Alveron
Pharma, a newly incorporated company established by Okklo Life Sciences BV and
Sanquinnovate (part of Sanquin), is pleased to announce the closing of a EUR
6.5M Series A financing round led by Thuja together with Waterman Ventures and
Oost NL. This investment allows Alveron Pharma to select a lead compound,
conduct non-clinical studies, and complete a first clinical study to demonstrate
safety and obtain a first clinical readout.
Alveron
Pharma will focus on a unique class of cyclodextrin-based drugs, that have the
ability to promote blood coagulation without inducing it in the absence of
bleeding. Alveron’s first target is to reverse the effects of anticoagulants.
Anticoagulants (“blood thinners”) are widely prescribed to patients with
increased thromboembolic risk. While effective in preventing thrombosis, these
drugs also inhibit blood coagulation when this is needed urgently, e.g. in
patients who suffer a major bleed or in case of emergency surgery.
Anticoagulant related bleedings are not uncommon, and may be serious, and
difficult to treat since no or only selective reversal agents are available.
Alveron’s cyclodextrins are unique in the sense that they restore coagulation
independent of the type of anticoagulant used. Sales of selective reversal
agents have been estimated at around $2 billion per annum.
Ben
Nichols, CEO of Alveron Pharma: “There is a clear unmet clinical need for a
universal anti-coagulant reversal agent, and the early pre-clinical data for
Alveron’s unique cyclodextrin compounds are compelling. Having recently taken a
new class of coagulant into clinical trials, I am thrilled to be joining the
founders of this business, Stephan
Peters (who conceived the original idea, CSO) and Alex Zwiers (COO), with their
expertise on cyclodextrins, and coagulation expert at Sanquin, Joost
Meijers (Chairman of the Scientific
Advisory Board). Alex was also involved in the development of the world’s first
cyclodextrin-based medicine, Bridion®.”
“Multiple
factors come together in this investment that are highly attractive”, says
Michel Briejer of Thuja, who led this transaction. “A highly experienced and
knowledgeable team, a promising set of drug leads, and a reputable partner in
the field of blood, Sanquin. We are extremely pleased to be part of this
venture and are confident that Alveron will create success for doctors and
patients alike.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-16 11:51:342019-05-21 11:52:43Alveron pharma closes series A round to advance a cyclodextrin based procoagulant medicine into the clinic← #nieuws
InteRNA
Technologies announced today the recent publication of data from a
collaboration with University Medical Center (UMC) Utrecht in Journal of
Neuroscience showing that the downregulation of miR-135a levels can reduce
seizure activity after the onset of spontaneous recurrent seizures (SRS) in
chronic-stage Mesial Temporal Lobe Epilepsy (mTLE) in mouse models. miR-135 is
a microRNA (miRNA) known to control neuronal morphology and synaptic function
that is upregulated in neurons in the epileptic brain. The data support the
overall potential of miRNAs as a treatment modality in complex diseases and
also underscore InteRNA’s expertise in the application of miRNAs as a new
therapeutic approach.
The paper
provides novel insights into the pathological mechanisms of mTLE, which remain
poorly understood to-date. In the study, it was shown for the first time that
miR-135a is significantly overexpressed in human mTLE hippocampal samples and
that the silencing of this specific miRNA in mouse models by an antimir
drastically reduces the number, severity and time spent in seizures in the SRS
stage, an advanced disease stage where current treatments fail to induce an
effect. Furthermore, novel neuronal downstream targets of miR-135a were
identified. In particular, the role of Mef2a, an activity-dependent
transcription factor regulating synaptic strength, was investigated in closer
detail. The data demonstrated that miR-135a and Mef2a show reciprocal
expression regulation in TLE and that Mef2a is required for miR135a-induced
dendritic spine changes in neurons that might induce synaptic defects involved
in the pathomechanisms of TLE. The research paper titled, “Antagonizing
increased miR-135a levels at the chronic stage of experimental TLE reduces
spontaneous recurrent seizures” was published in Journal of Neuroscience on
April 23, 2019.
“TLE
accounts for a third of all epilepsies and available treatments lack
disease-modifying effects and are ineffective in one third of all mTLE patients,”
commented Prof. Jeroen Pasterkamp, PhD, Department of Translational
Neuroscience, UMC Utrecht Brain Center. “The data from this study indicate that
targeting epilepsy-associated miRNAs has the potential to unlock a novel
therapeutic entity and provides key insights into the disease biology of
epilepsy.”
Roel Schaapveld, CEO of InteRNA added: “The published data provide evidence that targeting a single miRNA could represent a new modality in the epilepsy field where current treatments are solely based on ion channel blockers that only relieve symptoms rather than provide curative effects. We are encouraged by the results of this study that further validate InteRNA’s proprietary miRNA platform, which has the potential to target cancer, our primary focus, as well as other complex disease states outside of the realm of oncology.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-15 10:34:292019-05-21 10:40:52InteRNA and UMC Utrecht Publish Preclinical Evidence for Potential of miRNAs in Treatment of Epilepsy← #nieuws
DCprime, the front-runner in the field of relapse vaccines, today announced presentations of additional preclinical data sets for its lead program, DCP-001, at the 7th CCBIO Annual Symposium and the 2019 CIMT Annual Meeting. The data supports key product characteristics and sheds additional light on the mechanism-of-action of DCP-001, a whole cell-based vaccine derived from the company’s proprietary DCOne® human leukemic cell line. DCP-001 is currently studied in an international Phase II trial in AML patients who are ineligible for hematopoietic stem cell transplantations.
“While we are making constant progress evaluating DCP-001 in the clinic in AML and preparing additional trials in MDS and Multiple Myeloma, we continue to strengthen the body of preclinical data supporting this program,” commented Dr Jeroen Rovers, CMO of DCprime. “The preclinical results and animal models we have generated in our collaboration with the University of Bergen provide further insights into the vaccine’s mechanism-of-action and confirm several key product characteristics relevant to our approach. Overall, these data strongly support the rationale of DCP-001 as a relapse vaccine providing immune control over residual disease in hematological malignancies.”
DCOne cells endogenously express known and unknown tumor-associated antigens. In the DCP-001 manufacturing process, DCOne cells are differentiated and matured into cells with a mature dendritic cell phenotype, which is responsible for the strong immunogenic properties of the vaccine.
In its collaboration with the University of Bergen, Norway, DCprime has established a preclinical mouse model to study DCP-001 vaccinations in monotherapy and in combination therapy settings. The collaboration is supported by the Horizon 2020 EU grant AML-VACCiN. The data which were presented yesterday at the 7th CCBIO Annual Symposium showed that DCP-001 was able to suppress tumor growth in humanized immunocompetent mice. The results also demonstrated that the transformation of DCOne leukemic cells into DCP-001 leads to an immunogenic shift. Whereas the parental leukemic cells were poorly immunogenic, DCP-001 proved highly immunogenic, making it an attractive cancer vaccine candidate. Furthermore, DCP-001 induced the production of a broad range of pro-inflammatory cytokines in peripheral blood mononuclear cells (PBMCs) derived from healthy donors.
In preclinical studies, which will be presented at the upcoming 2019 CIMT Annual Meeting, DCprime was able to demonstrate that human antigen presenting cells (APCs) efficiently process DCP-001 through phagocytosis. These in vitro data suggest that in vivo, upon intradermal injection, DCP-001 will induce a strong inflammatory response, and is ingested by both resident and attracted host APCs, which subsequently prime tumor-reactive T cells. These data support the proposed mode of action whereby host APCs present DCP-001 antigens to the host immune system following intradermal vaccination.
Goed nieuws, de Australische overheid heeft besloten dat het
aanpassen van genetische eigenschappen met moderne gene-editing technieken
(zoals CRISPR-Cas) niet onder de wetgeving voor genetische modificatie valt. Onderzoekers
en bedrijven zijn blij met deze beslissing. Zij kunnen met deze moderne technieken veel sneller en
gerichter planten met gunstige eigenschappen en levensreddende therapieën
ontwikkelen. Australië erkent met deze stap de toegevoegde waarde van deze
toepassingen van gene-editing voor de samenleving en volgt met de vrijstelling andere
belangrijke landen als Canada, de US en Argentinië.
Voorwaarde voor de vrijstelling is dat de gene-editing
techniek alleen wordt gebruikt voor het knippen van het DNA en dat het
natuurlijke herstelmechanisme van de cel de breuk repareert. De redenatie
achter de beslissing van de Australische overheid is dat we al jaren planten en
dieren genetisch veranderen. In dieren doen we dit heel gericht via speciale
fokprogramma’s en planten bombarderen we met straling of behandelen we met
chemicaliën. Deze methoden zijn op basis van hun historische veiligheid
vrijgesteld van de wetgeving voor genetische modificatie. Het zou dan vreemd
zijn om technieken die veel gerichter mutaties kunnen aanbrengen opeens wel
onder die wetgeving te laten vallen.
HollandBIO vindt dit een logische en pragmatische redenatie.
Hopelijk volgt Europa snel met het vrijstellen van gene-editing, zodat ook hier
boeren, consumenten en patiënten snel kunnen profiteren van een duurzame
landbouw, gezonde voeding en baanbrekende behandelingen.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-14 19:21:582019-05-14 19:22:00Australië: gene-editing niet onder GM-wetgeving← #nieuws
Zakendoen of kennis opdoen over Life Sciences & Health, Robotics/AI of Climate Resilience in Boston, de hoofdstad van de Amerikaanse staat Massachusetts? Ga dan van 17 tot en met 19 juli mee met de economische missie in aanwezigheid van minister-president Mark Rutte, minister voor Medische Zorg en Sport, Bruno Bruins en de watergezant Henk Ovink. Rijksdienst voor Ondernemen organiseert deze handelsmissie in samenwerking met Task Force Health Care en zakelijk missieleider Hans de Boer, voorzitter van VNO-NCW. Aanmelden kan tot en met donderdag 15 mei
HollandBIO
welcomes InnSense as a new member. InnSense specializes in providing temporary
and long term resourcing solutions to knowledge and capacity issues through
consultancy, outsourcing, project management & staffing within the (bio)
pharmaceutical-, medical devices and life science industry in The Netherlands.
The
services are primarily within the clinical research, market access, medical and
commercial field through which InnSense is able to serve customers, both
start-ups and established companies, during the entire life cycle of their
products. The solutions are, irrespective of duration, size or complexity of a
project, applicable in a flexible way over the entire spectrum and range from a
single expert to an entire team and/or department with a complete
infrastructure.
InnSense is passionate about healthcare and the ambition is helping customers to succeed with sustainable growth in the new healthcare environment, in which customers can stay focused on their core activities and patients get access to value based therapies and medicines. Interested in their services or in collaboration? Please visit www.innsense.nl
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-14 17:52:272019-05-14 17:55:32InnSense has joined the HollandBIO community← #nieuws
HollandBIO is blij
met de start van de pilot “Parallelle Procedures CBG-ZIN” van hetCBG en Zorginstituut om
innovatieve geneesmiddelen sneller bij de patiënt te krijgen. De pilot sluit
naadloos aan bij het HollandBIO programma Sneller & beter van lab naar
patiënt, waarin wij werken aan oplossingen waarbij gezondheidswinst, innovatie
en betaalbaarheid hand in hand gaan.
Met de pilot slaan CBG en ZIN de handen ineen om de tijd van
lab naar patiënt te verkorten, door eerder te starten met het nationale vergoedingstraject
en dit (deels) parallel te laten lopen met het Europese registratietraject.
Normaal gesproken start het vergoedingstraject pas na het verkrijgen van de
handelsvergunning. HollandBIO en de VIG werken samen met het CBG en het
Zorginstituut om te inventariseren welke geneesmiddelen/ fabrikanten in
aanmerking zouden komen voor deelname aan de pilot, die zich in de opstartfase
bevindt. Zodra we meer informatie hebben over hoe fabrikanten kunnen
participeren in deze pilot laten we dat natuurlijk weten.
De pilot is een mooie eerste stap om van Nederland een
internationale proeftuin te maken voor oplossingen die de route van lab naar
patiënt versnellen en verbeteren. En dat is hard nodig. Want hoewel de
biotechsector haar belofte waarmaakt door de introductie van baanbrekende geneesmiddelen
en therapieën, duurt geneesmiddelontwikkeling te lang, is het te kostbaar en te
risicovol. Om ook in de toekomst de vruchten te kunnen plukken van innovatie,
moeten we daar verandering in aanbrengen. Helaas is er geen quick fix, duurzame
oplossingen vergen visie en tijd. In ons programma Sneller & beter van lab
naar patiënt pleit HollandBIO voor oplossingsrichtingen waarbij
gezondheidswinst, innovatie én betaalbaarheid hand in hand gaan: het
patiëntenperspectief centraal in elke fase van geneesmiddelenontwikkeling, de
ontwikkeling en inzet van betere meetmodellen en slimme diagnostica en een
flexibel ecosysteem waarin registratie, vergoeding en toegang naadloos op
elkaar aansluiten.
HollandBIO is ervan overtuigd dat de pilot “Parallelle
Procedures CBG-ZIN” mogelijkheden biedt om een flexibel gezondheidsecosysteem
een stap dichterbij te brengen, zodat Nederland klaar is om de volle potentie
van de geneesmiddelen van morgen te verzilveren!
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-13 19:47:222019-05-13 19:47:24Pilot “Parallelle Procedures CBG-ZIN” van start: innovatieve geneesmiddelen sneller bij de patiënt← #nieuws
Willen we in de toekomst
nieuwe, effectieve én betaalbare geneesmiddelen, dan moeten we vandaag
oplossingen implementeren die de route van lab naar patiënt versnellen en
verbeteren. Het opwerpen van extra drempels, door bijvoorbeeld al in de
vroegste fase van ontwikkeling voorwaarden te stellen aan de prijs van een
eventueel geneesmiddel bij publieke financiering van onderzoek, werkt juist
averechts. Het is simpelweg de verkeerde knop om aan te draaien. Dat stelt
HollandBIO in reactie op het vandaag verschenen rapport van SOMO en Wemos over
de rol van publieke financiering in geneesmiddelenonderzoek.
Er staat veel op het spel: in de strijd tegen ziekte staan we nog veel te
vaak met lege handen. Maar de route om een geneesmiddel van lab naar patiënt te
brengen, is wetenschappelijk uitdagend, lang, duur en risicovol. Dat staat onze
ambitie in de weg om op een dag elke patiënt van een effectieve en betaalbare
behandeling te voorzien. Het moet sneller en beter.
Inzicht in de kostendrijvers is een belangrijke voorwaarde voor de
implementatie van effectieve oplossingen. Een recente analyse van Gupta
concludeert dat mislukkingen en kapitaalkosten de grootste kostendrijvers zijn
in de ontwikkeling van geneesmiddelen. Maar liefst 92% van het
geneesmiddelenonderzoek dat het schopt tot de testfase op mensen, faalt alsnog.
Als investeerders al bereid zijn dit risico te nemen, moeten ze ook nog eens
engelengeduld hebben: de ontwikkeling van een geneesmiddel duurt in de regel
tien tot vijftien jaar. Om wat aan de prijs van geneesmiddelen te doen, hebben
we dus oplossingen nodig die het traject van lab naar patiënt de-risken en
versnellen.
Publieke financiering in de vroege fase van geneesmiddelenontwikkeling, of
het nou binnen een bedrijf, een onderzoeksinstelling of een stichting is, is
hard nodig. Want juist in deze fase laat de private markt het nog afweten.
Zetten we de publieke investeringen af tegen de private investeringen, dan is
er maar één conclusie mogelijk: dubbel betalen is pertinente onzin. Gupta
becijferde dat de ontwikkeling van één enkel geneesmiddel gemiddeld 2,2 miljard
euro kost. De publieke bijdrage ligt in de case studies van SOMO rond de 10
miljoen euro, en een aanzienlijk deel van die bijdrage wordt ook nog eens met
meer dan marktconforme rente terugbetaald. Dat een investeerder in ruil voor
0,5% financiering eisen kan stellen aan de uiteindelijke prijsstelling, zoals
SOMO bepleit, getuigt van weinig realiteitszin.
Nederland heeft een internationaal vooraanstaande kennispositie in medisch
onderzoek. Die kennis moet niet op de plank blijven liggen, maar moet
verzilverd worden in gezondheidswinst. Daarom verdient iedereen die de ambitie,
het inzicht en het doorzettingsvermogen heeft om een nieuw geneesmiddel te
ontwikkelen onze volledige steun, of dat nou in binnen een bedrijf, NGO of de
academie is. HollandBIO pleit voor meer publieke investeringen, tegen
stimulerende en internationaal concurrerende voorwaarden. De op te richten
investeringsinstelling Invest-NL biedt een uitgelezen kans om het huidige
marktfalen weg te nemen.
Laten we de handen ineen slaan en inzetten op effectieve oplossingen waarbij
gezondheid, innovatie en betaalbaarheid hand in hand gaan. Zo kan Nederland
uitgroeien tot het Boston van Europa, en profiteren we allemaal van onze
gezamenlijke investeringen in geneesmiddelenonderzoek.
Two Dutch companies – DDL, a state-of-the-art laboratory specializing in molecular diagnostic testing and assay development and ENPICOM, an innovative immunomics data analysis company – Today announced that they have started a collaboration to jointly deliver immune repertoire sequencing and analysis service to organizations studying immune system related diseases or developing drugs influencing this system.
Immune repertoire sequencing is a powerful way to analyze hundreds of thousands to millions of T or B cell receptors swiftly and in a cost-effective way. This new technology is rapidly gaining interest from researchers and physicians who want to unravel the adaptive immune responses in areas like basic and translational immunology, immuno-oncology, autoimmune disorders, inflammatory and infectious diseases, vaccine research and transplantation. From target discovery to patient stratification in clinical trials with immunotherapies, repertoire sequencing is becoming an important addition to accelerate the different drug development processes and make it more cost-effective.
“DDL is very pleased to start this partnership with ENPICOM in this new and challenging field of immunomics”, says Jan Lindeman, DDL’s Chief Executive Officer. “It further expands DDL’s portfolio of tailor-made molecular diagnostic services for our (bio)pharmaceutical customers. ENPICOM’s expertise and know-how are highly complementary to DDL’s expertise and we are confident that this partnership will offer a unique opportunity to accelerate immunotherapy discovery and development.”
Jos Lunenberg, ENPICOM’s Chief Executive Officer comments: “Last month we launched our IGX platform to manage, store, analyze, visualize and interpret immune repertoire sequencing data from T and B cell receptors. Now we are also able to support organizations lacking the specific expertise or capabilities to perform repertoire sequencing with our smart new analysis and visualization methods. In DDL we have found a highly respected business partner with an excellent track record as molecular diagnostic testing laboratory for the biopharmaceutical industry. I’m very pleased to be able to go public with this partnership today.” He continues: “There are a few other kits and services for this specific form of targeted sequencing available in the market, but none or very limited data analysis is included. Clonality analysis is just a first step to bridge the existing gap between producing raw sequencing data and obtaining important insights from the data. So, in essence, we start where others stop.”
The European Lead Factory (ELF) secured a total project budget of EUR 36.5 million under the second framework of the Innovative Medicines Initiative (IMI). Over the next 5 years, 20 partners in 7 countries will push forward the transformation of potential drug targets to new medicines in the new project ESCulab (European Screening Centre: unique library for attractive biology) under the European Lead Factory brand.
Jon de Vlieger, coordinator of the ESCulab consortium at Lygature, commented: “It’s truly exciting to continue the onboarding of new and innovative proposals for screening and provide high quality starting points for drug discovery to academics and SMEs throughout Europe. In an effort to broaden our scope we are not only looking for target-based approaches, but now also enable phenotypic screens.”
Proven platform for smart new ideas
Universities, research organisations and SMEs have a diverse range of potential drug targets but cannot easily access suitable compound libraries and screening facilities. Pharmaceutical companies need access to high quality targets to bring innovative therapies to the patient. The European Lead Factory combines the large high-quality compound libraries derived from the pharmaceutical industry and the Public Compound Collection and Europe’s leading screening facility with the innovative targets held by academic organisations in a public-private partnership. This offers an ideal platform to translate early-stage fundamental biological research into credible and investable starting points for drug discovery campaigns.
Stefan Jaroch, project lead of Bayer AG, confirms that: “the European Lead Factory has clearly shown how crowd sourcing and collective intelligence can indeed advance biological concepts into drug discovery projects that benefit academia, industry, society and ultimately patients.”
New ideas + New partners = New chances
Over the next five years,the European Lead Factory will initiate 185 new drug discovery projects by screening medically relevant drug targets from European researchers, small and medium-sized enterprises and pharmaceutical industry against the ELF library of 550,000 unique chemical compounds.
The successful concept of the European Lead Factory has already encouraged additional private partners to join. The ESCulab Project welcomes the two pharmaceutical companies Servier and Grünenthal as well as the Medicines for Malaria Venture (MMV), the leading product development partnership in the field of antimalarial drug research and development.
Tim Wells, Chief Scientific Officer at MMV, adds: “We are thrilled to participate as ESCulab and the European Lead Factory represent a chance for new partnerships for MMV, both with the EU through IMI and with a variety of highly creative companies. These partnerships give access to a novel, high quality chemical library that we believe is important to be screened against high priority malaria targets.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-05-07 18:19:362019-05-07 18:19:38The European Lead Factory, Europe’s largest collaborative drug discovery platform, continues its success story
Alveron pharma closes series A round to advance a cyclodextrin based procoagulant medicine into the clinic
InnovatieklimaatAlveron Pharma, a newly incorporated company established by Okklo Life Sciences BV and Sanquinnovate (part of Sanquin), is pleased to announce the closing of a EUR 6.5M Series A financing round led by Thuja together with Waterman Ventures and Oost NL. This investment allows Alveron Pharma to select a lead compound, conduct non-clinical studies, and complete a first clinical study to demonstrate safety and obtain a first clinical readout.
Alveron Pharma will focus on a unique class of cyclodextrin-based drugs, that have the ability to promote blood coagulation without inducing it in the absence of bleeding. Alveron’s first target is to reverse the effects of anticoagulants. Anticoagulants (“blood thinners”) are widely prescribed to patients with increased thromboembolic risk. While effective in preventing thrombosis, these drugs also inhibit blood coagulation when this is needed urgently, e.g. in patients who suffer a major bleed or in case of emergency surgery. Anticoagulant related bleedings are not uncommon, and may be serious, and difficult to treat since no or only selective reversal agents are available. Alveron’s cyclodextrins are unique in the sense that they restore coagulation independent of the type of anticoagulant used. Sales of selective reversal agents have been estimated at around $2 billion per annum.
Ben Nichols, CEO of Alveron Pharma: “There is a clear unmet clinical need for a universal anti-coagulant reversal agent, and the early pre-clinical data for Alveron’s unique cyclodextrin compounds are compelling. Having recently taken a new class of coagulant into clinical trials, I am thrilled to be joining the founders of this business, Stephan Peters (who conceived the original idea, CSO) and Alex Zwiers (COO), with their expertise on cyclodextrins, and coagulation expert at Sanquin, Joost Meijers (Chairman of the Scientific Advisory Board). Alex was also involved in the development of the world’s first cyclodextrin-based medicine, Bridion®.”
“Multiple factors come together in this investment that are highly attractive”, says Michel Briejer of Thuja, who led this transaction. “A highly experienced and knowledgeable team, a promising set of drug leads, and a reputable partner in the field of blood, Sanquin. We are extremely pleased to be part of this venture and are confident that Alveron will create success for doctors and patients alike.”
InteRNA and UMC Utrecht Publish Preclinical Evidence for Potential of miRNAs in Treatment of Epilepsy
GezondheidInteRNA Technologies announced today the recent publication of data from a collaboration with University Medical Center (UMC) Utrecht in Journal of Neuroscience showing that the downregulation of miR-135a levels can reduce seizure activity after the onset of spontaneous recurrent seizures (SRS) in chronic-stage Mesial Temporal Lobe Epilepsy (mTLE) in mouse models. miR-135 is a microRNA (miRNA) known to control neuronal morphology and synaptic function that is upregulated in neurons in the epileptic brain. The data support the overall potential of miRNAs as a treatment modality in complex diseases and also underscore InteRNA’s expertise in the application of miRNAs as a new therapeutic approach.
The paper provides novel insights into the pathological mechanisms of mTLE, which remain poorly understood to-date. In the study, it was shown for the first time that miR-135a is significantly overexpressed in human mTLE hippocampal samples and that the silencing of this specific miRNA in mouse models by an antimir drastically reduces the number, severity and time spent in seizures in the SRS stage, an advanced disease stage where current treatments fail to induce an effect. Furthermore, novel neuronal downstream targets of miR-135a were identified. In particular, the role of Mef2a, an activity-dependent transcription factor regulating synaptic strength, was investigated in closer detail. The data demonstrated that miR-135a and Mef2a show reciprocal expression regulation in TLE and that Mef2a is required for miR135a-induced dendritic spine changes in neurons that might induce synaptic defects involved in the pathomechanisms of TLE. The research paper titled, “Antagonizing increased miR-135a levels at the chronic stage of experimental TLE reduces spontaneous recurrent seizures” was published in Journal of Neuroscience on April 23, 2019.
“TLE accounts for a third of all epilepsies and available treatments lack disease-modifying effects and are ineffective in one third of all mTLE patients,” commented Prof. Jeroen Pasterkamp, PhD, Department of Translational Neuroscience, UMC Utrecht Brain Center. “The data from this study indicate that targeting epilepsy-associated miRNAs has the potential to unlock a novel therapeutic entity and provides key insights into the disease biology of epilepsy.”
Roel Schaapveld, CEO of InteRNA added: “The published data provide evidence that targeting a single miRNA could represent a new modality in the epilepsy field where current treatments are solely based on ion channel blockers that only relieve symptoms rather than provide curative effects. We are encouraged by the results of this study that further validate InteRNA’s proprietary miRNA platform, which has the potential to target cancer, our primary focus, as well as other complex disease states outside of the realm of oncology.”
Source: InteRNA
DCprime Presents Comprehensive Preclinical Results Supporting Lead Clinical Candidate DCP-001
GezondheidDCprime, the front-runner in the field of relapse vaccines, today announced presentations of additional preclinical data sets for its lead program, DCP-001, at the 7th CCBIO Annual Symposium and the 2019 CIMT Annual Meeting. The data supports key product characteristics and sheds additional light on the mechanism-of-action of DCP-001, a whole cell-based vaccine derived from the company’s proprietary DCOne® human leukemic cell line. DCP-001 is currently studied in an international Phase II trial in AML patients who are ineligible for hematopoietic stem cell transplantations.
“While we are making constant progress evaluating DCP-001 in the clinic in AML and preparing additional trials in MDS and Multiple Myeloma, we continue to strengthen the body of preclinical data supporting this program,” commented Dr Jeroen Rovers, CMO of DCprime. “The preclinical results and animal models we have generated in our collaboration with the University of Bergen provide further insights into the vaccine’s mechanism-of-action and confirm several key product characteristics relevant to our approach. Overall, these data strongly support the rationale of DCP-001 as a relapse vaccine providing immune control over residual disease in hematological malignancies.”
DCOne cells endogenously express known and unknown tumor-associated antigens. In the DCP-001 manufacturing process, DCOne cells are differentiated and matured into cells with a mature dendritic cell phenotype, which is responsible for the strong immunogenic properties of the vaccine.
In its collaboration with the University of Bergen, Norway, DCprime has established a preclinical mouse model to study DCP-001 vaccinations in monotherapy and in combination therapy settings. The collaboration is supported by the Horizon 2020 EU grant AML-VACCiN. The data which were presented yesterday at the 7th CCBIO Annual Symposium showed that DCP-001 was able to suppress tumor growth in humanized immunocompetent mice. The results also demonstrated that the transformation of DCOne leukemic cells into DCP-001 leads to an immunogenic shift. Whereas the parental leukemic cells were poorly immunogenic, DCP-001 proved highly immunogenic, making it an attractive cancer vaccine candidate. Furthermore, DCP-001 induced the production of a broad range of pro-inflammatory cytokines in peripheral blood mononuclear cells (PBMCs) derived from healthy donors.
In preclinical studies, which will be presented at the upcoming 2019 CIMT Annual Meeting, DCprime was able to demonstrate that human antigen presenting cells (APCs) efficiently process DCP-001 through phagocytosis. These in vitro data suggest that in vivo, upon intradermal injection, DCP-001 will induce a strong inflammatory response, and is ingested by both resident and attracted host APCs, which subsequently prime tumor-reactive T cells. These data support the proposed mode of action whereby host APCs present DCP-001 antigens to the host immune system following intradermal vaccination.
The poster presentation at the upcoming 2019 CIMT Annual Meeting will take place on May 21, 2019 at the Rheingoldhalle Congress Center Mainz, Germany. To read the full abstract, please go to:An in vitro study of the interactions between the cell-based cancer vaccine DCP-001 and antigen presenting cells
Source: DCprime
Australië: gene-editing niet onder GM-wetgeving
Wereld mét toekomst, Voedsel en materialenGoed nieuws, de Australische overheid heeft besloten dat het aanpassen van genetische eigenschappen met moderne gene-editing technieken (zoals CRISPR-Cas) niet onder de wetgeving voor genetische modificatie valt. Onderzoekers en bedrijven zijn blij met deze beslissing. Zij kunnen met deze moderne technieken veel sneller en gerichter planten met gunstige eigenschappen en levensreddende therapieën ontwikkelen. Australië erkent met deze stap de toegevoegde waarde van deze toepassingen van gene-editing voor de samenleving en volgt met de vrijstelling andere belangrijke landen als Canada, de US en Argentinië.
Voorwaarde voor de vrijstelling is dat de gene-editing techniek alleen wordt gebruikt voor het knippen van het DNA en dat het natuurlijke herstelmechanisme van de cel de breuk repareert. De redenatie achter de beslissing van de Australische overheid is dat we al jaren planten en dieren genetisch veranderen. In dieren doen we dit heel gericht via speciale fokprogramma’s en planten bombarderen we met straling of behandelen we met chemicaliën. Deze methoden zijn op basis van hun historische veiligheid vrijgesteld van de wetgeving voor genetische modificatie. Het zou dan vreemd zijn om technieken die veel gerichter mutaties kunnen aanbrengen opeens wel onder die wetgeving te laten vallen.
HollandBIO vindt dit een logische en pragmatische redenatie. Hopelijk volgt Europa snel met het vrijstellen van gene-editing, zodat ook hier boeren, consumenten en patiënten snel kunnen profiteren van een duurzame landbouw, gezonde voeding en baanbrekende behandelingen.
Bron: ABC News
Economische missie Boston – Life Sciences & Health
Hollandbio, Innovatieklimaat, UitgelichtZakendoen of kennis opdoen over Life Sciences & Health, Robotics/AI of Climate Resilience in Boston, de hoofdstad van de Amerikaanse staat Massachusetts? Ga dan van 17 tot en met 19 juli mee met de economische missie in aanwezigheid van minister-president Mark Rutte, minister voor Medische Zorg en Sport, Bruno Bruins en de watergezant Henk Ovink. Rijksdienst voor Ondernemen organiseert deze handelsmissie in samenwerking met Task Force Health Care en zakelijk missieleider Hans de Boer, voorzitter van VNO-NCW. Aanmelden kan tot en met donderdag 15 mei
InnSense has joined the HollandBIO community
HollandbioHollandBIO welcomes InnSense as a new member. InnSense specializes in providing temporary and long term resourcing solutions to knowledge and capacity issues through consultancy, outsourcing, project management & staffing within the (bio) pharmaceutical-, medical devices and life science industry in The Netherlands.
The services are primarily within the clinical research, market access, medical and commercial field through which InnSense is able to serve customers, both start-ups and established companies, during the entire life cycle of their products. The solutions are, irrespective of duration, size or complexity of a project, applicable in a flexible way over the entire spectrum and range from a single expert to an entire team and/or department with a complete infrastructure.
InnSense is passionate about healthcare and the ambition is helping customers to succeed with sustainable growth in the new healthcare environment, in which customers can stay focused on their core activities and patients get access to value based therapies and medicines. Interested in their services or in collaboration? Please visit www.innsense.nl
Pilot “Parallelle Procedures CBG-ZIN” van start: innovatieve geneesmiddelen sneller bij de patiënt
Gezondheid op maat, Hollandbio, GezondheidHollandBIO is blij met de start van de pilot “Parallelle Procedures CBG-ZIN” van het CBG en Zorginstituut om innovatieve geneesmiddelen sneller bij de patiënt te krijgen. De pilot sluit naadloos aan bij het HollandBIO programma Sneller & beter van lab naar patiënt, waarin wij werken aan oplossingen waarbij gezondheidswinst, innovatie en betaalbaarheid hand in hand gaan.
Met de pilot slaan CBG en ZIN de handen ineen om de tijd van lab naar patiënt te verkorten, door eerder te starten met het nationale vergoedingstraject en dit (deels) parallel te laten lopen met het Europese registratietraject. Normaal gesproken start het vergoedingstraject pas na het verkrijgen van de handelsvergunning. HollandBIO en de VIG werken samen met het CBG en het Zorginstituut om te inventariseren welke geneesmiddelen/ fabrikanten in aanmerking zouden komen voor deelname aan de pilot, die zich in de opstartfase bevindt. Zodra we meer informatie hebben over hoe fabrikanten kunnen participeren in deze pilot laten we dat natuurlijk weten.
De pilot is een mooie eerste stap om van Nederland een internationale proeftuin te maken voor oplossingen die de route van lab naar patiënt versnellen en verbeteren. En dat is hard nodig. Want hoewel de biotechsector haar belofte waarmaakt door de introductie van baanbrekende geneesmiddelen en therapieën, duurt geneesmiddelontwikkeling te lang, is het te kostbaar en te risicovol. Om ook in de toekomst de vruchten te kunnen plukken van innovatie, moeten we daar verandering in aanbrengen. Helaas is er geen quick fix, duurzame oplossingen vergen visie en tijd. In ons programma Sneller & beter van lab naar patiënt pleit HollandBIO voor oplossingsrichtingen waarbij gezondheidswinst, innovatie én betaalbaarheid hand in hand gaan: het patiëntenperspectief centraal in elke fase van geneesmiddelenontwikkeling, de ontwikkeling en inzet van betere meetmodellen en slimme diagnostica en een flexibel ecosysteem waarin registratie, vergoeding en toegang naadloos op elkaar aansluiten.
HollandBIO is ervan overtuigd dat de pilot “Parallelle Procedures CBG-ZIN” mogelijkheden biedt om een flexibel gezondheidsecosysteem een stap dichterbij te brengen, zodat Nederland klaar is om de volle potentie van de geneesmiddelen van morgen te verzilveren!
Dubbel betalen is pertinente onzin
Gezondheid op maat, Hollandbio, Gezondheid, UitgelichtWillen we in de toekomst nieuwe, effectieve én betaalbare geneesmiddelen, dan moeten we vandaag oplossingen implementeren die de route van lab naar patiënt versnellen en verbeteren. Het opwerpen van extra drempels, door bijvoorbeeld al in de vroegste fase van ontwikkeling voorwaarden te stellen aan de prijs van een eventueel geneesmiddel bij publieke financiering van onderzoek, werkt juist averechts. Het is simpelweg de verkeerde knop om aan te draaien. Dat stelt HollandBIO in reactie op het vandaag verschenen rapport van SOMO en Wemos over de rol van publieke financiering in geneesmiddelenonderzoek.
Er staat veel op het spel: in de strijd tegen ziekte staan we nog veel te vaak met lege handen. Maar de route om een geneesmiddel van lab naar patiënt te brengen, is wetenschappelijk uitdagend, lang, duur en risicovol. Dat staat onze ambitie in de weg om op een dag elke patiënt van een effectieve en betaalbare behandeling te voorzien. Het moet sneller en beter.
Inzicht in de kostendrijvers is een belangrijke voorwaarde voor de implementatie van effectieve oplossingen. Een recente analyse van Gupta concludeert dat mislukkingen en kapitaalkosten de grootste kostendrijvers zijn in de ontwikkeling van geneesmiddelen. Maar liefst 92% van het geneesmiddelenonderzoek dat het schopt tot de testfase op mensen, faalt alsnog. Als investeerders al bereid zijn dit risico te nemen, moeten ze ook nog eens engelengeduld hebben: de ontwikkeling van een geneesmiddel duurt in de regel tien tot vijftien jaar. Om wat aan de prijs van geneesmiddelen te doen, hebben we dus oplossingen nodig die het traject van lab naar patiënt de-risken en versnellen.
Publieke financiering in de vroege fase van geneesmiddelenontwikkeling, of het nou binnen een bedrijf, een onderzoeksinstelling of een stichting is, is hard nodig. Want juist in deze fase laat de private markt het nog afweten. Zetten we de publieke investeringen af tegen de private investeringen, dan is er maar één conclusie mogelijk: dubbel betalen is pertinente onzin. Gupta becijferde dat de ontwikkeling van één enkel geneesmiddel gemiddeld 2,2 miljard euro kost. De publieke bijdrage ligt in de case studies van SOMO rond de 10 miljoen euro, en een aanzienlijk deel van die bijdrage wordt ook nog eens met meer dan marktconforme rente terugbetaald. Dat een investeerder in ruil voor 0,5% financiering eisen kan stellen aan de uiteindelijke prijsstelling, zoals SOMO bepleit, getuigt van weinig realiteitszin.
Nederland heeft een internationaal vooraanstaande kennispositie in medisch onderzoek. Die kennis moet niet op de plank blijven liggen, maar moet verzilverd worden in gezondheidswinst. Daarom verdient iedereen die de ambitie, het inzicht en het doorzettingsvermogen heeft om een nieuw geneesmiddel te ontwikkelen onze volledige steun, of dat nou in binnen een bedrijf, NGO of de academie is. HollandBIO pleit voor meer publieke investeringen, tegen stimulerende en internationaal concurrerende voorwaarden. De op te richten investeringsinstelling Invest-NL biedt een uitgelezen kans om het huidige marktfalen weg te nemen.
Laten we de handen ineen slaan en inzetten op effectieve oplossingen waarbij gezondheid, innovatie en betaalbaarheid hand in hand gaan. Zo kan Nederland uitgroeien tot het Boston van Europa, en profiteren we allemaal van onze gezamenlijke investeringen in geneesmiddelenonderzoek.
DDL diagnostic laboratory and ENPICOM join forces
InnovatieklimaatTwo Dutch companies – DDL, a state-of-the-art laboratory specializing in molecular diagnostic testing and assay development and ENPICOM, an innovative immunomics data analysis company – Today announced that they have started a collaboration to jointly deliver immune repertoire sequencing and analysis service to organizations studying immune system related diseases or developing drugs influencing this system.
Immune repertoire sequencing is a powerful way to analyze hundreds of thousands to millions of T or B cell receptors swiftly and in a cost-effective way. This new technology is rapidly gaining interest from researchers and physicians who want to unravel the adaptive immune responses in areas like basic and translational immunology, immuno-oncology, autoimmune disorders, inflammatory and infectious diseases, vaccine research and transplantation. From target discovery to patient stratification in clinical trials with immunotherapies, repertoire sequencing is becoming an important addition to accelerate the different drug development processes and make it more cost-effective.
“DDL is very pleased to start this partnership with ENPICOM in this new and challenging field of immunomics”, says Jan Lindeman, DDL’s Chief Executive Officer. “It further expands DDL’s portfolio of tailor-made molecular diagnostic services for our (bio)pharmaceutical customers. ENPICOM’s expertise and know-how are highly complementary to DDL’s expertise and we are confident that this partnership will offer a unique opportunity to accelerate immunotherapy discovery and development.”
Jos Lunenberg, ENPICOM’s Chief Executive Officer comments: “Last month we launched our IGX platform to manage, store, analyze, visualize and interpret immune repertoire sequencing data from T and B cell receptors. Now we are also able to support organizations lacking the specific expertise or capabilities to perform repertoire sequencing with our smart new analysis and visualization methods. In DDL we have found a highly respected business partner with an excellent track record as molecular diagnostic testing laboratory for the biopharmaceutical industry. I’m very pleased to be able to go public with this partnership today.” He continues: “There are a few other kits and services for this specific form of targeted sequencing available in the market, but none or very limited data analysis is included. Clonality analysis is just a first step to bridge the existing gap between producing raw sequencing data and obtaining important insights from the data. So, in essence, we start where others stop.”
The European Lead Factory, Europe’s largest collaborative drug discovery platform, continues its success story
InnovatieklimaatThe European Lead Factory (ELF) secured a total project budget of EUR 36.5 million under the second framework of the Innovative Medicines Initiative (IMI). Over the next 5 years, 20 partners in 7 countries will push forward the transformation of potential drug targets to new medicines in the new project ESCulab (European Screening Centre: unique library for attractive biology) under the European Lead Factory brand.
Jon de Vlieger, coordinator of the ESCulab consortium at Lygature, commented: “It’s truly exciting to continue the onboarding of new and innovative proposals for screening and provide high quality starting points for drug discovery to academics and SMEs throughout Europe. In an effort to broaden our scope we are not only looking for target-based approaches, but now also enable phenotypic screens.”
Proven platform for smart new ideas
Universities, research organisations and SMEs have a diverse range of potential drug targets but cannot easily access suitable compound libraries and screening facilities. Pharmaceutical companies need access to high quality targets to bring innovative therapies to the patient. The European Lead Factory combines the large high-quality compound libraries derived from the pharmaceutical industry and the Public Compound Collection and Europe’s leading screening facility with the innovative targets held by academic organisations in a public-private partnership. This offers an ideal platform to translate early-stage fundamental biological research into credible and investable starting points for drug discovery campaigns.
Stefan Jaroch, project lead of Bayer AG, confirms that: “the European Lead Factory has clearly shown how crowd sourcing and collective intelligence can indeed advance biological concepts into drug discovery projects that benefit academia, industry, society and ultimately patients.”
New ideas + New partners = New chances
Over the next five years,the European Lead Factory will initiate 185 new drug discovery projects by screening medically relevant drug targets from European researchers, small and medium-sized enterprises and pharmaceutical industry against the ELF library of 550,000 unique chemical compounds.
The successful concept of the European Lead Factory has already encouraged additional private partners to join. The ESCulab Project welcomes the two pharmaceutical companies Servier and Grünenthal as well as the Medicines for Malaria Venture (MMV), the leading product development partnership in the field of antimalarial drug research and development.
Tim Wells, Chief Scientific Officer at MMV, adds: “We are thrilled to participate as ESCulab and the European Lead Factory represent a chance for new partnerships for MMV, both with the EU through IMI and with a variety of highly creative companies. These partnerships give access to a novel, high quality chemical library that we believe is important to be screened against high priority malaria targets.”
Source: European Lead Factory