Founded in 2020, based on 10+ years of research at the Swiss Federal Institute of Technology EPFL Brain Mind Institute and Bertarelli Foundation Gene Therapy Platform, Avrion Therapeutics targets genetically-linked neurodegenerative diseases using precision gene therapy to enable long-term solutions. Avrion has developed a first-in-class precision gene therapy platform to selectively and safely target discrete cells in the brain including neurons and astrocytes. This therapeutic platform is supported by a preclinical discovery engine to identify novel targets to treat neurodegenerative diseases with unmet medical need. Avrions’ first clinical candidate gene therapy product, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of motor neuron disease (amyotrophic lateral sclerosis - ALS), a fatal neuromuscular disorder.
The team of Peter M. Andersen at Umeå University has communicated breakthrough clinical data in amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. They followed an ALS patient treated with a novel anti-sense oligonuleotide (ASO) targeting SOD1 (tofersen), which is administered intrathecally every four weeks. Avrion Therapeutics is developing a precision gene therapy targeting SOD1 (AVR-001) based on single administration.
#genetherapy, #innovation, #startups
Researchers from the EPFL and LMU Munich – Ludwig-Maximilians-Universität München are drilling further into how the nervous system is affected in ALS. They have identified a novel drug target located on synapses, the connections between our neurons that are critical to communication between cells. The target emits an "eat me" signal to the immune cells of the nervous system, prompting them to “eat” or eliminate the synapses. This can lead to the impaired cognitive and motor function that we see in ALS.
The consortium is developing a therapeutic approach with a protein designed to mask this "eat me" signal, effectively preventing the immune cells from recognizing and destroying the synapses. By preserving these synaptic connections, the group aims to maintain spinal cord integrity and enhance motor function. In live mouse models, this innovative approach has already demonstrated significant spinal cord protection and improvements in motor function, marking a substantial advancement in this work.
This groundbreaking study highlights the potential of protecting synaptic pathways to mitigate ALS progression, offering a new avenue for therapeutic intervention. By safeguarding the synapses, the group hopes to slow down or even halt the neurodegenerative processes characteristic of ALS. This research exemplifies the cutting-edge work we are supporting to transform ALS treatment toward a future where ALS can be effectively managed.
#TargetALS#ALSResearch#NervousSystem#SynapseProtection#InnovativeTherapies
« Plus la maladie tente de me bâillonner, plus ma voix porte »
Voici les meilleurs extraits de l’interview d’Olivier Goy, qui est venu porter sa voix et celle des malades de SLA, sur le plateau de "Quelle époque" ce samedi 1er juin sur France 2.
Un témoignage poignant !
Un message essentiel !
Une voix à écouter !
Une homme dont s’inspirer !
« Si je déprime, je me punis 2x »
« j’ai la conviction que dévoiler ma maladie est indispensable »
« Il y aura un "après sans nous". Et qui que l’on soit, cela n’empêchera pas le monde de tourner. C’est pour cela qu’il faut transmettre de son vivant, vite et avec intensité. »
« À ceux qui ont peur [de la mort], je leur conseille de laisser un maximum de preuves de leur amour pour ceux qui comptent, pour qu’avec le temps qui s’écoulent, ils n’en doutent jamais. On est fort que de l’amour que l’on a reçu. »
« Maintenant je sais la valeur d’être tout simplement vivant, de ne pas se gâcher l’existence avec des pensées négatives, de prendre de la hauteur, mais cet amour de la vie est lié à une acceptation totale de l’existence de la mort. »
« La maladie ne m’empêche pas d’atteindre [mes] grands buts, au contraire elle me fournit un levier pour mener mes combats. Tout ce qui arrive avec "Invincible été", c’est grâce à elle. Je trouve que c’est un beau pied de nez à la maladie, plus elle m’abîme, plus mon message porte. Je suis invincible. »
#lundiildit
PS : tous les lundis, je vous partage la voix d’une personne inspirante.
The 2023 award for courage and tenacity went to Olivier Goy. He is an example to us all, reminding us that every challenge we face is nothing compared to his day-to-day life. He is our greatest motivation to complete the development of a gene therapy that will save patients suffering from Charcot's disease. (Max Murone and myself, we are now invincible!)
I connect people & businesses with the local life science ecosystem | Project Manager, Senior scientist | RNA Therapy, Drug Delivery, Nanomedicine, Longevity
Great opportunity to reconnect at the first Swiss biotech CFO Day organized by the Swiss Biotech Association, with Christian Mauriand from Roivant Sciences as Keynote speaker, and many outstanding panelists.
🔎 Cell therapy research: The Basel Area cluster is well positioned among the global leaders.
In this analysis by CIEB Universität Basel, discover how the gene therapy research landscape in Basel, #Switzerland, compares to other international clusters:
- Basel Area's gene therapy research cluster excels in catalyzing collaboration, particularly with international partners. The cluster demonstrates a strong connection between the academic and business sectors, with a substantial portion of its research stemming from the private sector.
- Basel’s research cluster surpasses the average of the world's top 50 clusters regarding research quality, as measured by the average impact factor of the journals in which articles are published. It also performs well in relevance, with an above-average number of citations and a share of the top 10% of the most cited articles.
- Boston is recognized as a global benchmark in this field. However, Basel's scientific rigour outperforms other notable clusters like London, Cambridge, and Heidelberg.
Get the landscape analysis in PDF here: https://lnkd.in/e8nZjjaP
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#genetherapeutics#biotech#geneediting#genetherapyBaselChristian Rutzer | Dragan Filimonovic | Valentina Francia, PhD | Frank Kumli | Paul Eschmann | Fabio Marelli | Anke Hollnagel | ¦Daniel Meier | Kirsten Detrick | Annelise Michel | Invest in Switzerland
