets through clinical trials, while maximizing our technology platforms for additional collaboration programs in retinal diseases, CNS and other disease areas with bellwether biopharma. Our aim is to overcome the current limitations of gene therapy and to bring a novel therapeutic approach to patients in need," she added.

On April 6, 2022 Servier reported that PROMETCO is the first real-world study conducted in oncology, in metastatic colorectal cancer (mCRC) (Press release, Servier, APR 6, 2022, View Source [SID1234611494]). A look back at the initiative, its first achievements and upcoming results.

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At Servier, patients are at the heart of every decision. This ambition is symbolized by PROMETCO, the first real-world study (see box) in oncology – and more specifically in metastatic colorectal cancer – conducted by Servier teams.

Developed in collaboration with the patient association Digestive Cancers Europe (DiCE), the PROMETCO real-world study will enable the Group to form a large cohort of patients, expressing the reality of metastatic colorectal cancer management from the point of view of the patient and the physician.

PROMETCO real-life study logo
First results…

In June 2021, initial results on the demographics and clinical characteristics of the first 277 patients enrolled in the study were presented at the European Society for Medical Oncology’s World Congress on Gastrointestinal Cancer (WCGIC) 2021. Differences in routine molecular testing practices across countries were revealed.

In January 2022, at the ASCO (Free ASCO Whitepaper)-GI (American Society of Clinical Oncology) conference on gastrointestinal cancer, Servier teams presented a poster on the subject. This poster presented the first data obtained from interim analyses and highlighted the heterogeneity and the complexity to care patients with metastatic colorectal cancer.

In addition, the PROMETCO teams published on a dedicated website an interactive visual of these first results, for the PROMETCO community of investigators.

… and publications

A publication in the scientific journal Future Onclogy describes PROMETCO as the first international real-world study to investigate the continuum of care for patients with mCRC. The article notes that the project will provide valuable information to the gastrointestinal medical community.

This publication was relayed digitally, using a variety of current communication media: podcast and poster.

The patient association Digestive Cancers Europe also published a communication underlining the involvement of patients in this study and the interest of this long-term collaboration.

PROMETCO in figures:
1,000 patients: recruitment target
125 research sites involved
18 countries involved

The next steps

The year 2022 marks the final stretch of patient recruitment for the PROMETCO study. Thanks to the ongoing efforts of the various research sites, more than half of the 1,000 patient recruitment target has already been reached.

A publication co-authored with patients highlighting the involvement of patients in real-world studies and what they can gain from them, as well as an abstract for ESMO (Free ESMO Whitepaper), are also planned for 2022.

Finally, the study report will be published by 2023, with the hope of improving future practices, particularly on the performance and personalization of treatments.

Understanding real-world studies

Improving medical knowledge
Real-world data are collected from a variety of sources and relate to the lived reality of patients and doctors, drawing on information such as the delivery of health care, treatments, as well as the impact of the disease and its treatment on patients’ lives. In other words, these data reflect the real lives of people affected by the illness in question and complement those derived from traditional clinical trials. Such additional robust analysis on the experience people have of living with diseases and medical treatment helps to expand therapeutic understanding.

A structured methodology for robust findings from clinical trials It is important to first understand the needs of all stakeholders in order to precisely identify the matters that require research, whether in relation to medical concerns, medical economics, or even regulation. Although it takes time to identify sources of data and deploy a suitable methodology, such steps are essential to obtain robust findings. Data are then analyzed using increasingly high performance digital platforms that enable the Group to compile ever larger data bases so as to exploit ever more homogeneous and standardized data.

To know more, read the annual report

This update is part of the #ECCAM2022 campaign (European Colorectal Cancer Awareness Month), also known as Blue March. Initiated by the patient organization Digestive Cancers Europe, this campaign aims to improve colorectal cancer screening programs, as well as to increase information and awareness about this disease.

On April 6, 2022 Catalent, the global leader in enabling biopharma, cell, gene, and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, reported the acquisition from Vaccine Manufacturing and Innovation Centre UK Limited (VMIC Ltd.) of a biologics development and manufacturing facility currently under construction near Oxford, U.K (Press release, Catalent, APR 6, 2022, https://www.catalent.com/catalent-news/catalent-acquires-facility-in-oxfordshire-to-expand-biologics-capabilities-in-the-uk-and-across-europe/ [SID1234611490]).

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Catalent plans to invest up to $160 million (£120 million) to complete the building of the facility and equip it with state-of-the-art capabilities for the development and manufacture of biologic therapies and vaccines, including mRNA, proteins, and other advanced modalities. It is expected that the new facility will employ more than 400 people and support public and private organizations seeking to develop and manufacture biotherapeutics.

"This acquisition allows Catalent to collaborate with the rich academic and biomedical science community centered around Oxford, with its world-class talent, and will result in a facility that provides opportunities to transform innovation into real treatments for patients across the United Kingdom, Europe, and beyond," commented Mike Riley, President of Catalent Biotherapeutics. "Our priority is to complete construction as soon as possible to be able to commence customer programs in 2022. We will then integrate its capabilities within our existing network of biologics facilities across Europe to offer a flexible range of manufacturing, technology, and development solutions for the pipeline of thousands of development programs currently underway."

"This deal ensures the VMIC site, when completed, will stay true to the original purpose of strengthening the U.K.’s vaccine manufacturing capability by bringing innovation to the sector and getting more vaccines to the clinic," said Professor Robin Shattock, Head of Mucosal Infection and Immunity at Imperial College London, and Chair of the Board of Directors of VMIC Ltd. "Catalent has the resources to ensure this facility can become operational and start contributing to the UK’s and Europe’s vaccines landscape at pace. I would like to recognize employees at VMIC for their passion and commitment during the pandemic and the success that their work brought to the U.K. In Catalent, we’ve found a new owner who shares the vision for what we wanted to achieve and has safeguarded the interests of the workforce."

The new facility is located to the south of Oxford at the world-renowned Harwell Science and Innovation Campus, a leading site in the U.K. for science and technology research breakthroughs and home to more than 40 life science companies.

Catalent has a long-standing presence in the U.K., where its workforce already exceeds 1,300 people at its four facilities focused on development and manufacturing of oral dose forms, located in Nottingham, Swindon, Haverhill, and Dartford, in addition to a clinical trial supply site in Bathgate, Scotland. The Catalent Biotherapeutics network also includes sites in Italy, Belgium, France, Switzerland, and the United States.

On April 6, 2022 Carina reported that has had five abstracts accepted for poster presentations at the AACR (Free AACR Whitepaper) Annual Meeting which will take place from April 8 to April 13 (Press release, Carina Biotech, APR 6, 2022, View Source [SID1234611488]).

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The poster presentations come after Carina’s recent submission of a pre-IND package to the FDA for Carina’s LGR5 CAR-T cell for the treatment of patients with advanced colorectal cancer.

HIGHLIGHTS

Five conference abstracts will be presented as poster presentations at AACR (Free AACR Whitepaper) in the immuno-oncology and preclinical immunotherapy sessions
Four presentations describe preclinical data from Carina’s LGR5-targeted CAR-T cell program, one poster will describe preclinical data from Carina’s ADAM-10 CAR-T program
With its LGR5 CAR-T, Carina is progressing towards a first-in-human clinical trial in patients with advanced colorectal cancer
Colorectal cancer is the third most commonly diagnosed cancer in Australia and in the United States, and the second leading cause of cancer death in Australia
Carina’s five presentations and their authors are:

Session OPO.CL06.01 – Immuno-oncology
(April 8, 2022, 12:00 PM – 1:00 PM CDT) Abstract online
5183 – LGR5 CAR-T cells: A novel potential treatment against high grade serous ovarian cancer authors B Wanqi Wang, Veronika Bandara, Noor Lokman, Silvana Napoli, Batjargal Gundsambuu, Martin Oehler, Simon Barry, Carmela Ricciardelli
Session OPO.IM02.01 – Preclinical immunotherapy
(April 8, 2022, 12:00 PM – 1:00 PM CDT) Abstract online
5575 – Pre-clinical validation of a LGR5-targeting CAR-T against colorectal cancer authors Veronika Bandara, Stuart Mills, Emma Thompson, Lih Tan, Batjargal Gundsambuu, Silvana Napoli, Jade Foeng, Dylan McPeake, Justin Coombs, Allison Cowin, Claudine Bonder, Timothy Sadlon, Shaun McColl, Simon Barry
Session OPO.IM02.01 – Preclinical immunotherapy
(April 8, 2022, 12:00 PM – 1:00 PM CDT) Abstract online
5574 – In vivo characterisation of a novel CAR-T cell therapy directed towards LGR5 for the treatment of colorectal cancer authors Dylan McPeake, Timona Tyllis, Jade Foeng, Veronika Bandara, Caitlin Abbott, Batjargal Gundsambuu, Elaheh Rohani-Rad, Silvana Napoli, Timothy Sadlon, Simon Barry, Shaun McColl
Session OPO.CL06.01 – Immuno-oncology
(April 8, 2022, 12:00 PM – 1:00 PM CDT) Abstract online
5184 – Real-time cytotoxicity assays as a pre-clinical screening tool for LGR5-targeting CAR-T cells for treatment of solid tumors authors Emma Thompson, Veronika Bandara, Timothy Sadlon, Batjargal Gundsambuu, Lih Yin Tan, Carmela Ricciardelli, Simon Barry, Claudine Bonder
Session OPO.IM02.01 – Preclinical immunotherapy
(April 8, 2022, 12:00 PM – 1:00 PM CDT) Abstract online
5505 – ADAM10-targeting CAR-T cells inhibit colon cancer cell growth in vivo authors Elaheh Rohani Rad, Jade Foeng, Dylan McPeake, Timona Tyllis, Caitlin Abbott, Veronika Bandara, Silvana Napoli, Batjargal Gundambuu, Timothy Sadlon, Simon Barry, Shaun McColl

About LGR5
LGR5 is a cancer stem cell marker that is highly expressed on advanced colorectal cancer and some other cancers. In colorectal cancer patients, LGR5+ expression has been correlated with a particularly poor prognosis.

Cancer stem cells are a small sub-population of cells within a tumour with the ability to self-renew, differentiate into the many cell types of a tumour, initiate new tumours, and resist chemotherapy and radiotherapy (leading to relapses).

By targeting cancer stem cells, it is hoped that this therapy will reduce the tumour’s ability to generate new cancer cells, resulting in durable tumour suppression and preventing the relapses that are very common in patients with colorectal cancer.

Carina’s pre-clinical studies of the LGR5-targeted CAR-T cell have shown highly promising results with complete tumour regression and no tumour recurrence. They have also demonstrated impressive tumour access and prolonged CAR-T cell survival.

On April 5, 2022 Asgard Therapeutics AB ("Asgard") reported that, in cooperation with the consortium members, Lund University, InSphero and Antineo, it has officially kicked-off the earlier announced Eurostars project (Press release, Asgard Therapeutics, APR 5, 2022, View Source [SID1234614857]). The project REPRogramming tumors INTo immune cells: a revolutionary gene therapy to treat cancer (REPRINT) will focus on providing definitive proof-of-principle for the use of the novel cancer immunotherapy TrojanDC developed by Asgard for the treatment of hard-to-treat solid tumors.

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Asgard, a private biotech company based in Lund, Sweden, is developing a paradigm-shifting cancer immunotherapy approach, based on its proprietary TrojanDC technology, to reinstate cancer immunogenicity. TrojanDC is a gene therapy that reprograms cancer cells into type-1 conventional dendritic cells, allowing the presentation of their own antigens to the immune system, thereby eliciting potent anti-cancer immune responses. In the context of this grant, Asgard collaborates with the partners to validate TrojanDC’s efficacy in ex vivo human spheroid models and 3D bioprinted models as well as in vivo rodent models. In vivo safety and pharmacology evaluation of TrojanDC will also be performed, bringing the gene therapy closer to IND-ready stage.

The Eurostars programme supports innovative international projects led by R&D-performing SMEs and is co-funded by the Eurostars partner states and the EU (through Horizon 2020). In October 2021, Asgard announced that together with three other partners it has been awarded the Eurostars project REPRINT (project number E!115376). The project started in 2021 October 1st with an online meeting but now the partners were able to meet in-person for an official kick-off event, held at Smile Incubator, Lund and hosted by Asgard. The three-year proposal was ranked #4 out of 644 European applications for Eurostars Cut off 15, and thereby secured a grant of 2 million euros.

Cristiana Pires, PhD, Co-Founder and Chief Executive Officer of Asgard Therapeutics, said: "The kick-off event was a great success! We had the chance to discuss the first results of the project and align on the next steps to use the Eurostars grant to further validate TrojanDC efficacy and safety. TrojanDC preclinical development will be accelerated within this grant allowing us to explore its game-changing properties. Designed as an off-the-shelf gene therapy, TrojanDC induces a personalized immune response, and thus overcomes many of the logistic and manufacturing hurdles of conventional cell-based therapies."

Filipe Pereira head of the Cell Reprogramming in Hematopoiesis Group of the LSCC commented: "The Pereira lab was excited to meet all partners of this multidisciplinary collaboration with academic and industrial partners. We are grateful to join forces paving the way for an entirely new cancer immunotherapy platform based on cellular reprogramming. By bringing our knowledge in ex-vivo and in-vivo dendritic cell reprogramming we will help advancing the pre-clinical development of an off-the-shelf gene therapy for cancer. We are keen to explore and unravel new possibilities of the cDC1 reprogramming approach developed at our lab!"

Wolfgang Moritz, Head of Ext. Collaborations and IP at InSphero AG commented: "InSphero is pleased to be part of this exciting project aiming at a ground-breaking new therapeutic approach for a variety of different cancer indications. During the meeting, we had an interesting discussion on our first steps in the project and coordinated the development activities with other consortium members. Our 3D in vitro technology will be used in the project to evaluate the clinical benefits, but also characterize the safety profile associated with the reprogramming process and induced immune responses. InSphero’s goal is to develop a new in vitro platform for simultaneous testing of on- and off- target effects of novel immuno-therapy strategies to cure cancer."

Elsa Kress, CBO of Antineo, said: "Antineo is thrilled to be involved in the preclinical development of such a game-changing therapy! It will be a great opportunity for us to develop ex vivo assays based on 3D-bioprinting tumoral and non tumoral cells. It will add a new, innovative and 3R-compliant are of preclinical services."

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In-person kick-off meeting of the Eurostars-funded REPRINT project. Top row from left to right: Ervin Aščić (Lund University), Hreinn Benónísson (Lund University), Michele Tavecchio (Asgard Therapeutics), André Rosa (Asgard Therapeutics). Middle row from left to right: Elsa Kress (Antineo), Irina Agarkova (Insphero), Michal Rudnik (Insphero), Olga Zimmermannova (Lund University), Nadezhda Rotankova (Insphero). Bottom row from left to right: Sanne Lock (Catalyze), Wolfgang Moritz (Insphero), Filipe Pereira (Lund University), Fritiöf Åkerström (Asgard Therapeutics), Cristiana Pires (Asgard Therapeutics), Malavika Nair (Lund University), Fábio Rosa (Asgard Therapeutics).