Een tas van echt leer, zonder dat daarvoor dieren gedood zijn. Hoe cool zou dat zijn? Nog even en dan is het zover. Dan kun je zelf je tas, jas of schoenen ontwerpen en laten maken in het lab. Qorium, een biotech bedrijf gevestigd op de Brightlands Maastricht Campus, is het gelukt leer te kweken uit huidcellen van runderen. Eerst vermeerderen ze de cellen, fibroblasten, in een bioreactor. Daarna vormen de fibroblasten laagjes in een weefselweek. De cellen leggen zelf verbindingen tijdens de weefselkweek, waardoor er leer ontstaat. Voordeel is niet alleen het voorkomen van dierenleed, het proces om leer te maken is ook nog eens veel duurzamer. De verwerking vergt minder chemicaliën en minder water. Een fantastische ontwikkeling die HollandBIO van harte toejuicht!
https://www.hollandbio.nl/wp-content/uploads/2018/06/kweekleer.jpg484727hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-19 15:37:322018-06-19 15:45:41Na kweekvlees nu ook kweekleer← #nieuws
Over 18.000 attendees from all over the world joined the 25th anniversary of BIO International Convention in Boston two weeks ago. The conference saw a stunning number of 50.000 partnering meetings, and was awarded the Guinness World Record for “The Largest Business Partnering Event”. A perfect birthday gift for the organization. With a well-visited Health~Holland Pavilion and a strong delegation of 130 Dutch biotech entrepreneurs, this year’s BIO at the epicentre of biotech proved to be a fruitful edition for the Dutch too.
Orange living room
One of the eye-catchers of the exhibition floor was the Health~Holland Pavilion. A not-to-miss orange landmark in a sea of country pavilions, featuring the Dutch LSH strengths, coffee and more. The Pavilion served as the central information point for international visitors with questions regarding the Dutch business and innovation climate and the national life sciences & health sector. In addition, the living room-like pavilion offered the Dutch conference participants a welcoming base to meet business partners.
Place to be
On Tuesday, the Holland Hospitality Reception was the exhibition floor’s best-visited happy hours. In a convincing and hilarious quiz-style speech, Hans Schikan introduced the Dutch Strength to the eager crowd. His take-home message: the Netherlands is the place to be for life sciences companies. After the traditional raffle, the Loyens & Loeff bike was won by a very lucky man from Sweden. For all disappointed participants: rest assured, there will be a new bike to win at BIO 2019 in Philadelphia.
Global biotech hotspot
During Wednesday’s Global Innovation Hub, HollandBIO’s Annemiek Verkamman explained why the Netherlands has Europe’s most attractive business climate for biopharmaceutical companies. With the relocation of the EMA, the Netherlands has what it takes to evolve into a true global biotech hotspot.
https://www.hollandbio.nl/wp-content/uploads/2018/06/IMG_1291-1.jpg15362048hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-19 10:51:222018-06-19 11:57:12Biotech at its best at BIO 2018 in Boston← #nieuws
Argenx has the first data on a new subcutaneous formulation of its rare autoimmune disease drug efgartigimod that could make it a much more patient-friendly prospect.
The company already has an intravenous formulation of efgartigimod (ARGX-113) in trials for myasthenia gravis (MG), immune thrombocytopenia (ITP) and pemphigus vulgaris (PV), all of which are characterized by high levels of pathogenic immunoglobulin G antibodies. Now, Argenx has data showing that its subcutaneous formulation behaves in the same way as the IV version in the body, and seems to be equally effective at depleting IgG levels.
The results raise the hope that the antibody fragment-based drug could be delivered via an IV loading dose to begin with, and then given by simple injection—potentially by the patient at home—as a maintenance therapy.
That would provide “flexible dosing options and greater convenience for patients with severe auto-immune conditions requiring long-term treatment,” according to Argenx’s recently appointed COO Keith Woods, who said the new formulation has now been added to its product pipeline. “With an SC formulation, we also plan to explore the potential for an off-the-shelf delivery system using a fine bore needle size to allow patients additional optionality around administration,” he added.
The Belgian-Dutch biotech has been on a roll over the last few months, completing an impressive $115 million IPO on the Nasdaq, following that up with a $231 million additional offering as 2017 drew to a close, and reporting positive results for IV efgartigimod in MG. If approved, it would compete with big-selling drugs such as Alexion’s $3 billion blockbuster Soliris (eculizumab) in MG, but with a differentiated mechanism of action.
In that study, the drug achieved a clinically significant improvement in symptoms (muscle weakness) in 75% of patients, compared to 25% of the placebo group. Last week, the biotech said it had completed an end-of-study meeting with the FDA and is designing a pivotal, phase 3 trial that should get underway before the end of the year. And it’s also waiting for readouts from phase 2 trials of efgartigimod in ITP and PV in the second half.
Argenx also has a second R&D track focusing on cancer, headed by CD70 antagonist ARG-110 for patients newly diagnosed with acute myeloid leukemia (AML) which has progressed to phase 2 as a combination with widely used AML chemotherapeutic drug azacytidine, phase 1 candidate ARGX-111 for cancer metastases, and AbbVie-partnered cancer immunotherapy ARGX-115.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-14 15:05:582018-06-19 15:09:19Argenx boosts pipeline with new version of lead drug← #nieuws
In a lab at a U.K. research park, researchers from a startup called Tropic Biosciences are using CRISPR to create a better banana. The startup, which is also using gene editing to improve coffee, believes that technology could help save the fruit. Today, the company announced that it raised $10 million to commercialize its varieties of both coffee and bananas.
Both coffee and bananas are an important part of the food market–bananas are a staple food in some areas and the bestselling fruit in the world after tomatoes, and coffee is the most-consumed drink after water. Both are a major source of jobs; around 25 million people globally grow coffee. “If you look at the impacts on livelihoods, then it really is a very critical crop for some of the poorest people globally,” says Gilad Gershon, CEO of Tropic Biosciences. Both crops also face serious challenges.
Bananas are at risk from disease. The Cavendish banana, the variety now common in grocery stores after a fungus decimated a tastier variety, is now at risk from a new strain of the fungus that can quickly spread and kill plants. Another lethal bacterial infection, which is spreading in East Africa, causes bananas to rot. Because bananas are created by cloning other trees–a field of banana trees is basically identical genetically–if one tree is susceptible, the rest will be too.
“That creates a lot of exposure for the industry because a disease that kills one banana can wipe out the entire industry,” says Gershon. “A beetle that attacks one banana tree can attack all of them.”
Because the plants are cloned rather than bred, there’s no variation. “What you have is pretty much what you’re stuck with,” he says. “Because traditional breeding is not an option, gene editing makes a lot of sense, because the only way you can change the banana now is through genetics. If we don’t [take] this type of role and save the banana, I’m not sure there’s any other way to do it.” If bananas are more disease resistant, the industry can reduce its use of agrochemicals; right now, Gershon says, the industry spends roughly a quarter of production costs on fungicide.
The team is also working on editing bananas to help the very perishable fruit survive longer as it’s delivered to consumers. “Using gene editing in a non-GMO way, you can extend the shelf life of bananas,” says Gershon. “And by extending the shelf life you can significantly reduce waste.” CRISPR allows them to precisely edit DNA by, for example, snipping out a tiny portion of the genetic code, along with other advanced gene editing techniques. It compares these to a faster version of natural selection or traditional breeding; unlike some older GMO techniques, no foreign DNA ends up in the food.
For coffee, the startup has already successfully genetically edited a variety of bean that is naturally decaffeinated. Right now, producers typically remove caffeine through a process that involves soaking beans and steaming them. “The problem is that it’s a costly and relatively aggressive process,” Gershon says. The process can impact both flavor and nutrition. “If you grow the beans without the caffeine or with a lower amount of caffeine to begin with, then you can achieve an end product that is a lot closer in taste to normal coffee, and you can maintain a larger content of the very healthy compounds that are naturally found in coffee.”
With the new round of funding, the company, which was founded in 2016, will begin testing its new varieties globally. It will also help drive the regulatory approval process–as with other genetically edited foods, there is likely to be some consumer resistance (though CRISPR-edited foods are already available in the U.S.). The company will also explore additional possible foods to work on. In the past, companies using genetic modification have focused on major row crops like corn and soy. Tropic Biosciences wants to use advanced gene editing to focus on foods that have seen less innovation, but where there is an opportunity to make a meaningful impact.
At some point, it’s possible that companies that genetically engineer food might develop entirely new products–perhaps even a caffeinated banana, for example–but Tropic Biosciences plans to focus on “developing traits that really make sense to people, traits we can communicate easily,” Gershon says. “A lot of people drink decaffeinated coffee globally. We can produce something that they’re already drinking but it’s going to taste better, be healthier. It makes sense to us.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-13 15:35:452018-06-26 15:37:33This startup wants to save the banana by editing its genes← #nieuws
ProQR announced a collaboration with nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF). ProQR is a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases. Under the agreement, EBRP and EBMRF will provide up to approximately $5 million in matching funding to ProQR under a venture philanthropy model. The funding will be used to develop QR-313 for patients with dystrophic epidermolysis bullosa (DEB) caused by mutations in exon 73 of the COL7A1 gene.
“The EB Research Partnership and EB Medical Research Foundation are exceptional organizations and we look forward to collaborate on our mission to develop a life changing treatment for patients with this devastating disease,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “With the funding from these partners, we plan to accelerate the development of QR-313 for the treatment of DEB. And if we are successful with QR-313, we believe there is potential to expand this approach of exon skipping into other mutations that cause DEB and help even more patients.”
DEB is a rare debilitating skin disease caused by the absence of a protein called collagen type VII (C7). Symptoms are present from birth and include easy blistering of the skin, poorly healing wounds, skin infections and, in adulthood, some patients develop very aggressive forms of skin cancer. There are currently no approved treatment options for DEB. The WINGS trial, a first-in-human Phase 1/2 clinical trial of QR-313 in patients with DEB, is expected to initiate enrollment of patients in the first half of 2018. The trial is expected to report interim proof of mechanism results later this year, with full results expected in 2019.
“DEB has a huge impact on patients’ quality of life and there are currently no approved disease modifying treatment options beyond wound care,” said Alex Silver, founder and Chairman of EBRP and Paul Joseph, CFO of EBMRF. “Our organizations are excited to partner with the ProQR team, in the hopes that the development of QR-313 will provide a much-needed treatment option to this community.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-12 18:23:042018-06-12 18:23:045 Million for ProQR to develop QR-313 as treatment of Dystrophic Epidermolysis Bullosa← #nieuws
Chemisch technologiebedrijf Avantium is begonnen met de bouw van een nieuwe demonstratiefabriek in Nederland voor de productie van biologische ethyleenglycol uit hernieuwbare suikers. De faciliteit moet in 2019 in gebruik worden en krijgt maximaal twintig werknemers.
Volgens Avantium maakt de nieuwe fabriek deel uit van een eerder aangekondigde investering van 15 miljoen tot 20 miljoen euro in nieuwe technologieën. Ethyleenglycol is een belangrijk bestanddeel voor plastic producten. Avantium stelt dat op biologische basis geproduceerde ethyleenglycol een sterk groeipotentieel heeft.
Verder gaf Avantium aan dat zijn bioraffinaderij in Delfzijl bijna klaar is. In die fabriek worden houtsnippers met behulp van zoutzuur omgezet in glucose en lignine.
Het Zorginstituut publiceert vandaag de update van de Horizonscan Geneesmiddelen. De scan heeft als doel om patiënten, behandelaars, ziekenhuizen, zorgverzekeraars en overheidsorganen vroegtijdig op de hoogte te stellen van de ontwikkelingen op het gebied van innovatieve geneesmiddelen. De Horizonscan bevat een overzicht van geneesmiddelen of indicatie-uitbreidingen die de komende twee jaar verwacht worden. Diverse HollandBIO leden leverden informatie aan voor een representatieve scan. Ook zijn, mede op aangeven van HollandBIO, verbeteringen aangebracht in de nieuwe editie van de Horizonscan.
Het Zorginstituut stelt de Horizonscan Geneesmiddelen samen op basis van input van “(Horizon)scanners”, werkgroepen van experts en geneesmiddelenontwikkelaars. Die laatsten kregen niet alleen de kans om in een vroeg stadium data aan te leveren, maar deze ook voor publicatie te actualiseren. Dit komt de voorspellende waarde van de Horizonscan ten goede.
Het Zorginstituut heeft ten opzichte van de vorige scan, die begin dit jaar verscheen, een aantal verbeteringen doorgevoerd. Zo heeft het maximaal patiëntvolume plaatsgemaakt voor een onderbouwd realistisch patiëntvolume. Dit leidt tot een nauwkeurigere schatting van de behandelkosten. Daarnaast maakt de nieuwe scan ook melding van de ontwikkelfase waarin de geneesmiddelen zich bevinden. Het zijn twee aanpassingen waar HollandBIO erg blij mee is.
Hoewel deze update net is afgerond, start het Zorginstituut eind juni weer met de uitvraag voor de volgende Horizonscan, die december 2018, januari 2019 live gaat. Die scan wordt nog vollediger: naast nieuwe geneesmiddelen en toepassingen zal deze ook generieke geneesmiddelen en biosimilars in kaart brengen. HollandBIO roept haar leden op om ook hier weer een bijdrage aan te leveren.
Benieuwd naar de Horizonscan?
Bekijk dan de website van de Horizonscan geneesmiddelen. We horen graag of de gegevens van jouw bedrijf er goed in staan en of je mogelijkheden tot verdere verbetering van de scan ziet. https://www.horizonscangeneesmiddelen.nl/
Het vertrouwen van internationale investeerders in het Nederlandse vestigingsklimaat is het afgelopen jaar verder toegenomen. Dat is een van de conclusies van de EY barometer Nederlands vestigingsklimaat 2018. Daarbovenop bevestigt een recent verschenen rapport van KPMG de rol van Nederland als koploper op het gebied van life sciences. De rapporten maken wederom duidelijk dat het vestigingsklimaat in Nederland in de lift zit.
In het KPMG-rapport over de aantrekkelijkheid van vestigingslocaties voor life sciences bedrijven scoort Nederland op alle vlakken in de Europese (sub)top. Het aantal bedrijven in Nederland is hoog, we hebben een dijk van een pijplijn en prijken in de top van het Europese Innovatie Scorebord. Nederland eindigt zelfs op de eerste plaats als het gaat om lange termijn investeringen. Hierbij kijkt KPMG naar breed scala aan indicatoren als productiviteit, educatie, internationalisering en transport.
Nederland blijft overtuigen
De belangrijkste conclusie van EYs barometer is dat Nederland blijft overtuigen als investeringsland. Na recordjaar 2016 ligt het niveau van buitenlandse investeringen in 2017 weliswaar lager, maar alsnog noteert Nederland een mooie vierde plaats, achter het Verenigd Koninkrijk, Duitsland en Frankrijk. Investeerders waarderen Nederland om de kwaliteit van leven, de logistiek en het opleidingsniveau, maar geven tegelijk aan dat het behoud van het goede fiscale klimaat een aandachtspunt is. De totale investeringen in R&D namen ten opzichte van 2016 licht toe. EY verwacht bovendien dat, mede door de komst van het Europese Medicijn Agentschap, de investeringen in R&D de komende jaren verder toenemen.
Beste van Europa
Het aantrekkelijke Nederlandse vestigingsklimaat voor de life sciences stond al in de schijnwerpers door de publicatie van het Health~Holland bidbook eerder dit jaar. Nederland presenteert zich hierin als hét land bij uitstek voor biofarmaceutische bedrijven. Het bidbook helpt buitenlandse bedrijven te overtuigen om een kantoor in Nederland te vestigen of om juist extra te investeren.
HollandBIO maakt werk van een ijzersterk innovatieklimaat en een aantrekkelijk vestigingsklimaat. De drie publicaties samen maken duidelijk dat we in de lift zitten. Nu zijn we niet snel tevreden bij HollandBIO, wat ons betreft kan het nog veel beter. Op Europees niveau doet Nederland mee in de top, maar als we doorzetten is ook de wereldtop haalbaar. HollandBIO zal niet rusten voordat ook dat uit de lijstjes blijkt.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2018-06-12 17:16:092018-06-12 17:16:09Nederlandse vestigingsklimaat in de lift← #nieuws
Chemelot Ventures, LIOF and the founders will invest €500,000 in Vertoro, a company located on the Brightlands Chemelot Campus in Geleen. Vertoro has developed a biobased process to derive bio-oil from waste product lignin (biomass) and will use the funds to scale up production. As bio-oil is made from biomass, it helps to reduce CO2 emissions and paves the way for the production of sustainable products.
Vertoro: developer and producer of bio-oil
Vertoro, which is located on the Brightlands Chemelot Campus in Geleen, is the outcome of a project by Chemelot InSciTe, a public-private partnership. An initial upscaling of the bio-oil has already been completed as part of this project. Vertoro and InSciTe are now preparing to construct a pilot plant on the Brightlands Chemelot Campus in Geleen. In early 2019, Vertoro expects to be the first company in the world to produce a barrel of bio-oil from lignin. The company then aims to build a production facility at Chemelot in 2022 with an annual production capacity of 10,000 tonnes of bio-oil.
Vertoro, founded in 2017, was a participant in the Brightlands Innovation Factory’s start-up programme in the same year. This is part of the reason that the company is now ready for the current Seed Financing by Chemelot Ventures and LIOF.
Vertoro derives bio-oil from the raw material lignin is a wood polymer found in the cell wall of plants and trees. Currently, lignin is a by-product in the production of paper and biofuels (bioethanol) and burnt as a waste stream. Vertoro and its development partners have succeeded in developing a technology to convert lignin into valuable bio-oil through an innovative production process that can make an important contribution towards reducing CO2 emissions.
Increasing demand for bio-oil
Global demand for bio-oil is increasing, particularly from industrial companies looking to produce sustainable products from biomass. Similar to fossil-based oil, Vertoro’s bio-oil can be used as a basic raw material for applications in energy (i.e. additive for biodiesel), chemicals, and materials (i.e. bio-phenol for plastics).
Suppliers of lignin also have a keen interest in Vertoro’s production process, as this is the first time that there has been an efficient process for converting the low-grade lignin into a raw material for high-grade products. Following the local upscaling, Vertoro – literally ‘green gold’ – will license out the technology and market bio-oil worldwide.
Michael Boot CEO of Vertoro and Panos Kouris CTO of Vertoro: ‘Our bio-oil provides a fantastic basis for producing clean energy and sustainable chemicals and materials. We can see that Brightlands brings together all these success factors.’
Alexion and Complement Pharma announced a partnership to co-develop the preclinical C6 complement inhibitor CP010 for neurodegenerative disorders. C6 inhibition prevents the formation of the destructive membrane attack complex (MAC), a complex of terminal complement proteins that mediate cellular injury following complement activation, and has the potential to treat a variety of central nervous system disorders. Under the terms of the agreement, Alexion will provide Complement Pharma with up to € 14 million in milestone-dependent payments through Phase 1b development. The agreement also provides Alexion with the option to acquire Complement Pharma during the term of the agreement.
“Alexion has been a pioneer in complement biology since the development of Soliris, the world’s first complement inhibitor, which has demonstrated the significant impact that C5 inhibition can have on several diseases caused by uncontrolled complement activation,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “This collaboration provides an exciting opportunity to expand on our more than two decades of complement expertise to potentially treat additional diseases by targeting C6, a different component of the complement system.”
“Our understanding of the role of complement in many disorders continues to increase and many neurodegenerative diseases show activation of the complement system, even in early stages of disease,” said Frank Baas, M.D., Ph.D., Chief Scientific Officer at Complement Pharma. “We believe that C6 inhibition has the potential to treat multiple neurodegenerative disorders, and we look forward to working with Alexion, a partner with a worldwide leading position in complement biology, to realize the possibilities of this approach.”
Under the terms of the agreement, Alexion and Complement Pharma will collaborate on the development program for CP010. Complement Pharma will be responsible for conducting preclinical and Phase 1 studies and for manufacturing CP010. The phased agreement extends through the completion of Phase 1b development. Alexion has the option to acquire Complement Pharma during the term of the agreement.
CP010 is a humanized monoclonal antibody in preclinical development that binds to C6 in circulation to inhibit its function throughout the body by preventing MAC formation in both the periphery and the central nervous system. An end product of the activated complement cascade, MAC has been shown to play a role in neurodegeneration. CP010 has demonstrated C6 inhibition in vitro and in vivo; C6 inhibition prevents the formation of the MAC following complement activation which may allow treatment of a variety of central nervous system disorders.
Na kweekvlees nu ook kweekleer
Voedsel en materialenEen tas van echt leer, zonder dat daarvoor dieren gedood zijn. Hoe cool zou dat zijn? Nog even en dan is het zover. Dan kun je zelf je tas, jas of schoenen ontwerpen en laten maken in het lab. Qorium, een biotech bedrijf gevestigd op de Brightlands Maastricht Campus, is het gelukt leer te kweken uit huidcellen van runderen. Eerst vermeerderen ze de cellen, fibroblasten, in een bioreactor. Daarna vormen de fibroblasten laagjes in een weefselweek. De cellen leggen zelf verbindingen tijdens de weefselkweek, waardoor er leer ontstaat. Voordeel is niet alleen het voorkomen van dierenleed, het proces om leer te maken is ook nog eens veel duurzamer. De verwerking vergt minder chemicaliën en minder water. Een fantastische ontwikkeling die HollandBIO van harte toejuicht!
Bron: technisch weekblad
Biotech at its best at BIO 2018 in Boston
HollandbioOver 18.000 attendees from all over the world joined the 25th anniversary of BIO International Convention in Boston two weeks ago. The conference saw a stunning number of 50.000 partnering meetings, and was awarded the Guinness World Record for “The Largest Business Partnering Event”. A perfect birthday gift for the organization. With a well-visited Health~Holland Pavilion and a strong delegation of 130 Dutch biotech entrepreneurs, this year’s BIO at the epicentre of biotech proved to be a fruitful edition for the Dutch too.
Orange living room
One of the eye-catchers of the exhibition floor was the Health~Holland Pavilion. A not-to-miss orange landmark in a sea of country pavilions, featuring the Dutch LSH strengths, coffee and more. The Pavilion served as the central information point for international visitors with questions regarding the Dutch business and innovation climate and the national life sciences & health sector. In addition, the living room-like pavilion offered the Dutch conference participants a welcoming base to meet business partners.
Place to be
On Tuesday, the Holland Hospitality Reception was the exhibition floor’s best-visited happy hours. In a convincing and hilarious quiz-style speech, Hans Schikan introduced the Dutch Strength to the eager crowd. His take-home message: the Netherlands is the place to be for life sciences companies. After the traditional raffle, the Loyens & Loeff bike was won by a very lucky man from Sweden. For all disappointed participants: rest assured, there will be a new bike to win at BIO 2019 in Philadelphia.
Global biotech hotspot
During Wednesday’s Global Innovation Hub, HollandBIO’s Annemiek Verkamman explained why the Netherlands has Europe’s most attractive business climate for biopharmaceutical companies. With the relocation of the EMA, the Netherlands has what it takes to evolve into a true global biotech hotspot.
Argenx boosts pipeline with new version of lead drug
GezondheidArgenx has the first data on a new subcutaneous formulation of its rare autoimmune disease drug efgartigimod that could make it a much more patient-friendly prospect.
The company already has an intravenous formulation of efgartigimod (ARGX-113) in trials for myasthenia gravis (MG), immune thrombocytopenia (ITP) and pemphigus vulgaris (PV), all of which are characterized by high levels of pathogenic immunoglobulin G antibodies. Now, Argenx has data showing that its subcutaneous formulation behaves in the same way as the IV version in the body, and seems to be equally effective at depleting IgG levels.
The results raise the hope that the antibody fragment-based drug could be delivered via an IV loading dose to begin with, and then given by simple injection—potentially by the patient at home—as a maintenance therapy.
That would provide “flexible dosing options and greater convenience for patients with severe auto-immune conditions requiring long-term treatment,” according to Argenx’s recently appointed COO Keith Woods, who said the new formulation has now been added to its product pipeline. “With an SC formulation, we also plan to explore the potential for an off-the-shelf delivery system using a fine bore needle size to allow patients additional optionality around administration,” he added.
The Belgian-Dutch biotech has been on a roll over the last few months, completing an impressive $115 million IPO on the Nasdaq, following that up with a $231 million additional offering as 2017 drew to a close, and reporting positive results for IV efgartigimod in MG. If approved, it would compete with big-selling drugs such as Alexion’s $3 billion blockbuster Soliris (eculizumab) in MG, but with a differentiated mechanism of action.
In that study, the drug achieved a clinically significant improvement in symptoms (muscle weakness) in 75% of patients, compared to 25% of the placebo group. Last week, the biotech said it had completed an end-of-study meeting with the FDA and is designing a pivotal, phase 3 trial that should get underway before the end of the year. And it’s also waiting for readouts from phase 2 trials of efgartigimod in ITP and PV in the second half.
Argenx also has a second R&D track focusing on cancer, headed by CD70 antagonist ARG-110 for patients newly diagnosed with acute myeloid leukemia (AML) which has progressed to phase 2 as a combination with widely used AML chemotherapeutic drug azacytidine, phase 1 candidate ARGX-111 for cancer metastases, and AbbVie-partnered cancer immunotherapy ARGX-115.
Source: FierceBiotech
This startup wants to save the banana by editing its genes
Voedsel en materialenIn a lab at a U.K. research park, researchers from a startup called Tropic Biosciences are using CRISPR to create a better banana. The startup, which is also using gene editing to improve coffee, believes that technology could help save the fruit. Today, the company announced that it raised $10 million to commercialize its varieties of both coffee and bananas.
Both coffee and bananas are an important part of the food market–bananas are a staple food in some areas and the bestselling fruit in the world after tomatoes, and coffee is the most-consumed drink after water. Both are a major source of jobs; around 25 million people globally grow coffee. “If you look at the impacts on livelihoods, then it really is a very critical crop for some of the poorest people globally,” says Gilad Gershon, CEO of Tropic Biosciences. Both crops also face serious challenges.
Bananas are at risk from disease. The Cavendish banana, the variety now common in grocery stores after a fungus decimated a tastier variety, is now at risk from a new strain of the fungus that can quickly spread and kill plants. Another lethal bacterial infection, which is spreading in East Africa, causes bananas to rot. Because bananas are created by cloning other trees–a field of banana trees is basically identical genetically–if one tree is susceptible, the rest will be too.
“That creates a lot of exposure for the industry because a disease that kills one banana can wipe out the entire industry,” says Gershon. “A beetle that attacks one banana tree can attack all of them.”
Because the plants are cloned rather than bred, there’s no variation. “What you have is pretty much what you’re stuck with,” he says. “Because traditional breeding is not an option, gene editing makes a lot of sense, because the only way you can change the banana now is through genetics. If we don’t [take] this type of role and save the banana, I’m not sure there’s any other way to do it.” If bananas are more disease resistant, the industry can reduce its use of agrochemicals; right now, Gershon says, the industry spends roughly a quarter of production costs on fungicide.
The team is also working on editing bananas to help the very perishable fruit survive longer as it’s delivered to consumers. “Using gene editing in a non-GMO way, you can extend the shelf life of bananas,” says Gershon. “And by extending the shelf life you can significantly reduce waste.” CRISPR allows them to precisely edit DNA by, for example, snipping out a tiny portion of the genetic code, along with other advanced gene editing techniques. It compares these to a faster version of natural selection or traditional breeding; unlike some older GMO techniques, no foreign DNA ends up in the food.
For coffee, the startup has already successfully genetically edited a variety of bean that is naturally decaffeinated. Right now, producers typically remove caffeine through a process that involves soaking beans and steaming them. “The problem is that it’s a costly and relatively aggressive process,” Gershon says. The process can impact both flavor and nutrition. “If you grow the beans without the caffeine or with a lower amount of caffeine to begin with, then you can achieve an end product that is a lot closer in taste to normal coffee, and you can maintain a larger content of the very healthy compounds that are naturally found in coffee.”
With the new round of funding, the company, which was founded in 2016, will begin testing its new varieties globally. It will also help drive the regulatory approval process–as with other genetically edited foods, there is likely to be some consumer resistance (though CRISPR-edited foods are already available in the U.S.). The company will also explore additional possible foods to work on. In the past, companies using genetic modification have focused on major row crops like corn and soy. Tropic Biosciences wants to use advanced gene editing to focus on foods that have seen less innovation, but where there is an opportunity to make a meaningful impact.
At some point, it’s possible that companies that genetically engineer food might develop entirely new products–perhaps even a caffeinated banana, for example–but Tropic Biosciences plans to focus on “developing traits that really make sense to people, traits we can communicate easily,” Gershon says. “A lot of people drink decaffeinated coffee globally. We can produce something that they’re already drinking but it’s going to taste better, be healthier. It makes sense to us.”
Source: FastCompany
5 Million for ProQR to develop QR-313 as treatment of Dystrophic Epidermolysis Bullosa
Gezondheid, InnovatieklimaatProQR announced a collaboration with nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF). ProQR is a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases. Under the agreement, EBRP and EBMRF will provide up to approximately $5 million in matching funding to ProQR under a venture philanthropy model. The funding will be used to develop QR-313 for patients with dystrophic epidermolysis bullosa (DEB) caused by mutations in exon 73 of the COL7A1 gene.
“The EB Research Partnership and EB Medical Research Foundation are exceptional organizations and we look forward to collaborate on our mission to develop a life changing treatment for patients with this devastating disease,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “With the funding from these partners, we plan to accelerate the development of QR-313 for the treatment of DEB. And if we are successful with QR-313, we believe there is potential to expand this approach of exon skipping into other mutations that cause DEB and help even more patients.”
DEB is a rare debilitating skin disease caused by the absence of a protein called collagen type VII (C7). Symptoms are present from birth and include easy blistering of the skin, poorly healing wounds, skin infections and, in adulthood, some patients develop very aggressive forms of skin cancer. There are currently no approved treatment options for DEB. The WINGS trial, a first-in-human Phase 1/2 clinical trial of QR-313 in patients with DEB, is expected to initiate enrollment of patients in the first half of 2018. The trial is expected to report interim proof of mechanism results later this year, with full results expected in 2019.
“DEB has a huge impact on patients’ quality of life and there are currently no approved disease modifying treatment options beyond wound care,” said Alex Silver, founder and Chairman of EBRP and Paul Joseph, CFO of EBMRF. “Our organizations are excited to partner with the ProQR team, in the hopes that the development of QR-313 will provide a much-needed treatment option to this community.”
Source: ProQR
Avantium begint bouw demonstratiefabriek
Voedsel en materialen, InnovatieklimaatChemisch technologiebedrijf Avantium is begonnen met de bouw van een nieuwe demonstratiefabriek in Nederland voor de productie van biologische ethyleenglycol uit hernieuwbare suikers. De faciliteit moet in 2019 in gebruik worden en krijgt maximaal twintig werknemers.
Volgens Avantium maakt de nieuwe fabriek deel uit van een eerder aangekondigde investering van 15 miljoen tot 20 miljoen euro in nieuwe technologieën. Ethyleenglycol is een belangrijk bestanddeel voor plastic producten. Avantium stelt dat op biologische basis geproduceerde ethyleenglycol een sterk groeipotentieel heeft.
Verder gaf Avantium aan dat zijn bioraffinaderij in Delfzijl bijna klaar is. In die fabriek worden houtsnippers met behulp van zoutzuur omgezet in glucose en lignine.
Bron: IEX
Update Horizonscan Geneesmiddelen Zorginstituut
Hollandbio, GezondheidHet Zorginstituut publiceert vandaag de update van de Horizonscan Geneesmiddelen. De scan heeft als doel om patiënten, behandelaars, ziekenhuizen, zorgverzekeraars en overheidsorganen vroegtijdig op de hoogte te stellen van de ontwikkelingen op het gebied van innovatieve geneesmiddelen. De Horizonscan bevat een overzicht van geneesmiddelen of indicatie-uitbreidingen die de komende twee jaar verwacht worden. Diverse HollandBIO leden leverden informatie aan voor een representatieve scan. Ook zijn, mede op aangeven van HollandBIO, verbeteringen aangebracht in de nieuwe editie van de Horizonscan.
Het Zorginstituut stelt de Horizonscan Geneesmiddelen samen op basis van input van “(Horizon)scanners”, werkgroepen van experts en geneesmiddelenontwikkelaars. Die laatsten kregen niet alleen de kans om in een vroeg stadium data aan te leveren, maar deze ook voor publicatie te actualiseren. Dit komt de voorspellende waarde van de Horizonscan ten goede.
Het Zorginstituut heeft ten opzichte van de vorige scan, die begin dit jaar verscheen, een aantal verbeteringen doorgevoerd. Zo heeft het maximaal patiëntvolume plaatsgemaakt voor een onderbouwd realistisch patiëntvolume. Dit leidt tot een nauwkeurigere schatting van de behandelkosten. Daarnaast maakt de nieuwe scan ook melding van de ontwikkelfase waarin de geneesmiddelen zich bevinden. Het zijn twee aanpassingen waar HollandBIO erg blij mee is.
Hoewel deze update net is afgerond, start het Zorginstituut eind juni weer met de uitvraag voor de volgende Horizonscan, die december 2018, januari 2019 live gaat. Die scan wordt nog vollediger: naast nieuwe geneesmiddelen en toepassingen zal deze ook generieke geneesmiddelen en biosimilars in kaart brengen. HollandBIO roept haar leden op om ook hier weer een bijdrage aan te leveren.
Benieuwd naar de Horizonscan?
Bekijk dan de website van de Horizonscan geneesmiddelen. We horen graag of de gegevens van jouw bedrijf er goed in staan en of je mogelijkheden tot verdere verbetering van de scan ziet. https://www.horizonscangeneesmiddelen.nl/
Nederlandse vestigingsklimaat in de lift
Hollandbio, InnovatieklimaatHet vertrouwen van internationale investeerders in het Nederlandse vestigingsklimaat is het afgelopen jaar verder toegenomen. Dat is een van de conclusies van de EY barometer Nederlands vestigingsklimaat 2018. Daarbovenop bevestigt een recent verschenen rapport van KPMG de rol van Nederland als koploper op het gebied van life sciences. De rapporten maken wederom duidelijk dat het vestigingsklimaat in Nederland in de lift zit.
In het KPMG-rapport over de aantrekkelijkheid van vestigingslocaties voor life sciences bedrijven scoort Nederland op alle vlakken in de Europese (sub)top. Het aantal bedrijven in Nederland is hoog, we hebben een dijk van een pijplijn en prijken in de top van het Europese Innovatie Scorebord. Nederland eindigt zelfs op de eerste plaats als het gaat om lange termijn investeringen. Hierbij kijkt KPMG naar breed scala aan indicatoren als productiviteit, educatie, internationalisering en transport.
Nederland blijft overtuigen
De belangrijkste conclusie van EYs barometer is dat Nederland blijft overtuigen als investeringsland. Na recordjaar 2016 ligt het niveau van buitenlandse investeringen in 2017 weliswaar lager, maar alsnog noteert Nederland een mooie vierde plaats, achter het Verenigd Koninkrijk, Duitsland en Frankrijk. Investeerders waarderen Nederland om de kwaliteit van leven, de logistiek en het opleidingsniveau, maar geven tegelijk aan dat het behoud van het goede fiscale klimaat een aandachtspunt is. De totale investeringen in R&D namen ten opzichte van 2016 licht toe. EY verwacht bovendien dat, mede door de komst van het Europese Medicijn Agentschap, de investeringen in R&D de komende jaren verder toenemen.
Beste van Europa
Het aantrekkelijke Nederlandse vestigingsklimaat voor de life sciences stond al in de schijnwerpers door de publicatie van het Health~Holland bidbook eerder dit jaar. Nederland presenteert zich hierin als hét land bij uitstek voor biofarmaceutische bedrijven. Het bidbook helpt buitenlandse bedrijven te overtuigen om een kantoor in Nederland te vestigen of om juist extra te investeren.
HollandBIO maakt werk van een ijzersterk innovatieklimaat en een aantrekkelijk vestigingsklimaat. De drie publicaties samen maken duidelijk dat we in de lift zitten. Nu zijn we niet snel tevreden bij HollandBIO, wat ons betreft kan het nog veel beter. Op Europees niveau doet Nederland mee in de top, maar als we doorzetten is ook de wereldtop haalbaar. HollandBIO zal niet rusten voordat ook dat uit de lijstjes blijkt.
Lees meer:
Rapport EY: https://www.ey.com/Publication/vwLUAssets/EY-barometer-nederlands-vestigingsklimaat-2018/$FILE/EY-barometer-nederlands-vestigingsklimaat-2018.pdf
KPMG: https://assets.kpmg.com/content/dam/kpmg/ch/pdf/site-selection-for-life-sciences-companies-europe-2018-en.pdf
Bidbook The Netherlands: Europe’s most attractive and innovative biopharmaceutical industry environment: https://www.health-holland.com/internationalisation/bidbook
Vertoro receives Seed Financing from Chemelot Ventures and LIOF
Voedsel en materialen, InnovatieklimaatChemelot Ventures, LIOF and the founders will invest €500,000 in Vertoro, a company located on the Brightlands Chemelot Campus in Geleen. Vertoro has developed a biobased process to derive bio-oil from waste product lignin (biomass) and will use the funds to scale up production. As bio-oil is made from biomass, it helps to reduce CO2 emissions and paves the way for the production of sustainable products.
Vertoro: developer and producer of bio-oil
Vertoro, which is located on the Brightlands Chemelot Campus in Geleen, is the outcome of a project by Chemelot InSciTe, a public-private partnership. An initial upscaling of the bio-oil has already been completed as part of this project. Vertoro and InSciTe are now preparing to construct a pilot plant on the Brightlands Chemelot Campus in Geleen. In early 2019, Vertoro expects to be the first company in the world to produce a barrel of bio-oil from lignin. The company then aims to build a production facility at Chemelot in 2022 with an annual production capacity of 10,000 tonnes of bio-oil.
Vertoro, founded in 2017, was a participant in the Brightlands Innovation Factory’s start-up programme in the same year. This is part of the reason that the company is now ready for the current Seed Financing by Chemelot Ventures and LIOF.
Vertoro derives bio-oil from the raw material lignin is a wood polymer found in the cell wall of plants and trees. Currently, lignin is a by-product in the production of paper and biofuels (bioethanol) and burnt as a waste stream. Vertoro and its development partners have succeeded in developing a technology to convert lignin into valuable bio-oil through an innovative production process that can make an important contribution towards reducing CO2 emissions.
Increasing demand for bio-oil
Global demand for bio-oil is increasing, particularly from industrial companies looking to produce sustainable products from biomass. Similar to fossil-based oil, Vertoro’s bio-oil can be used as a basic raw material for applications in energy (i.e. additive for biodiesel), chemicals, and materials (i.e. bio-phenol for plastics).
Suppliers of lignin also have a keen interest in Vertoro’s production process, as this is the first time that there has been an efficient process for converting the low-grade lignin into a raw material for high-grade products. Following the local upscaling, Vertoro – literally ‘green gold’ – will license out the technology and market bio-oil worldwide.
Michael Boot CEO of Vertoro and Panos Kouris CTO of Vertoro: ‘Our bio-oil provides a fantastic basis for producing clean energy and sustainable chemicals and materials. We can see that Brightlands brings together all these success factors.’
Alexion and Complement Pharma co-develop complement inhibitor for neurodegenerative disorders
Gezondheid, InnovatieklimaatAlexion and Complement Pharma announced a partnership to co-develop the preclinical C6 complement inhibitor CP010 for neurodegenerative disorders. C6 inhibition prevents the formation of the destructive membrane attack complex (MAC), a complex of terminal complement proteins that mediate cellular injury following complement activation, and has the potential to treat a variety of central nervous system disorders. Under the terms of the agreement, Alexion will provide Complement Pharma with up to € 14 million in milestone-dependent payments through Phase 1b development. The agreement also provides Alexion with the option to acquire Complement Pharma during the term of the agreement.
“Alexion has been a pioneer in complement biology since the development of Soliris, the world’s first complement inhibitor, which has demonstrated the significant impact that C5 inhibition can have on several diseases caused by uncontrolled complement activation,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “This collaboration provides an exciting opportunity to expand on our more than two decades of complement expertise to potentially treat additional diseases by targeting C6, a different component of the complement system.”
“Our understanding of the role of complement in many disorders continues to increase and many neurodegenerative diseases show activation of the complement system, even in early stages of disease,” said Frank Baas, M.D., Ph.D., Chief Scientific Officer at Complement Pharma. “We believe that C6 inhibition has the potential to treat multiple neurodegenerative disorders, and we look forward to working with Alexion, a partner with a worldwide leading position in complement biology, to realize the possibilities of this approach.”
Under the terms of the agreement, Alexion and Complement Pharma will collaborate on the development program for CP010. Complement Pharma will be responsible for conducting preclinical and Phase 1 studies and for manufacturing CP010. The phased agreement extends through the completion of Phase 1b development. Alexion has the option to acquire Complement Pharma during the term of the agreement.
CP010 is a humanized monoclonal antibody in preclinical development that binds to C6 in circulation to inhibit its function throughout the body by preventing MAC formation in both the periphery and the central nervous system. An end product of the activated complement cascade, MAC has been shown to play a role in neurodegeneration. CP010 has demonstrated C6 inhibition in vitro and in vivo; C6 inhibition prevents the formation of the MAC following complement activation which may allow treatment of a variety of central nervous system disorders.
Source: Alexion