Simon Groot, een 84-jarige oer-Hollandse zesde generatie zaadveredelaar uit Enkhuizen, ontvangt deze week prestigieuze World Food Prize, ook wel de Nobelprijs voor Voedsel genoemd. Een bijzondere prestatie, die symbool staat voor nog iets veel groters. Want met onze Nederlandse kennis helpen wij de wereld te voeden. In een mooie video legt NOS op 3 uit hoe innovatief Nederland is op voedselgebied, met daarin een glansrol voor de Nederlandse zaadveredeling. Bekijk ‘m hier!
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-15 11:16:132019-10-15 11:16:14Nederlandse zaadveredeling in het zonnetje gezet← #nieuws
IenW minister Cora van Nieuwenhuizen wil op verantwoorde wijze ruimte geven aan innovatieve toepassingen uit de biotech sector die bijdragen aan een duurzame en gezonde samenleving. In haar openingsspeech voor het recente internationale COGEM-symposium over het genetisch bewerken van planten, pleit Van Nieuwenhuizen gepassioneerd voor het grondig moderniseren van de achterhaalde Europese wet- en regelgeving rond genetische modificatie en het versimpelen en verkorten van de nationale procedures in Nederland. De minister is een groot voorstander van een toekomstig systeem met een juiste balans tussen het inschatten van risico’s en het toelaten van innovaties die de wereld een stukje beter maken. HollandBIO kan niet wachten tot het zover is en blijft in de tussentijd meehelpen aan het verwezenlijken van deze toekomst.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-15 11:13:572019-10-15 21:57:07Minister Van Nieuwenhuizen pleit voor toekomstbestendige regelgeving← #nieuws
Nederland is de meest concurrerende economie van Europa volgens de meest recente Global Competitiveness Index van het World Economic Forum (WEF). Zo stelt het rapport onder andere dat‘de Nederlandse economie veel dynamischer is geworden’. Wel moet Nederland, om in top-10 mee te blijven draaien, verder investeren in innovatie en nieuwe technologie. Ook staatssecretaris Mona Keijzer (Economische Zaken en Klimaat) onderkent deze aanbeveling. Zij stelt dat Nederland een antwoord moet vinden op de economische en maatschappelijke uitdagingen die op ons afkomen. En dat investeringen in bijvoorbeeld biotechnologie en kunstmatige intelligentie de sleutel zijn tot het behouden van de Nederlandse toppositie in de toekomst. Lees meer
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-15 11:12:182019-10-15 22:05:57Nederland stijgt naar vierde plek in Global Competitiveness Index← #nieuws
Veelbelovende technologieën zoals CRISPR-Cas en kweekvlees kunnen in potentie de wereld veranderen. Alleen, hoe neem je daar de maatschappij in mee? In een artikel in het FD komt dit vraagstuk aan bod op basis van kweekvlees als centrale casus en de vraag welk verhaal het beste bij zo’n hightech innovatie past. Is dat het frame van technologische en wetenschappelijke vooruitgang, of toch het duurzaamheidsframe? Er is een heel dunne scheidslijn tussen aan de ene kant het door de sector veelgebruikte idyllische, wetenschappelijke verhaal met labjassen en DNA-strengen en aan de andere kant het door critici en NGO’s gecreëerde dystopische toekomstverhaal met ‘Frankenstein Foods’. Beter is het om het verhaal en de beeldvorming daaromheen op te bouwen vanuit de positieve bijdrage die een toepassing levert aan een betere maatschappij. Daarmee is dit ‘groene frame’ een essentieel instrument om in een vroege fase op een positieve wijze de dialoog aan te gaan met het bredere publiek.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-15 11:08:172019-10-15 11:08:19Toepassingen uit nieuwe technologie hebben het juiste verhaal nodig← #nieuws
Xenikos, which develops innovative immunotherapies for treating patients with severe immune disease and post-transplant rejection, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to T-Guard, Xenikos’ flagship product designed to treat steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following allogeneic stem cell transplantation. Xenikos is currently preparing to initiate a U.S. pivotal Phase 3 trial with T-Guard.
The Fast Track program is designed to facilitate the development of new therapies for treating severe medical conditions in order to fill a currently unmet medical need. Fast Track designation allows for early and frequent communications with the FDA, as well as rolling submission of the market application.
“We are delighted that the FDA has given Fast Track designation to T-Guard, as this will support our goal of getting T-Guard to patients as quickly as possible,” said Dr. Ypke van Oosterhout, Chief Executive Officer of Xenikos. “We’re also looking forward to starting our Phase 3 pivotal trial in the U.S. Effective new therapies for treating SR-aGVHD are urgently needed, and we believe that T-Guard, which can rapidly and safely reset the patient’s immune system, has the potential to provide an important new treatment option to patients with this devastating, potentially fatal condition.”
T-Guard: Helping reset the body’s immune system
T-Guard is designed to safely and swiftly reset the body’s immune system in life-threatening T cell‒mediated conditions, including transplant-related rejection, acute solid-organ rejection, and severe autoimmune disease. T Guard consists of a unique combination of toxin-conjugated monoclonal antibodies that target CD3 and CD7 molecules on T cells and NK cells. Preclinical and early clinical testing have shown that T-Guard can specifically identify and eliminate mature T cells and NK cells with minimal treatment-related side effects. Importantly, T-Guard’s action is short-lived, thereby significantly reducing the patient’s vulnerability to opportunistic infections compared to currently available therapies. Xenikos successfully completed a Phase 1/2 study for the second-line treatment of SR-aGVHD in patients following hematopoietic stem cell transplantation (HSCT). The results of this study showed that just one week of T-Guard treatment triggered a strong clinical response and doubled the 6-month overall survival rate. These results were published in the peer-reviewed journal Biology of Blood and Marrow Transplantation. A U.S. Phase 3 registration trial involving patients with SR-aGVHD following allogeneic stem cell transplantation will begin soon, and T-Guard has been granted Fast Track designation by the FDA, as well as Orphan Drug Designation status in both the EU and the U.S.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-14 16:29:322019-10-22 16:34:17Xenikos receives FDA Fast Track designation← #nieuws
This year, the scholarships have been awarded to Gerard Boink, PhD (CEO at PacingCure BV, the Netherlands) and Sarah Triest, PhD (scientist at Confo Therapeutics Belgium). Gerard and Sarah will participate in the 3-part blended course Clinical Development, a highly rated course from Paul Janssen Futurelab. The scholarship is for start-ups who plan to run their first-in-human clinical trial within the next 1.5 years.
During the course, they will use Question-Based Clinical Development to design the optimal clinical developmental program for their product. The course includes an online introduction course, an interactive on-campus course with cases based on real R&D projects from industry and an extended course focused on writing a clinical trial proposal with guidance from an experienced mentor.
The online course is now running and a new round is planned to take place in spring next year. The on-campus courses will take place in June 2020.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-11 11:47:052019-10-11 11:47:07Janssen Campus the Netherlands has made available scholarships for 2 promising candidates from young start-up companies← #nieuws
Every year, an estimated 300.000
hemodialysis patients worldwide die as they run out of medical alternatives.
One of the main reasons for these deaths is failure of the vascular access
site, the blood vessel where blood is removed, filtered and returned to the
body during dialysis. To solve this problem, a medical device was developed
that enables a patient’s body to create blood vessels using the patient’s own
cells. Vacis, the company commercializing this technology, has successfully
closed a further funding round to set up GMP production, execute clinical
trials and obtain CE certification.
The
company received funding from Netherlands-based Brightlands Venture Partners
and LIOF through their Limburg Business Development Fund (LBDF), as well as the
German High-Tech Gründerfonds (HTGF). This was supplemented with a so-called
Innovation Credit from RVO (the Netherlands Enterprise Agency). “The financing
and support of the investors will enable us to quickly scale up the clinical
trial and accelerate the route to market by at least a year”, says Ken Messier.
“Vacis
combines elegant science with a solid management team. Brightlands truly is the
ecosystem in which Vacis can validate its technology in patients and go to
market fast.”, says Casper Bruens – Director at Brightlands Venture Partners.
Tys van Elk, Director at LIOF adds: “Vacis is an exciting addition to the local
community of regenerative medicine and realizing this consortium fits perfectly
with LIOF’s increasing cross border ambition in the Euregio.”
The
medical device is a synthetic rod with a specialized surface that is inserted
under the skin. This induces the body to form a tissue capsule, which in the
right environment, further matures into a functional blood vessel that is used
as a vascular access site for hemodialysis. “We found the simplicity of the
solution and the biocompatibility data very convincing and are delighted to
support Vacis” adds Anke Cassing, Investment Manager at HTGF.
The team
has been expanded with two seasoned biotech managers, Ken Messier and Josephine
Brackman. Ken brings a wealth of clinical development and regulatory expertise,
with over 30 years’ experience in Biotech. He has held various Director and Sr.
Program Management positions within biotech companies such as Genzyme and DSM
Biomedical. Josephine brings a broad expertise that covers (project) management,
intellectual property and R&D. She has over 25 years’ experience in
materials and biotech and has held several senior positions within DSM. They
both join Dominik Klump, the Chief Technology Officer of Vacis, in the
management team at the location in Geleen.
Leiden University Medical Center and Maastricht University played a key role in the development of the technology. The project was also supported by The Dutch Kidney Patients Association and the Biomedical Materials Program (BMM), a public-private partnership of leading industrial and academic players and the Dutch government.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-10 18:58:462019-11-05 19:03:53Vacis raises capital from Brightlands Venture Partners, HTGF, LIOF and RVO← #nieuws
ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced positive top-line results from the PQ-110-001 study, a Phase 1/2 dose range finding, first-in-human trial of sepofarsen (QR-110) in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene.
“We reported today that patients receiving sepofarsen had a clinically
meaningful improvement in vision, and in some cases the patient’s vision
improved to a level that could be deemed life changing. This is very
encouraging for the LCA10 community and the Inherited Retinal Disease community
as a whole,” said Stephen R. Russell, MD, Schrage Professor of Ophthalmology
and Visual Sciences and Principal Investigator at the University of Iowa.
“LCA10 is a severe inherited retinal disease that leads to blindness, and for
which there is currently no treatment.”
David Rodman, M.D., Executive Vice President of Research & Development
of ProQR, said, “We are very pleased with the data reported from the Phase 1/2
study, in which LCA10 patients treated with sepofarsen experienced a rapid and
durable improvement in vision. The top-line data from this study strengthen our
confidence in the design of the ongoing Phase 2/3 trial, which could be the
sole registration trial for the sepofarsen program. We appreciate the patients’
and medical communities’ ongoing support for the sepofarsen clinical studies,
and we will continue to work with the regulators to advance this program as
efficiently as possible.”
Top-line results from
the Phase 1/2 trial
Based on positive 3-month interim results from the Phase 1/2 trial (Nature
Medicine 2018) the Phase 2/3
Illuminate trial was initiated earlier this year. The 12-month
top-line results from the Phase 1/2 trial that will be presented today confirm
durable activity of sepofarsen for up to one year in patients with LCA10.
Furthermore, the results support the assumptions used in the design and
powering of Illuminate,
including:
the target registration dose (80 µg with a 160 µg
loading dose) was associated with a clinically meaningful and statistically
significant improvement in vision and had a favorable benefit/risk
profile,
a six-month dosing frequency, as used in Illuminate, was
associated with durable improvements in vision,
the response observed at 12 months in the target
registration dose was equal to or greater than the response at the 3-month
interim analysis,
and subjects with better vision than light perception
(BCVA> LogMAR 3.0) at baseline, the study population in Illuminate, were more
likely to respond to treatment with sepofarsen.
Table: Change from
baseline in treated eye
Endpoint
Direction of improvement
Pooled analysis, all dose groups
(SEM)
n
Target registration dose group,
160/80 µg (SEM)
n
BCVA
in LogMAR
Down ↓
-0.55 (0.26)
p<0.05
11
-0.93 (0.43)
p<0.01
6
FST red
in Log(cd/m2)
Down ↓
-0.92 (0.18)
p<0.01
10
-0.66 (0.14)
p<0.01
6
FST blue
in Log(cd/m2)
Down ↓
-0.79 (0.23)
p<0.02
10
-0.63 (0.31)
p<0.01
6
Mobility course
in Levels
Up ↑
+2.5 (0.99)
p=0.1
10
+4.0 (1.27)
p<0.01
6
Analysis: Pooled
analysis: Unpaired t-test, between group change from baseline versus
untreated. Target registration dose group: MMRM with repeated measures,
within group change from baseline versus baseline
Visual acuity
Statistically significant improvement from baseline in best corrected visual acuity (BCVA) was observed as assessed by the Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart and the Berkeley Rudimentary Vision Test (BRVT). In the pooled analysis with all patients after twelve months of treatment, the mean improvement (and standard error of mean, SEM) was -0.55 LogMAR (SEM 0.26). The mean change in the untreated contralateral eye was -0.11 LogMAR (SEM 0.07).
In the target registration dose group, the mean change from baseline at
twelve months was -0.93 LogMAR (SEM 0.43) with four of six subjects showing an
improvement of more than -0.3 LogMAR from baseline equivalent to 3 lines, or 15
letters, on ETDRS chart (generally considered clinically meaningful by US
regulators). Five of six subjects showed an improvement greater than -0.2
LogMAR (generally considered clinically meaningful by EU regulators). The mean
change in the untreated contralateral eye in this group was -0.22 LogMAR (SEM
0.11). In all five subjects in this group, in which a six-month dosing
frequency was explored, the observed benefit in visual acuity was maintained
during the twelve-month follow up.
Full field stimulus threshold test (FST)
Improvements in visual function were supported by a meaningful increase in the ability to detect flashes of red or blue light as determined by the FST test. In the pooled analysis after twelve months, mean improvement in red light sensitivity was -0.92 log Cd/m2 (SEM 0.18) and improvement in blue light sensitivity was -0.79 log Cd/m2 (SEM 0.23). The mean change in the untreated contralateral eye was -0.16 log Cd/m2 (SEM 0.16) for red light and 0.02 log Cd/m2 (SEM 0.11) for blue light.
In the target registration dose group, the mean change from baseline at
twelve months in red light sensitivity was -0.66 log Cd/m2 (SEM
0.14) and improvement in blue light sensitivity was -0.63 log Cd/m2
(SEM 0.31). Three of six subjects showed an improvement of greater than -0.5
log Cd/m2 for blue light, which can be regarded as clinically
meaningful. Five of six showed a clinically meaningful improvement for red
light. The mean change in the untreated contralateral eye in this group was
0.05 log Cd/m2 (SEM 0.17) for red light and -0.12 log Cd/m2
(SEM 0.16) for blue light.
Mobility course
Most patients demonstrated improvement in functional vision, as assessed using a series of mobility courses at increasing difficulty and multiple light intensities. In the pooled analysis the mean improvement for patients navigating the mobility course after twelve months of treatment was 2.5 levels (SEM 0.99). The mean change in the untreated contralateral eye was 1.75 (SEM 0.75). This increase was likely due to a training effect. An adjusted mobility course endpoint is being validated in parallel with the Illuminate trial.
In the target registration dose group, the mean change at twelve months of
treatment was 4.0 levels (SEM 1.27) with five of six subjects improving by more
than 2.0 levels, which can be regarded as clinically meaningful. The mean
change in the untreated contralateral eye was 2.7 levels (SEM 1.11).
SafetySubjects received up to four doses of sepofarsen (range 1-4), and all eleven subjects in the study completed twelve months of follow up. This represents data equivalent to over 4,000 treatment days. Sepofarsen was observed to be well-tolerated with manageable safety findings. In total, eight cases of lens opacities (cataract) were observed (three in the target registration dose cohort and five in high dose cohort). All six of the subjects that had lens replacement surgery regained their pre-cataract vision. Four cases (in three subjects) of retinal findings were observed in the now retired 320/160 µg dose group: Two incidences of mild cystoid macular edema were resolved with topical treatment and two incidences of subclinical retinal thinning stabilized within two months of last dose without additional treatment.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-10 05:19:432019-10-16 05:30:38ProQR Announces Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients← #nieuws
bluebird bio and Novo Nordisk have entered into a research collaboration to jointly develop next-generation in vivo genomeediting treatments for genetic diseases, including haemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate with the ambition of offering people with haemophilia A a lifetime free of factor replacement therapy.
The research collaboration will utilise bluebird bio’s proprietary mRNA-based megaTAL™ technology that has the potential to provide a highly specific and efficient way to silence, edit or insert genetic components. Aligned with Novo Nordisk’s haemophilia portfolio, the research collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
“We are pleased to announce our collaboration with bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives,” said Marcus Schindler, senior vice president for Global Drug Discovery at Novo Nordisk. “This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisk’s enduring commitment and dedication to inventing disease-modifying medicines that can truly change the lives of people living with haemophilia and other genetic diseases.”
“bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” said Philip Gregory, D. Phil., chief scientific officer, bluebird bio. “We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases. Moreover, we are thrilled to be able to combine this new platform technology with Novo Nordisk’s deep expertise in haemophilia research and therapeutics. We believe this collaboration will move us toward our shared goal of recoding the treatment paradigm and substantially reduce the burden of diseasefor patients with factor VIII deficiency.”
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-10-09 22:17:302019-10-15 22:21:24bluebird bio and Novo Nordisk Enter into Research Agreement← #nieuws
“Nu staat de genetische revolutie voor de deur van de brouwerijen.” De NRC wetenschap bijlage van het afgelopen weekend stond in het teken van de ongekende toepassingen van genetische modificatie van gist en de maatschappelijke acceptatie.
Genetici kunnen sneller dan ooit biergisten met allerlei gewenste smaken en capaciteiten maken. Gisten kunnen bijvoorbeeld worden aangepast zodat ze gewenste fruitige aroma’s produceren, maar niet het daarmee gepaarde hogere alcoholpercentage. Ook wordt er onderzocht hoe gist bij een hogere temperatuur kan fermenteren. Nu moeten vele duizenden liters bier op koelkast temperatuur worden gebrouwen. Als dit op een hogere temperatuur kan zou dit niet alleen kosten besparen, maar ook winst zijn voor het milieu.
De grote
biermerken investeren in wetenschappelijke onderzoek naar genetische
modificatie van hun gisten, maar lopen op eieren als het gaat om de
daadwerkelijke implementatie. De merken laten zich leiden door de acceptatie
van genetische modificatie door de consument. Onderzoeker Gorter de Vries heeft
zijn hoop daarom gevestigd op kleine brouwerijen om koploper te worden. “Ik
denk dat er een producent zou kunnen opkomen die zich daarop profileert, en van
de voordelen die genetische modificatie zou kunnen opleveren echt een selling
point maakt”
Acceptatie van genetische modificatie is een kwestie die niet alleen de biersector bezighoudt, de hele levensmiddelenindustrie worstelt er mee. In 2016 deden Nobelprijswinnaars een krachtige oproep aan overheden om genetische modificatie te stimuleren. De oproep werd door 147 Nobelprijswinnaars en duizenden wetenschappers ondertekend. Ondanks deze outcry van de wetenschap werden vorig jaar de nieuwste veredelingsmethoden door het Europees hof onder strenge wetgeving geplaatst. De Europese wetgeving werpt een hoge drempel op voor plantenveredelaars en de levensmiddelen industrie. Daarop lanceerde HollandBIO Crisp.vision, een website die de onmisbare voordelen van genetische modificatie laat zien. Ons motto: ruim baan voor biotech! Want als het aan HollandBIO ligt is de toekomst is diervriendelijk, duurzaam en sensationeel.
Nederlandse zaadveredeling in het zonnetje gezet
Voedsel en materialenSimon Groot, een 84-jarige oer-Hollandse zesde generatie zaadveredelaar uit Enkhuizen, ontvangt deze week prestigieuze World Food Prize, ook wel de Nobelprijs voor Voedsel genoemd. Een bijzondere prestatie, die symbool staat voor nog iets veel groters. Want met onze Nederlandse kennis helpen wij de wereld te voeden. In een mooie video legt NOS op 3 uit hoe innovatief Nederland is op voedselgebied, met daarin een glansrol voor de Nederlandse zaadveredeling. Bekijk ‘m hier!
Minister Van Nieuwenhuizen pleit voor toekomstbestendige regelgeving
Wereld mét toekomst, Hollandbio, Voedsel en materialenIenW minister Cora van Nieuwenhuizen wil op verantwoorde wijze ruimte geven aan innovatieve toepassingen uit de biotech sector die bijdragen aan een duurzame en gezonde samenleving. In haar openingsspeech voor het recente internationale COGEM-symposium over het genetisch bewerken van planten, pleit Van Nieuwenhuizen gepassioneerd voor het grondig moderniseren van de achterhaalde Europese wet- en regelgeving rond genetische modificatie en het versimpelen en verkorten van de nationale procedures in Nederland. De minister is een groot voorstander van een toekomstig systeem met een juiste balans tussen het inschatten van risico’s en het toelaten van innovaties die de wereld een stukje beter maken. HollandBIO kan niet wachten tot het zover is en blijft in de tussentijd meehelpen aan het verwezenlijken van deze toekomst.
Benieuwd naar de hele speech? Lees ‘m hier (in het Engels) terug.
Nederland stijgt naar vierde plek in Global Competitiveness Index
IJzersterk innovatieklimaat, InnovatieklimaatNederland is de meest concurrerende economie van Europa volgens de meest recente Global Competitiveness Index van het World Economic Forum (WEF). Zo stelt het rapport onder andere dat‘de Nederlandse economie veel dynamischer is geworden’. Wel moet Nederland, om in top-10 mee te blijven draaien, verder investeren in innovatie en nieuwe technologie. Ook staatssecretaris Mona Keijzer (Economische Zaken en Klimaat) onderkent deze aanbeveling. Zij stelt dat Nederland een antwoord moet vinden op de economische en maatschappelijke uitdagingen die op ons afkomen. En dat investeringen in bijvoorbeeld biotechnologie en kunstmatige intelligentie de sleutel zijn tot het behouden van de Nederlandse toppositie in de toekomst. Lees meer
Toepassingen uit nieuwe technologie hebben het juiste verhaal nodig
Wereld mét toekomst, Voedsel en materialenVeelbelovende technologieën zoals CRISPR-Cas en kweekvlees kunnen in potentie de wereld veranderen. Alleen, hoe neem je daar de maatschappij in mee? In een artikel in het FD komt dit vraagstuk aan bod op basis van kweekvlees als centrale casus en de vraag welk verhaal het beste bij zo’n hightech innovatie past. Is dat het frame van technologische en wetenschappelijke vooruitgang, of toch het duurzaamheidsframe? Er is een heel dunne scheidslijn tussen aan de ene kant het door de sector veelgebruikte idyllische, wetenschappelijke verhaal met labjassen en DNA-strengen en aan de andere kant het door critici en NGO’s gecreëerde dystopische toekomstverhaal met ‘Frankenstein Foods’. Beter is het om het verhaal en de beeldvorming daaromheen op te bouwen vanuit de positieve bijdrage die een toepassing levert aan een betere maatschappij. Daarmee is dit ‘groene frame’ een essentieel instrument om in een vroege fase op een positieve wijze de dialoog aan te gaan met het bredere publiek.
Artikel FD (log in verplicht)
Xenikos receives FDA Fast Track designation
GezondheidXenikos, which develops innovative immunotherapies for treating patients with severe immune disease and post-transplant rejection, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to T-Guard, Xenikos’ flagship product designed to treat steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following allogeneic stem cell transplantation. Xenikos is currently preparing to initiate a U.S. pivotal Phase 3 trial with T-Guard.
The Fast Track program is designed to facilitate the development of new therapies for treating severe medical conditions in order to fill a currently unmet medical need. Fast Track designation allows for early and frequent communications with the FDA, as well as rolling submission of the market application.
“We are delighted that the FDA has given Fast Track designation to T-Guard, as this will support our goal of getting T-Guard to patients as quickly as possible,” said Dr. Ypke van Oosterhout, Chief Executive Officer of Xenikos. “We’re also looking forward to starting our Phase 3 pivotal trial in the U.S. Effective new therapies for treating SR-aGVHD are urgently needed, and we believe that T-Guard, which can rapidly and safely reset the patient’s immune system, has the potential to provide an important new treatment option to patients with this devastating, potentially fatal condition.”
T-Guard: Helping reset the body’s immune system
T-Guard is designed to safely and swiftly reset the body’s immune system in life-threatening T cell‒mediated conditions, including transplant-related rejection, acute solid-organ rejection, and severe autoimmune disease. T Guard consists of a unique combination of toxin-conjugated monoclonal antibodies that target CD3 and CD7 molecules on T cells and NK cells. Preclinical and early clinical testing have shown that T-Guard can specifically identify and eliminate mature T cells and NK cells with minimal treatment-related side effects. Importantly, T-Guard’s action is short-lived, thereby significantly reducing the patient’s vulnerability to opportunistic infections compared to currently available therapies. Xenikos successfully completed a Phase 1/2 study for the second-line treatment of SR-aGVHD in patients following hematopoietic stem cell transplantation (HSCT). The results of this study showed that just one week of T-Guard treatment triggered a strong clinical response and doubled the 6-month overall survival rate. These results were published in the peer-reviewed journal Biology of Blood and Marrow Transplantation. A U.S. Phase 3 registration trial involving patients with SR-aGVHD following allogeneic stem cell transplantation will begin soon, and T-Guard has been granted Fast Track designation by the FDA, as well as Orphan Drug Designation status in both the EU and the U.S.
Source: Xenikos
Janssen Campus the Netherlands has made available scholarships for 2 promising candidates from young start-up companies
InnovatieklimaatThis year, the scholarships have been awarded to Gerard Boink, PhD (CEO at PacingCure BV, the Netherlands) and Sarah Triest, PhD (scientist at Confo Therapeutics Belgium). Gerard and Sarah will participate in the 3-part blended course Clinical Development, a highly rated course from Paul Janssen Futurelab. The scholarship is for start-ups who plan to run their first-in-human clinical trial within the next 1.5 years.
During the course, they will use Question-Based Clinical Development to design the optimal clinical developmental program for their product. The course includes an online introduction course, an interactive on-campus course with cases based on real R&D projects from industry and an extended course focused on writing a clinical trial proposal with guidance from an experienced mentor.
The online course is now running and a new round is planned to take place in spring next year. The on-campus courses will take place in June 2020.
Vacis raises capital from Brightlands Venture Partners, HTGF, LIOF and RVO
InnovatieklimaatEvery year, an estimated 300.000 hemodialysis patients worldwide die as they run out of medical alternatives. One of the main reasons for these deaths is failure of the vascular access site, the blood vessel where blood is removed, filtered and returned to the body during dialysis. To solve this problem, a medical device was developed that enables a patient’s body to create blood vessels using the patient’s own cells. Vacis, the company commercializing this technology, has successfully closed a further funding round to set up GMP production, execute clinical trials and obtain CE certification.
The company received funding from Netherlands-based Brightlands Venture Partners and LIOF through their Limburg Business Development Fund (LBDF), as well as the German High-Tech Gründerfonds (HTGF). This was supplemented with a so-called Innovation Credit from RVO (the Netherlands Enterprise Agency). “The financing and support of the investors will enable us to quickly scale up the clinical trial and accelerate the route to market by at least a year”, says Ken Messier.
“Vacis combines elegant science with a solid management team. Brightlands truly is the ecosystem in which Vacis can validate its technology in patients and go to market fast.”, says Casper Bruens – Director at Brightlands Venture Partners. Tys van Elk, Director at LIOF adds: “Vacis is an exciting addition to the local community of regenerative medicine and realizing this consortium fits perfectly with LIOF’s increasing cross border ambition in the Euregio.”
The medical device is a synthetic rod with a specialized surface that is inserted under the skin. This induces the body to form a tissue capsule, which in the right environment, further matures into a functional blood vessel that is used as a vascular access site for hemodialysis. “We found the simplicity of the solution and the biocompatibility data very convincing and are delighted to support Vacis” adds Anke Cassing, Investment Manager at HTGF.
The team has been expanded with two seasoned biotech managers, Ken Messier and Josephine Brackman. Ken brings a wealth of clinical development and regulatory expertise, with over 30 years’ experience in Biotech. He has held various Director and Sr. Program Management positions within biotech companies such as Genzyme and DSM Biomedical. Josephine brings a broad expertise that covers (project) management, intellectual property and R&D. She has over 25 years’ experience in materials and biotech and has held several senior positions within DSM. They both join Dominik Klump, the Chief Technology Officer of Vacis, in the management team at the location in Geleen.
Leiden University Medical Center and Maastricht University played a key role in the development of the technology. The project was also supported by The Dutch Kidney Patients Association and the Biomedical Materials Program (BMM), a public-private partnership of leading industrial and academic players and the Dutch government.
Source: Vacis
ProQR Announces Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients
GezondheidProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced positive top-line results from the PQ-110-001 study, a Phase 1/2 dose range finding, first-in-human trial ofsepofarsen (QR-110) in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene.
“We reported today that patients receiving sepofarsen had a clinically meaningful improvement in vision, and in some cases the patient’s vision improved to a level that could be deemed life changing. This is very encouraging for the LCA10 community and the Inherited Retinal Disease community as a whole,” said Stephen R. Russell, MD, Schrage Professor of Ophthalmology and Visual Sciences and Principal Investigator at the University of Iowa. “LCA10 is a severe inherited retinal disease that leads to blindness, and for which there is currently no treatment.”
David Rodman, M.D., Executive Vice President of Research & Development of ProQR, said, “We are very pleased with the data reported from the Phase 1/2 study, in which LCA10 patients treated with sepofarsen experienced a rapid and durable improvement in vision. The top-line data from this study strengthen our confidence in the design of the ongoing Phase 2/3 trial, which could be the sole registration trial for the sepofarsen program. We appreciate the patients’ and medical communities’ ongoing support for the sepofarsen clinical studies, and we will continue to work with the regulators to advance this program as efficiently as possible.”
Top-line results from the Phase 1/2 trial
Based on positive 3-month interim results from the Phase 1/2 trial (Nature Medicine 2018) the Phase 2/3 Illuminate trial was initiated earlier this year. The 12-month top-line results from the Phase 1/2 trial that will be presented today confirm durable activity of sepofarsen for up to one year in patients with LCA10. Furthermore, the results support the assumptions used in the design and powering of Illuminate, including:
Table: Change from baseline in treated eye
in LogMAR
p<0.05
p<0.01
in Log(cd/m2)
p<0.01
p<0.01
in Log(cd/m2)
p<0.02
p<0.01
in Levels
p=0.1
p<0.01
Analysis: Pooled analysis: Unpaired t-test, between group change from baseline versus untreated. Target registration dose group: MMRM with repeated measures, within group change from baseline versus baseline
Visual acuity
Statistically significant improvement from baseline inbest corrected visual acuity (BCVA) was observed as assessed by the Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart and the Berkeley Rudimentary Vision Test (BRVT). In the pooled analysis with all patients after twelve months of treatment, the mean improvement (and standard error of mean , SEM) was -0.55 LogMAR (SEM 0.26). The mean change in the untreated contralateral eye was -0.11 LogMAR (SEM 0.07).
In the target registration dose group, the mean change from baseline at twelve months was -0.93 LogMAR (SEM 0.43) with four of six subjects showing an improvement of more than -0.3 LogMAR from baseline equivalent to 3 lines, or 15 letters, on ETDRS chart (generally considered clinically meaningful by US regulators). Five of six subjects showed an improvement greater than -0.2 LogMAR (generally considered clinically meaningful by EU regulators). The mean change in the untreated contralateral eye in this group was -0.22 LogMAR (SEM 0.11). In all five subjects in this group, in which a six-month dosing frequency was explored, the observed benefit in visual acuity was maintained during the twelve-month follow up.
Improvements in visual function were supported by a meaningful increase in the ability to detect flashes of red or blue light as determined by the FST test. In the pooled analysis after twelve months, mean improvement in red light sensitivity was -0.92 log Cd/m2 (SEM 0.18) and improvement in blue light sensitivity was -0.79 log Cd/m2 (SEM 0.23). The mean change in the untreated contralateral eye was -0.16 log Cd/m2 (SEM 0.16) for red light and 0.02 log Cd/m2 (SEM 0.11) for blue light.
In the target registration dose group, the mean change from baseline at twelve months in red light sensitivity was -0.66 log Cd/m2 (SEM 0.14) and improvement in blue light sensitivity was -0.63 log Cd/m2 (SEM 0.31). Three of six subjects showed an improvement of greater than -0.5 log Cd/m2 for blue light, which can be regarded as clinically meaningful. Five of six showed a clinically meaningful improvement for red light. The mean change in the untreated contralateral eye in this group was 0.05 log Cd/m2 (SEM 0.17) for red light and -0.12 log Cd/m2 (SEM 0.16) for blue light.
Mobilitycourse
In the target registration dose group, the mean change at twelve months of treatment was 4.0 levels (SEM 1.27) with five of six subjects improving by more than 2.0 levels, which can be regarded as clinically meaningful. The mean change in the untreated contralateral eye was 2.7 levels (SEM 1.11).
SafetySubjects received up to four doses ofsepofarsen (range 1-4), and all eleven subjects in the study completed twelve months of follow up. This represents data equivalent to over 4,000 treatment days. Sepofarsen was observed to be well-tolerated with manageable safety findings. In total, eight cases of lens opacities (cataract) were observed (three in the target registration dose cohort and five in high dose cohort). All six of the subjects that had lens replacement surgery regained their pre-cataract vision. Four cases (in three subjects) of retinal findings were observed in the now retired 320/160 µg dose group: Two incidences of mild cystoid macular edema were resolved with topical treatment and two incidences of subclinical retinal thinning stabilized within two months of last dose without additional treatment.
Source ProQR (press release)
bluebird bio and Novo Nordisk Enter into Research Agreement
Gezondheidbluebird bio and Novo Nordisk have entered into a research collaboration to jointly develop next-generation in vivogenomeediting treatments for genetic diseases, including haemophilia . During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate with the ambition of offering people with haemophilia A a lifetime free of factor replacement therapy.
The research collaboration will utilise bluebird bio’s proprietary mRNA-based megaTAL™ technology that has the potential to provide a highly specific and efficient way to silence, edit or insert genetic components. Aligned with Novo Nordisk’s haemophilia portfolio, the research collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
“We are pleased to announce our collaboration with bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives,” said Marcus Schindler, senior vice president for Global Drug Discovery at Novo Nordisk. “This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisk’s enduring commitment and dedication to inventing disease-modifying medicines that can truly change the lives of people living with haemophilia and other genetic diseases.”
“bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” said Philip Gregory, D. Phil., chief scientific officer, bluebird bio. “We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases. Moreover, we are thrilled to be able to combine this new platform technology with Novo Nordisk’s deep expertise in haemophilia research and therapeutics. We believe this collaboration will move us toward our shared goal of recoding the treatment paradigm and substantially reduce the burden of diseasefor patients with factor VIII deficiency.”
Source: bluebird bio (press release)
Welke brouwer wordt koploper?
Wereld mét toekomst, Hollandbio, Voedsel en materialen“Nu staat de genetische revolutie voor de deur van de brouwerijen.” De NRC wetenschap bijlage van het afgelopen weekend stond in het teken van de ongekende toepassingen van genetische modificatie van gist en de maatschappelijke acceptatie.
Genetici kunnen sneller dan ooit biergisten met allerlei gewenste smaken en capaciteiten maken. Gisten kunnen bijvoorbeeld worden aangepast zodat ze gewenste fruitige aroma’s produceren, maar niet het daarmee gepaarde hogere alcoholpercentage. Ook wordt er onderzocht hoe gist bij een hogere temperatuur kan fermenteren. Nu moeten vele duizenden liters bier op koelkast temperatuur worden gebrouwen. Als dit op een hogere temperatuur kan zou dit niet alleen kosten besparen, maar ook winst zijn voor het milieu.
De grote biermerken investeren in wetenschappelijke onderzoek naar genetische modificatie van hun gisten, maar lopen op eieren als het gaat om de daadwerkelijke implementatie. De merken laten zich leiden door de acceptatie van genetische modificatie door de consument. Onderzoeker Gorter de Vries heeft zijn hoop daarom gevestigd op kleine brouwerijen om koploper te worden. “Ik denk dat er een producent zou kunnen opkomen die zich daarop profileert, en van de voordelen die genetische modificatie zou kunnen opleveren echt een selling point maakt”
Acceptatie van genetische modificatie is een kwestie die niet alleen de biersector bezighoudt, de hele levensmiddelenindustrie worstelt er mee. In 2016 deden Nobelprijswinnaars een krachtige oproep aan overheden om genetische modificatie te stimuleren. De oproep werd door 147 Nobelprijswinnaars en duizenden wetenschappers ondertekend. Ondanks deze outcry van de wetenschap werden vorig jaar de nieuwste veredelingsmethoden door het Europees hof onder strenge wetgeving geplaatst. De Europese wetgeving werpt een hoge drempel op voor plantenveredelaars en de levensmiddelen industrie. Daarop lanceerde HollandBIO Crisp.vision, een website die de onmisbare voordelen van genetische modificatie laat zien. Ons motto: ruim baan voor biotech! Want als het aan HollandBIO ligt is de toekomst is diervriendelijk, duurzaam en sensationeel.
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