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Combigene in collaboration with Spark to bring gene therapy for epilepsy to patients

In a press release on the 12th of October, Combigene announced agreement with Spark (Roche Group) to develop and commercialize CG01 for treatment of drug-resistant epilepsy.

Merab Kokaia, scientific co-founder of Combigene. Photo: Ingmar Hultquist

 

Merab Kokaia, scientific co-founder of Combigene and director of Epilepsy Center comments:

The exclusive licence contract between Combigene and Spark is an important milestone in the gene therapy project to treat epilepsy. This contract guarantees financial support of upcoming clinical trial in hard-to-treat cases of epilepsy patients. If everything goes as planned, the clinical trial will be initiated in 2022 and the collaboration with Spark will be instrumental to perform the study in best possible way. As a basic scientist behind the preclinical data that were generated by me and my collaborators and as a co-founder of Combigene, I couldn’t be happier to be able to see the whole process through, from scientific discovery to clinical trials in patients who suffer from epilepsy that is not possible to treat with currently available options. I would like to believe that this step will open for new avenues not only in epilepsy treatment, but also for those other neurological conditions in a broader perspective that are not treatable today.

More reading in news articles from Dagens Industri and Biostock.

oktober 13, 2021

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New publication in Frontiers in Molecular NeuroScience

Development and Validation of CRISPR Activator Systems for Overexpression of CB1 Receptors in Neurons

For several years, approaches using viral vectors have been the focus of gene therapy research against neurological disorders. These are typically limited to the delivery of single genes and often do not manipulate the expression of the endogenous locus.

In the last years the advent of CRISPR-Cas9 technologies, allowing efficient manipulation of endogenous genes, have revolutionized many areas of scientific research. Although successfully used in neurons, they have not yet been extensively applied for the overexpression of genes involved in synaptic transmission.

In this study, Ledri et.al describe the development and application of two different CRISPRa systems, and demonstrate their efficiency in expressing the Cnr1 gene, coding for neuronal CB1 receptors. Both approaches were similarly efficient in primary neuronal cultures, while one approach was more efficient in vivo. 

The results show that CRISPRa techniques could be successfully used in neurons to target overexpression of genes involved in synaptic transmission, and potentially represent a next-generation gene therapy approach against neurological disorders.

Read the full article here

september 9, 2020

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Summer greetings

June means examination and strawberries. At Epilepsy Center three students defended their thesis; Diana Hatamian and Mohanad Hayatleh who have done their master projects under the supervision of My Andersson, and Kerstin Laurin who has been a bachelor student in Marco Ledri’s group. Congratulations to the three of you and best of luck in the future! We hope to be remembered by the gift, whenever you are having a cup of coffee.

Other news at Epilepsy Center is that we are now partner of two new consortia;

EpiEpiNet – Epileptogenesis and Epilepsy Network: from genes, synapses and circuits to pave the way for novel drugs and strategies. This is a program within Horizon 2020, coordinated by Instituto de Medicina Molecular Joao Lobo Antunes, Portugal.

and Epi-Cluster – which is a community network reinforced by EBRA (European Brain Research Area), with partners of EU-funded research projects and organisations with focus on epilepsy.

Summer greetings!

juni 21, 2020

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BMC A11 in times of Corona

Colourful creation of pipettes and hand disinfection stop at BMC, A11
Unleashed creativity (a colourful creation by Ulla Jarl, BMA at A11) and a hand disinfection post.

The corridors at BMC A11 seem somewhat tidier and a bit more deserted than usual, or at least compared to say February this year. With the pandemic ghost howling above us we are adapting to what has become the new usual.

The conference room is no longer used – instead we meet in digital rooms with our pictures patched together like live hive cells on the screen. Administrative personal are working from home but this is not a choice for everyone and in the laboratories people are still running experiments to keep their projects going.

At the joint coffee breaks we have developed a new integrity distance, the crowded kitchen during breakfast has been solved with premade sandwiches and at lunchtime people tend to eat in shifts. The professors (I’ve been told) are not utterly disappointed with this unpredicted pause in travelling although from a PhD student’s point of view, with conferences being cancelled, this means less opportunity to present your data and interact with other researchers outside the lab.

In the middle of all this, the main entrance closed down and the area in front of BMC has become a construction site. Rumours says that the reception will move to where the A10 kitchen is now located, pending the continuous construction of Forum Medicum.

As a positive twist and on behalf of everyone at Epilepsy Center I would like to welcome Ling Cao to A11. Ling will work as a research engineer within the groups of Merab, My and Marco.

Keep up the good spirit and take care! * air-high-five *

maj 20, 2020

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Dissertation behind closed doors

Today, Friday March 27, Esbjörn Melin defended his thesis, the last milestone of his research studies.

Advancing gene therapy for epilepsyTranslational pre-clinical studies with neuropeptide Y and glial cell-line derived neurotrophic factor.

Due to the corona pandemic the dissertation was a bit more special than usual with both the opponent David Henshall, one of the committee members and most of the audience participating over video link. There to support Esbjörn was his closest family, supervisors and a few colleagues and friends. Despite these conditions we were given a thorough introduction on epilepsy and gene therapy and a fantastic presentation without any technical problems. Unfortunately, to keep a stable link with the opponent during the discussion the second link was cut and the audience following the defense from home or from an office missed out on the last part.

When the committee, chaired by Cecilia Lundberg, announced that Esbjörn had passed the exam, the opponent wanted to share an Irish tradition – and quite unexpectedly Esbjörn’s father magically pulled out a glass and a Guinness from a handbag so that Esbjörn could join David in a toast.  A very nice gesture, and a special moment! 

When everyone was gathering outside the auditorium to congratulate Esbjörn, his supervisor Merab Kokaia revealed the secret that all these years Esbjörn was actually part of a single blinded experiment with the hypothesis that a technical student from LTH could be successful in the medical field. And for sure, he concluded, this was right!

We salut you Doctor Melin, and wish you the best of luck in the future! 

mars 27, 2020

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Nailing – Esbjörn Melin

Today, Friday February 28, Esbjörn Melin put the symbolic nail through his thesis; Advancing gene therapy for epilepsy – Translational pre-clinical studies with neuropeptide Y and glial cell-line derived neurotrophic factor.

From the beginning the primary purpose of nailing was to make the text available to people, giving everyone the opportunity to read it in advance to be able to ask relevant questions at the public defence. This tradition goes back to when Martin Luther nailed his 95 theses to the door of Wittenberg Castle church in 1517. Now a days the nailing is still a mandatory component of the academia, at least 3 weeks before the defence the thesis has to be electronically nailed and distributed. Printed copies are available for everyone to read but the ceremony of nailing it to the wall (or the floor) at your departement is still a nice excuse to celebrate.

februari 28, 2020

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Världsepilepsidagen 2020

Monday, February 10 was the International Epilepsy Day. From Epilepsy Center a group of researches took the opportunity to meet with the public, first at the hospital in Kristianstad and later in the afternoon at Lunds stadsbibliotek. The event in Lund was organised by My Andersson, PI at Epilepsy Center, together with Lars A Ohlsson, Epilepsiföreningen Lund.

At the library My Andersson and colleagues presented some of the on-going research at Epilepsy Center, discussing future treatments like gene therapy, and the importance of testing on human tissue to evaluate the clinical potential of different factors. Christine Ekdahl-Clementson, researcher and clinician, presented the results from a cohort study suggesting that physical activity could prevent the development of epilepsy. Another speaker showed that seizures could induce inflammation in the eye, raising the question whether this might have an effect on the vision.

Lars Ohlsson talked warmheartedly about Epilepsiföreningen as an organisation where you can turn for support with practicalities and questions and emphasised that the most important part of their work is to organise social activities where people living with epilepsy, as a patient or as a relative, can meet. Lars also shared his personal story, describing the feeling of being the only parents with a child with epilepsy, when in fact once we talk about it; there are so many sharing the same experience.

To the speakers and the audience, Thank you!

Program Världsepilepsidagen Lund, 2020

februari 14, 2020

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European Union Horizon2020: Training4CRM ITN. Cell-based therapies for neurodegenerative diseases.

We are very proud to present this video produced by the PhD students at Training4CRM Research School, Barcelona, in October this year. Our compliments to Ana and Eliska at Epilepsy center, and to all the others!

Horizon 2020 is the funding tool for research and development of the European Union, and one of the largest funding organization of research in the world. The Training4CRM project is a Horizon 2020-funded initial training network (ITN), which runs from 2016 to 2020. In this project, 15 PhD students and their supervisors are working at 9 academic or industrial organizations, as well as associated organizations, to develop new treatment options for Neurodegenerative Diseases, such as Parkinsons, Alzheimers or Huntingtons Disease. This research is an interception between the fields of regenerative medicine, cell biology, optogenetics, electronics and nanotechnology. This Video gives an overview of our work and the purpose of this project. For more information, please visit our website: http://training4crm.com/

december 20, 2019

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Physical Activity Reduces Epilepsy Incidence

An illustration by Linnéa Haviland showing a collage of a face, the brain and a cross-country skier.

 

This week, the results from a retrospective cohort study lead by Christine Ekdahl Clementson, Epilepsy Center, Lund University, was published in Sports Medicine Open. The incidence of epilepsy was investigated during 20 years in a cohort of 200 000 participants from the long-distance Swedish cross-country ski race Vasaloppet and the results show that it was reduced to close to 50% compared to the control group.

These results together with experimental findings in epilepsy-prone knockout mice indicate that physical activity may prevent or delay the development of epilepsy.

 

In Swedish at lu.se

Read the article by Ahl et. al here

december 19, 2019

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New publication in Molecular Therapy – Methods & Clinical Development

Gene therapy has been suggested as a plausible novel approach to achieve seizure control in patients with focal epilepsy that do not adequately respond to pharmacological treatment.

In this study, Melin et.al have investigated the seizure-suppressant potential of combinatorial neuropeptide Y and Y2 receptor single vector gene therapy, first in a dose-response study, and second in an efficacy study.

The latter demonstrated a favorable outcome of the gene therapy, with a 31% responder rate and 13% seizure-freedom rate. In addition, the seizure duration was significantly reduced and the intervals between the seizures were prolonged in the treated group compared to the controls.

This study establishes the suppressant ability of the single vector combinatorial neuropeptide Y/Y2 receptor gene therapy in a clinically relevant chronic model of epilepsy.

read the full article hear

december 6, 2019

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