GenE-HumDi Second Period in perspective

Two years ago, COST Action CA21113 was given green light and we started this adventure of seeking for members willing to help us build an European network aiming to make Genome Editing for the treatment of Human Disease a reality.

We have now reach half of the action’s lifespan and is a great moment to reflect on the achievements and contribution of the academic and industry researchers, Advance Medicine technicians and engineers, Healthcare professionals, Scientific Divulgators, Regulatory and patient advocacy groups.

We are very proud of the hard work of volunteers, and the commitment of young talent to help GeneHumdi initiatives.

Today we wanted to compile some of the recent achievements in this post entry, to pay tribute to the good work of the volunteer members. During this 2 years we had to rethink our structure and embrace in a single WG the interest of industry a regulatory actors, leaving the action with the following structure:

  • WG2 – Improvement of GE technology
  • WG3 – Delivery Strategies
  • WG4 – Safety issues: Monitoring and standardization
  • WG5 – Translation into the clinic
  • WG6 – Technology transfer and industry / Regulatory issues
  • WG7 – Dissemination

This structure is helping both advancing within the focused scope of the WG and also nurturing collaborative works that materialized the following COST Action achievements:

Genome Editing tools Reference Document
GeneHumdi consensual hand-book on cells types where further improvements are required to achieve efficient and specific GE.

This has been expanded to the realization of actual Training Schools to instruct younger scientist and future industry leaders into the use of genome editing tools

Standardized procedure protocols for the determination of off-target for experimental use in preclinical and clinical studies

Fruit of interactions between members from different Working Group members, GenE-HumDi COST action is nourishing collaborative works, and the Scientific articles produced by Members are Proof:

Summary of the SEcond Period General Meeting held in Limassol , Cypryus

During our second general meeting we engaged EU genome editing leaders in Limassol in a collaborative meeting . This Meeting was instrumental for the materialization of collaborations that are yielding scientific revision on genome editing and to promote the capabilities of young researchers in our network.

Guidelines and reflections concerning regulatory aspects of new gene editing tools

Technology transfer, industry and regulatory issues working group, leaded by Carsten Lederer, have recently shared a great reference document elaborated by WG6 members. The Full text is openly available.

Strategic Dissemination Plan

Originally drafted by the action SCC, Fco. Javier Molina, and former WG7 leader, Luís Montoliu. And approved by MC members, the plan describes the methods and protocols for the liaison between networks members, working groups and external stakeholders.

Booklet summarizing all the main conference of each WG during the half-life of the action

This a great moment in the Actions life to reckon on the progress of the network activity. We have elaborated a proceeding booklet summarizing all the main conference of each WG during the half life of the action. This detailed summary of the WG records is helping us to learn from the many successes achieved and identify areas that will need further attention in the upcoming years of the network

Summary of state of the art concerns and opportunities for Genome Editing in the Medical Field.

During our Kick-off Meeting we manage to gather European leaders. Our call is still open to all different gears involved in the research, translation, experimental medical application, medical approval and healthcare access and patient advocacy. But our scope is fixed in making Genome editing technologies an actual medical option.

FINAL PROGRAM

Updated on 2024.10.09
Dr. Karim Benabdellah
Dr. Fco Javier Molina Estevez
GHM: Raquel Soriano e mail: raquel.soriano@juntadeandalucia.es

Coupling Genome Editing Tools with Delivery Systems

Genome editing tools and their delivery systems are advancing quickly, but they rely on each other to improve genome editing methods. This meeting will showcase the latest developments in both areas and foster connections and collaborations among scientists to boost the effectiveness of their research projects.

This is open event organized by Genome Editing for the treatment of Human Disease ( GeneHumdi| COST action 21113). A network committed to foster therapeutic genome editing therapeutic by nourishing collaborative networks and promoting early career scientist and industry engagement with the field. In this Workshop we will:

  • 1. Identify and evaluate the existing endonuclease-independent platforms (EIP) and their potential uses in various applications.
  • 2. Identify and evaluate the existing endonuclease-dependent platforms and their potential uses in various applications.
  • 3. Create a comprehensive overview of the current delivery methods used for genome editing (GE) in various cell types for clinical applications.
  • 4. Create a comprehensive overview of the current delivery methods used for in vivo genome editing (GE) in different animal models and clinical environments

This workshop is made possible thanks to a collaboration of GenE-Humdi and IDIBELL. We are thrilled to announce that several field leaders are now confirmed speakers:

  • Dr. Marc Güell (Pompeu Fabra University, Spain)
  • Dr. Vittoria. Raffa (Associate Professor in Molecular Biology, Ph.D. in Nanotechnology, M.Sc. in Chemical Engineering, Italy)
  • Dr.  Giedrius Gasiunas (Caszyme, Lithuania)
  • Dr. Peng Ling (Aix-Marseille Université, CNRS, Centre Interdisciplinaire de Nanoscience de Marseille, France)
  • Dr. Julian Cerón Madrigal (Institut d’Investigació Biomèdica de Bellvitge, Spain)
  • Dr. Karen O´Hanlon Cohrt (CRISPR Medicine News, Denmark)
  • Dr. Alessia Cavazza (UCL, UK)
  • Dr. Fco Javier Molina Estévez (GENyO, Spain)
  • Dr. Laura Batlle Morera (CRG, Barcelona)
  • Dr. Benabdellah Karim (GENyO, Spain)
  • Dr. Gloria Gonzalez Aseguinolaza (DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Spain).
  • Dr. Cecilia Jiménez-Mallebrera (Neuromuscular Unit. Institut de Recerca Sant Joan de Deu. Hospital Sant Joan de Déu Barcelona, Spain).
  • Dr. Mariana Köber (Biomedical Research Network on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Spain)
  • Dr. Carles J. Ciudad (Universitat de Barcelona, Spain)
  • Dr. Jan Gorodkin (Department of Veterinary and Animal Sciences, Center for non-coding RNA in Technology and Health, University of Copenhagen, Denmark.).

Venue:
Residencia de Investigadores del CSIC

Carrer de l’Hospital, 64, Ciutat Vella, 08001 Barcelona


Two Days Packed with Science:
Thursday October 3rd ( 2:00 pm to 8 pm ) and Friday October 4th (9:00 am to 15 pm), 2024.

Free attendance:
Registration is needed as space is limited to maximize networking. Signature control will be required and assistance certificates will be available upon request.

To register as attendee or presenter, fill the form and send it to GeneHumdi Grant Holder Manager (raquel.soriano@juntadeandalucia.es)Before September 15th!

FINAL PROGRAM

Organizing committee and contacts:
Dr. Julian Cerón Madrigal
Dr. Francisco Martin Molina
Dr. Karim Benabdellah
Dr. Fco Javier Molina Estevez
GHM: Raquel Soriano e mail: raquel.soriano@juntadeandalucia.es

GeneHumdi-Aarhus University 2024 Course #ClinicalApplications

GeneHumdi chair Karim Benabdellah has confirmed the participation of the COST Action CA21113 in the incoming training school “Clinical Applications of In Vivo Gene Editing: Methods, Challenges, and Solutions” in collaboration with the Department
of Biomedicine, Aarhus University. It will be held 17th -20th September in Aarhus, Denmark
.

The course will be locally organized by esteemed Dr. Yonglun Luo, and  Dr. Karim Benabdellah, and will be coordinated by the European COST action “Genome Editing to Treat Human Diseases” (GenE-Humdi; action CA21113), an EU-funded network that connects researchers and innovators across Europe and beyond.

In this course, we investigate into the complex landscape of gene editing within a clinical framework, focusing on the generation of a comprehensive map detailing current delivery methodologies employed for in vivo gene editing across various animal models and clinical scenarios, with some insight for ex vivo studies. The course includes a meticulous comparison of efficacy and specificity data, elucidating the diverse array of gene editing tools administered through different delivery methods within specific tissues and organs of interest

Main Objectives:

3. To explore LNP (Lipid Nanoparticle) systems: Attendees will delve into the principles and applications of LNPs in gene editing, particularly their use in delivering mRNA and other gene editing tools

4. To understand the application of LVs (Lentiviral Vectors): The program will provide insights into the use of lentiviral vectors for gene editing.

5. case studies and practical examples of their use in treating different pathologies such as, cancer (Immunotherapy) Neuromuscular Disorders (NMDs), neurological disorders, and Retinal, Pancreatic, and Cardiac Diseases.
Check the full program bellow:

For more info on the Summer School Applications and COST support options, follow us GeneHumdi on the LinkedIn GeneHumdi Page, Twitter @genehumdi or our web updates at www.genehumdi.eu

GenEHumDi 2024 ITC/NCC Second Call

COST Action CA20113 Encourage the participation of new Inclusiveness Target Countries (ITCs) and Near Neighbour Countries (NNCs) in International Meetings and Conferences.

COST Action CA21113, Genome editing for the treatment of human disease: Gene-Humdi, is opening the 2024 call for Inclusive Target Countries and Near Neighbour Countries. We provide funding opportunities to ITC and NCC members attending to Conferences and Meetings aligned with mission of the GeneHumdi Network. You can prepare by learning more about our mission at www.genehumdi.eu or looking for a suitable mentor among our amazing members listed HERE.

2024.08.06 UPDATE

We are funding ESGCT presenting students!!

⚠Be aware of 2024 policies ⚠


Please refer to the guidelines for more info.:

2nd 2024 GeneHumdi Call for Short-Term Scientific Missions (STSMs)

This is the 2nd Call for applications for Short-Term Scientific Missions (STSMs) funded by the COST Action CA21113 “Genome Editing to Treat Humans Diseases” (GenE-Humdi) CA21113.

Why do we encourage Short-Term Scientific Missions (STSM) within the framework of the GeneHumdi COST Action?

The board of GeneHumdi is pleased to announce the availability of Short-Term Scientific Missions (STSMs) through the COST Action program and encourages researchers to apply. These missions offer researchers the opportunity to conduct short visits to research institutions or laboratories in other COST countries, with the aim of strengthening existing networks and fostering collaboration. STSMs also provide researchers with the chance to learn new techniques or use equipment, data, and/or methods that may not be available in their own institution, while contributing to the scientific objectives of the Action. We strongly urge interested researchers to take advantage of this unique opportunity to expand their scientific knowledge and contribute to the advancement of their respective fields.

Participants in the COST Action program’s Short Term Scientific Missions (STSMs) have a great opportunity to focus their work on research topics that have been highlighted by GenE-Humdi’s 8 Working Groups (WGs) or to introduce new ideas that address the objectives of the project. We encourage researchers to take advantage of this opportunity to enhance their scientific knowledge and contribute to the advancement of their respective fields. (Details on the content and goals of WGs is available in the Action’s Memorandum of Understanding (MoU).

Guidelines for applicants:
  • Individuals are supported financially with travel grants for four Short Term Scientific Missions (STSMs) offered by GenE-Humdi. Grantees are responsible for making their own arrangements for travel, accommodation, and other logistics.
  • STSMs must be completed within the 2nd Grant Period of the Action, which ends in October 2024. While there is no maximum duration for visits, the minimum visit should be at least 5 days, including travel time.
  • The deadline for submitting applications is August 24th, 2024. Notification of decisions will be made by September 9th, 2024.
  • During this period, STSM grants are awarded up to a maximum of 4,000€. Applicants are encouraged to review the Annotated Rules for COST Actions.
  • The STSM travel grants are open to researchers and innovators who are associated with a legal entity in a COST Full/Cooperative Member, Near Neighbour Country, or European RTD. While all eligible candidates are encouraged to apply, young researchers and innovators under the age of 40 are particularly encouraged to participate.
Application instructions:
  1. To apply for an STSM, applicants must utilize the e-COST management tool. It is mandatory for all applicants to possess an e-COST profile.
  2. To apply for an STSM, the applicant must utilize the e-COST management tool to submit their request. Along with the necessary information provided on the website, applicants are required to upload specific documents in a single PDF file:
  • A brief CV that highlights recent publications.
  • The STSM proposal outlining the research objectives and methodology.
  • The requested budget in euros.
  • A confirmation letter from the host institution.
  • Applicants who require further information on the submission process via the e-COST system can refer to the Grant Awarding User Guide.
  • After completing the STSM, the recipient must submit a brief report outlining the results of their visit. The report must be submitted no later than 30 days following the end date of the STSM, or 15 days after the end of the Grant Period, whichever comes first. It is important to note that if the report is not submitted on time, the grant will be revoked. Once the report has been approved, the grant payment is expected to be processed within 30 days.
Evaluation of applications:

The GeneHumdi Grant Awarding committee will assess each proposal individually and provide an evaluation score based on several factors. These factors include:

  1. The clarity of the proposal.
  2. The degree to which the proposed STSM complements or contributes to the strategic priorities and objectives of the Action as defined in the MoU.
  3. The feasibility of the planned work plan and outputs, and the ability of the STSM applicant to successfully complete the proposed STSM and disseminate relevant outputs.

It is important that the proposal clearly demonstrates how the proposed STSM will benefit both the applicant and the Action.

The proposal will be categorized based on its evaluation score, within 5 categories:

  1. Poor: (0-25): The proposal will be considered poor if it is unsound, incomprehensive, and lacks clear links to any of the WGs
  2. Fair: (26-50): A proposal with limited understanding, unclear objectives, and weak linkage to at least one Working Group
  3. Good: (51-75): A proposal is one that is well-linked to at least one WG and needs input to develop feasible STSM
  4. Excellent: (76-100): well-thought-out plan with clear feasibility and expected outcomes. It is highly relevant to at least one of the WGs

The top four proposals with the highest scores will be awarded the STSM grants.

Topics

Funded STSM must align with at least one (or more) of the following topics:

  • Topic 1: Mapping the existing endonuclease-Independ platforms (EIP) and their potential output in different applications, efficacy, specificity of the available endonucleases independent GE tools.
  • Topic 2: Mapping the existing endonuclease platforms (EIP) and their potential output in different applications, efficacy, specificity of the available endonucleases independent GE tools.
  • Topic 3: Delivery methods used for GE of the different cell types that are used for clinical applications. non-viral systems, based on ribonucleoparticles (RNP) or plasmids, are the ex vivo systems of choice to deliver the GE complex into the target cells.
  • Topic 4: Delivery methods used for in vivo GE in different animal models and in clinical settings. Including comparison of efficacy and specificity data for different GE tools delivered by different methods in each tissue/organ of interest.
  • Topic 5: The establishment of a standardized pipeline for the unbiased identification and quantification of CRISPR off-target sites for therapeutic applications.
  • Topic 6: Assessment of recombination events in gene editing approaches
  • Topic 7: Comparison of the different constrains in the HDR-mediated gene editing in the most primitive HSPCs, and how the HDR may be enhanced.
  • Topic 8: The evaluation of different GE strategies to target Hematopoietic stem cells in Fanconi anaemia cells in vitro, the sickle cell disease and beta-thalassemia, collagen VI deficiency and Emery Derfuss muscular dystrophy.
  • Topic 9: Market research study and competitive analysis on gene editing based medicines including the following tasks: (i) Market opportunity. Target niche; (ii) Definition of targeted market, sales by category (product, territory, class and market share (sales peak, defining at least three different scenarios), positioning, market drivers and barriers.
  • Topic 10: The analyse of regulatory requirements for the commercialization of gene editing based medicines.
Question and inquiries:

Please contact GeneHumdi:

Webinar series 2024

🚀 GenE-HumDi News! 🚀

Are you a young researcher looking to expand your knowledge and network? Look no further! We’re thrilled to announce the launch of our new webinar series organized by young researchers, for young researchers.

🔬 Each session will feature an invited speaker sharing insights on cutting-edge topics in science and career development. Join us for an hour of inspiration, learning, and networking.

🗓️ Mark your calendars! Our next session is on June 25th at 3 pm CET. Expert, Paula Rio, will discuss “CRISPR Clinical Trials: current progress and future perspectives in in vivo approaches.”

💬 Don’t miss out on the chance to engage with our speakers! Each session includes a Q&A, and for those interested in diving deeper, we offer one-on-one “Meet the Expert” sessions where you can discuss science and career development. We have limited spots available, so reply to this email if you’d like to be selected for the meet-the-expert session.

📝 We’ll be back to you soon with the webinar link, so stay tuned and mark the date on your agenda!

🤙Spread the word to your young researcher peers. If you know someone who would be interested, encourage them to join us for our webinar series!

Paula Río, Biologist, PhD (UCM) is the head of Bone Marrow Failure Unit at CIEMAT/CIBERER/IIS-FJD, UAM; Madrid, Spain. She has been working in the field of gene therapy of Fanconi anaemia (FA) for more than 20 years, where she has focused on the design of a lentiviral vector for the correction of haematopoietic stem cells in patients with FA and its application in the clinic, with very promising results for patients. She is currently an associate researcher in two gene therapy clinical trials based on the pre-clinical studies developed by the group.

Since 2010 her work has focused on the search for new strategies to correct haematopoietic stem cells from FA patients by gene editing, a field in which she is coordinator of a European project and participates as principal investigator in other national and European projects.

She has published more than 55 papers in the field of gene therapy. She is elected president of the Spanish Society of Gene and Cell Therapy and member of several scientific advisory committees, including the American Fanconi Anemia Research Foundation, the Gene Editing committees of the American Society for Gene Therapy and European Society for Gene and Cell Therapy and the Emerging Gene and Cell Therapies subcommittee from the American Society of Hematology.

We are happy to announce that CRISPR NEWS MEDICINE has offered to help us organizing the first webinars.
So, please register online to attend this free webinar at:
https://crisprmedicinenews.com/cmn-webinars/ghumdi-strategies-and-tools-for-ex-vivo-and-in-vivo-genome-editing/

Past Webinars 2024

🚀 GenE-HumDi News! 🚀

watch again our June 11th, 20204 Webinar “CRISPR Clinical Trials: current progress and future perspectives in ex vivo approaches.”

Are you a young researcher looking to expand your knowledge and network? Look no further! We’re thrilled to announce the launch of our new webinar series organized by young researchers, for young researchers.

🔬 Each session will feature an invited speaker sharing insights on cutting-edge topics in science and career development. Join us for an hour of inspiration, learning, and networking.

🗓️ Mark your calendars! Our inaugural session is on June 11th at 3 pm CET. Our first expert, Annarita Miccio, will discuss “CRISPR Clinical Trials: current progress and future perspectives in ex vivo approaches.”

💬 Don’t miss out on the chance to engage with our speakers! Each session includes a Q&A, and for those interested in diving deeper, we offer one-on-one “Meet the Expert” sessions where you can discuss science and career development. We have limited spots available, so reply to this email if you’d like to be selected for the meet-the-expert session.

📝 We’ll be back to you soon with the webinar link, so stay tuned and mark the date on your agenda!

🤙Spread the word to your young researcher peers. If you know someone who would be interested, encourage them to join us for our webinar series!

Prof. Annarita Miccio, Lab Director of the Laboratory of Chromatin and Gene Regulation during Development at the Imagine Institute in Paris will discuss “CRISPR Clinical Trials: current progress and future perspectives in ex vivo approaches” past June 11th ,at 3:00 pm CET.
Stay tuned for the CRISPR MEDICINE NEWS link to secure your seat and chat with field experts about the present and future of therapeutic genome editing.

Did you miss Annarita Miccio Webinar?
You can Watch it on Demand thanks to CRISPR NEWS MEDICINE.
Follow this link and watch again our June 11th, 20204 Webinar “CRISPR Clinical Trials: current progress and future perspectives in ex vivo approaches.”

🚀 GenE-HumDi News! 🚀

Are you a young researcher looking to expand your knowledge and network? Look no further! We’re thrilled to announce the launch of our new webinar series organized by young researchers, for young researchers.

🔬 Each session will feature an invited speaker sharing insights on cutting-edge topics in science and career development. Join us for an hour of inspiration, learning, and networking.

🗓️ Mark your calendars! Our inaugural session is on May 27th at 10 am CET. Our first expert, Marc Güell, will discuss “Strategies and tools for ex vivo and in vivo genome editing.”

💬 Don’t miss out on the chance to engage with our speakers! Each session includes a Q&A, and for those interested in diving deeper, we offer one-on-one “Meet the Expert” sessions where you can discuss science and career development. We have limited spots available, so reply to this email if you’d like to be selected for the meet-the-expert session.

📝 We’ll be back to you soon with the webinar link, so stay tuned and mark the date on your agenda!

🤙Spread the word to your young researcher peers. If you know someone who would be interested, encourage them to join us for our webinar series!

WG3 , delivery strategies webinar

Rosario M Sanchez Martin from Genyo center (University of Granada, Granada, Spain) will introduce us to the world of nanoparticles and their potential applications to  HSCs. This will be followed by an open discussion.:

Nanoparticles based delivery of gene editing tools in HSCs

Day: February 29th

Time: 10:30 CET

Chair: Annarita Miccio

Connect to the meeting online:

No longer available

GeneHumdi #SummerCourse2024

GeneHumdi chair Karim Benabdellah has confirmed the.. participation of the COST Action CA21113 in the incoming training school “Comprehensive Understanding of the latest advancements in gene editing delivery methods” in collaboration with the National Institute of chemistry. It will be held this summer June 26-28 in Ljubljana, Slovenia.

The course will be locally organized by esteemed Dr. Duško Lainšček and Prof Roman Jerala from the National Chemistry Institut (Ljubljana, Slovenia). They will count with the collaboration of Dr. Dhano Gupta from Institute of Developmental & Regenerative Medicine, Oxford university (Oxford, UK).

The course orchestrates around dissecting efficacy and specificity of cutting-edge genome editing technologies considering the major layers that are needed for the success of clinical genome editing interventions. The syllabus articulates around 3 blocks:

Evaluating efficacy and specificity of various genome editing tools. And including the comparison of different delivery methods for optimal outcomes.

Investigating in vivo genome editing techniques in animal models and clinical scenarios. And assessing efficacy and specificity within specific tissues and organs.
Check the full program bellow:

From the Genome Editing for the treatment of human diseases (GeneHumdi, COST CA21113), we are happy to announce that we count with the support and expertise of many members interested and the participation of the action chair Karim Benabdel Lah and SCC F. Javier Molina as coordinators.

AUGUST 2024 UPDATE.
Review the Course contents and contributions in the following document:

For more info on the Summer School Applications and COST support options, follow us GeneHumdi on the LinkedIn GeneHumdi Page, Twitter @genehumdi or our web updates at www.genehumdi.eu

#GeneHumdi2024ITC

COST Action 20113 Encourage the participation of new Inclusiveness Target Countries (ITCs) and Near Neighbour Countries (NNCs) in Action activities.


COST Action CA21113, Genome editing for the treatment of human disease: Gene-Humdi, is opening the 2024 call for Inclusive Target Countries and Near Neighbour Countries. We provide funding opportunities to ITC and NCC members attending to Conferences and Meetings aligned with mission of the GeneHumdi Network. You can prepare by learning more about our mission at www.genehumdi.eu or looking for a suitable mentor among our amazing members listed HERE.

2024.03.05 UPDATE

As now the first call for ICT/NCC applicants is Open!!

⚠Be aware of 2024 policies ⚠


Please refer to the guidelines for more info.:
https://drive.google.com/file/d/1eaQHq96aC9BjnKhoadYz24sNRbgvTdlV/view?usp=drive_link

#GeneHumdi2024STMS

Do you want to give a boost to your Genome Editing project abroad?
Are you an enthusiastic student/young investigator from an EU member country?
Would you like to embrace an international experience supervised by a top GenEd scientist?


COST Action CA21113, Genome editing for the treatment of human disease: Gene-Humdi, is opening the 2024 call for Short Term Scientific Missions Soon. We provide funding opportunities for short stays in laboratory across EU. This year we also feature support for Inclusiveness Target Countries and or Near Neighbor Countries. You can prepare by learning more about our mission at www.genehumdi.eu or looking for a suitable mentor among our amazing members listed HERE.

2024.03.05 UPDATE

As now the first call for STMS applicants is Open!!

STSM stands for Shor Term Scientific Mission, which means we want you to live the most exciting scientific experience of your carrer so far.

STMS offer the possibility to grow as a scientist, experience and learn excellence in research by the hand of any of our outstanding members. Polish your communicative abilities and contribute to the advance of our scientific community.

STMS will ask from you to present a thorough proposal, to explain the research that you perform, to become a relevant member contributing to the mission of our COST Action CA23113 network.


Please refer to the guidelines for more info.:
https://drive.google.com/file/d/1eaQHq96aC9BjnKhoadYz24sNRbgvTdlV/view?usp=drive_link

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