Alnylam, the leading RNAi therapeutics company, announced today that the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), met its primary efficacy endpoint and the majority of secondary endpoints. Specifically, givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks relative to placebo (p less than 0.00000001) and achieved statistically significant results for five of nine secondary endpoints (p less than 0.0001), with a safety and tolerability profile that the Company believes is encouraging, especially in this high unmet disease. Based on these results, the Company plans to complete its rolling submission of a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) in mid-2019.
“Patients living with AHP experience debilitating and sometimes life-threatening neurovisceral attacks as well as chronic disease manifestations which negatively impact their quality of life. We believe the ENVISION results demonstrate a robust therapeutic benefit of givosiran treatment on the debilitating aspects of this disease. Based on these results, we believe givosiran has the potential, if approved, to be a transformative medicine for AHP patients and their families,” said Akshay Vaishnaw, M.D., Ph.D., President, R&D at Alnylam. “We extend our profound thanks to all the patients, investigators, and study staff who participated in the ENVISION study.”
“These positive ENVISION results represent another landmark event in Alnylam’s pioneering efforts to advance RNAi therapeutics as a whole new class of medicines. Notably, givosiran is the first GalNAc-conjugate siRNA to achieve positive Phase 3 results, and the second example of Alnylam’s R&D strategy bearing fruit due to our focus on genetically validated targets for the advancement of potential high impact medicines,” said John Maraganore, Ph.D., CEO of Alnylam. “Assuming favorable regulatory review, we very much look forward to adding givosiran as the second product in our global commercialization efforts. Indeed, we believe today’s news brings us one important step closer to meeting our Alnylam 2020 goals of building a multi-product, global commercial company with a deep clinical pipeline for continued growth and a robust product engine for sustainable innovation.”
Givosiran has received Breakthrough Therapy and Prime designation by the FDA and EMA, respectively, and has Orphan Drug status in the U.S. and EU for the treatment of AHP. The Phase 1 results of givosiran were recently published in TheNew England Journal of Medicine1. FullENVISION study results will be presented in an oral plenary session on Saturday, April 13 at EASL in Vienna, Austria. The ENVISION results have not yet been reviewed by regulatory authorities.
ENVISION Study Results
ENVISION, a randomized, double-blind, placebo-controlled trial, enrolled 94 patients with AHP (including 89 with genetically-confirmed acute intermittent porphyria [AIP], the most common subtype of AHP), at 36 study sites in 18 countries around the world, and is the largest ever interventional study conducted in AHP. Patients were randomized 1:1 to givosiran or placebo, with givosiran administered subcutaneously at 2.5 mg/kg monthly. The primary endpoint for the study was reduction relative to placebo in the annualized rate of composite porphyria attacks, defined as those requiring hospitalization, urgent healthcare visit, or hemin administration, in patients with AIP over six months.
At six months, givosiran met the primary endpoint in patients with AIP (p less than 0.00000001). All the components of the composite primary endpoint and all subgroup analyses for the primary endpoint favored givosiran. Secondary endpoints listed below also demonstrated statistically significant favorable differences in the givosiran arm compared to placebo (p less than 0.0001):
Urinary ALA levels at three months in AIP patients;
Urinary ALA levels at six months in AIP patients;
Urinary PBG levels at six months in AIP patients;
Annualized days of administered hemin doses in AIP patients; and
Annualized attack rate in patients with AHP (including AIP).
The remaining four secondary endpoints did not meet the hierarchical threshold for significance and included: daily worst pain (p equal to 0.053), daily worst fatigue (p equal to 0.29), daily worst nausea (p equal to 0.25), and the physical component summary of the SF-12 health survey in AIP patients (p equal to 0.022).
Adverse events (AEs) were reported in 43/48 (89.6 percent) of givosiran and 37/46 (80.4 percent) of placebo patients and serious adverse events (SAEs) were reported in 10/48 (20.8 percent) of givosiran and 4/46 (8.7 percent) of placebo patients. There were no deaths in the study. One patient, described below, in the givosiran arm (2.1 percent) discontinued treatment due to an AE. AEs reported in greater than 10 percent of givosiran patients and seen more frequently compared to placebo were: nausea, injection site reactions, chronic kidney disease and fatigue; those reported in greater than 10 percent of placebo patients and seen more frequently than in givosiran-treated patients were headache, urinary tract infection, vomiting and pyrexia. The AEs of chronic kidney disease were reported in five givosiran-treated patients (10.4 percent) and no placebo patients; these events were all in patients with renal dysfunction at baseline and patients continued dosing throughout the study. Liver transaminase increases greater than three times upper limit of normal (ULN) were observed in 7/48 (14.6 percent) patients on givosiran and 1/46 (2.2 percent) patients on placebo; all had evidence of iron overload or liver disease at baseline. As previously reported and as noted above, one patient on givosiran discontinued treatment due to an increase in alanine aminotransferase (ALT) levels greater than 8 times ULN, a protocol-defined stopping rule; this elevation did not meet Hy’s Law and subsequently resolved. Peak ALT levels in the other six givosiran-treated patients ranged from 3.0-5.4 times ULN and were not accompanied by bilirubin increases. The patients were asymptomatic, and all events resolved with continued dosing (n=5) or after a brief pause in dosing (n=1).
Upon completion of dosing in the ENVISION double-blind period, all eligible patients (93/94 or 99 percent) enrolled in the ENVISION open-label extension (OLE) study, receiving monthly givosiran administration. In addition, patients continue dosing in the Phase 1/2 OLE study with over two years of exposure to givosiran.
Virtuvax and
the trustee of biotech company Mucosis, Mr. Holtz LLM announced the results of
a first-in-human study of the intranasal Respiratory Syncytial Virus (RSV)
vaccine candidate, SynGEM. The study was executed by Imperial College London.
Results of the study have been posted (in press) on the website of the American Journal of Respiratory and Critical Care Medicine. The vaccine was found to be safe and immunogenic in healthy adults.
“Virus-specific
immunogenicity induced after SynGEM vaccination was demonstrated by systemic
IgG+ and IgA+ responses in terms of antibody-secreting cells (ASCs)” indicated
prof. Openshaw, professor of Experimental Medicine at Imperial College London.
Professor Openshaw added that “SynGEM induced some RSV-specific mucosal IgA in
most individuals, particularly in those with low pre-existing F-specific IgA
titers. Indeed, higher doses of the vaccine induced prolonged IgA-producing ASC
responses in some volunteers.” Prof. Openshaw further explained that “The
publication includes in vitro data supporting the clinical observations,
showing that SynGEM induces a dose-dependent antibody response associated with
cytokine production from T-helper cells.”
Dr. Chiu,
principal investigator of the clinical trial, commented: “Previously, we showed
that antibodies induced by natural RSV infection were only short-lived and we
hypothesized that RSV-induced immunomodulatory mechanisms impaired the antibody
responses against the virus. In this study, SynGEM not only induced immune
responses but also boosted long-term systemic RSV-specific antibodies for at
least 6 months. Vaccination with the SynGEM RSV vaccine therefore was not
associated with evidence of immune modulation, as seen after infection with
live RSV.”
In another
publication also in AJRCCM, senior author Prof. Louis Bont of University
Medical Center Utrecht, The Netherlands commented on the SynGEM trial. Prof.
Bont argued that in the absence of a correlate of protection to RSV, vaccine
developers are working in the dark. In the case of SynGEM, preclinical data had
shown protection against infection suggesting evidence of the vaccine providing
protective immunity. However, despite the induction of broad immune responses,
neutralizing antibodies were not detected in the clinical trial. In their
comment, the authors suggested that testing of the SynGEM vaccine in a human
challenge model might be a quick way to finally determine proof of concept of
efficacy.
The
investigators of Imperial College were surprised that no detectable
neutralizing antibody responses were found, since extensive stability tests had
shown stable binding of such antibodies by SynGEM. Openshaw said: “the system
needs further optimization, but establishes proof of principal that the nasal vaccine
approach can work. It was a shame that the programme was ended before the
infection challenge component could be performed”. Trustee of Mucosis, Mr.
Holtz, commented: “During the clinical trial, Mucosis was confronted with a
sudden shortage of finances and the company had to apply for bankruptcy before
the final results were known as published today. Since these long-term data
have become available as of now, any further development of the vaccine may be
taken forward by other companies interested in doing so.”
SynGEM is
based on a unique pre-fusion version of the F subunit of RSV, shown to raise
more potent serum neutralizing antibodies against RSV compared with the
post-fusion F antigen approach others have used. The pre-fusion conformation
(pre-F), which predominates on infectious virions, displays a
distinct
antigenic site (site Ø) preferentially targeted by the most potent neutralizing
antibodies. Preclinical studies using SynGEM showed induction of high levels of
both systemic and mucosal antibodies.
Delivered
via the mucous membranes in the nose, the vaccine candidate is also able to
recruit antibodies produced in the mucosal linings of the body, with the
potential to effectively stop the virus from entering the body via the mucosal
pathways, where over 90% of pathogens enter the body.
SynGEM is
based on Mucosis’ patented Mimopath technology, which uses bacterium-like
particles (BLP) derived from food-grade bacteria, to deliver the antigen in a
more natural conformation and boost the body’s immune response to the virus.
RSV is a
common contagious infection of the lungs and respiratory pathways, eliciting
cold-like symptoms in adults. Whilst RSV infection is normally mild in healthy
adults, the infection can lead to serious and sometimes fatal diseases such as
pneumonia and bronchiolitis, most often in susceptible populations such as the
young and elderly. Every year in the US, RSV infections lead to 57,527 hospitalizations
of children under 5 year’s old (2)and are responsible for up to 200,000 deaths
annually worldwide (3). The annual global burden of RSV illness is significant,
with 33.8 million estimated new episodes of RSV-associated acute lower
respiratory infection (ALRI) worldwide in children under 5, and over 3.4
million hospital admissions associated with severe RSV disease. Global
mortality was estimated at 253,500 deaths in 2010.
The Wellcome Trust translational fund supported the clinical trial of SynGEM with a staged award of up to €3.44 million (£2.77 million).
BiosanaPharma has received permission from the Australian Bellberry Human Research Ethics Committee HREC to start a phase I clinical trial of their first pipeline product. The trial consists of a bioequivalence double-blind, randomized, two-parallel-group phase I study of a humanized anti-immunoglobulin E monoclonal antibody ‘BP001’ as lyophilized powder formulation compared with the standard omalizumab (Xolair®) lyophilized powder formulation in healthy male volunteers. Prior to the submission of the Investigative Brochure to the HREC an extensive comparability exercise was performed between BP001 and Xolair®, indicating functional, binding and structural similarity between the two molecules. This extensive laboratory exercise was also sufficient for Biosana to obtain a waiver for preclinical studies in animal models from the EMA.
The Phase I trial is expected to be
concluded and fully reported in Q4 2019.
Ard Tijsterman, CEO of BiosanaPharma: “We are extremely happy and proud that we’ve been able to develop our first biosimilar product, produced by our innovative ‘3C process’, the first fully continuous process to deliver a product entering phase I. We will start negotiations with potential marketing & sales partners now to perform phase III studies and secure future global distribution of the product.”
Omalizumab
Omalizumab is marketed by Roche/Genentech (USA) and Novartis (ROW) as Xolair® since 2006. It is used to treat moderate to severe allergic asthma and chronic idiopathic urticaria in patients aged 12 years and above in the US and the EU. Additionally, it is approved for paediatric patients aged 6 to 11 years in the EU. Xolair® is delivered via subcutaneous injections. Total sales for Xolair® in 2018 were US$ 2.9M with a consistent growth in both indications of 11-13% annually. The US market accounted for roughly 2/3 of total Xolair® sales. The patent on Xolair® has expired in 2017 in the US and Europe (Novartis, 2016).
BiosanaPharma
BiosanaPharma is a Dutch/Australian biotechnology company with a disruptive approach to the manufacture and delivery of monoclonal antibody therapeutics (mAbs). BiosanaPharma’s “3C process” offers a 90% reduction in the cost of developing and manufacturing EMA/FDA quality mAbs. The 3C process is a fully-continuous, small footprint platform and produced BiosanaPharma’s first pipeline drug BP001, now approved to enter Phase I (see above).
BiosanaPharma is also developing a platform for oral delivery of biologicals, which has delivered successful preclinical results. The current pipeline consists of biosimilars and oral versions of blockbuster biologics.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-03-05 23:08:042019-03-05 23:08:06BiosanaPharma gets approval to start phase I clinical trial for a biosimilar version of omalizumab← #nieuws
Scientists from the Hubrecht Institute, the University of Utrecht, and MIMETAS have successfully grown kidney tubules derived from human kidney adult stem cells in microfluidic chips. This research has been published in Nature Biotechnology by the world-leading research group from Professor Hans Clevers.
Henriette Lanz, Ph.D., Director of Biology at MIMETAS and co-author on the paper explains: “Scientists at the Hubrecht Institute managed to reprogram stem cells found in the human kidney cortex to cells that are similar to tubular cells of the kidney. From these cells, my team has grown three-dimensional perfused kidney tubules. We have discovered that these tubules are fully polarized, meaning that they distinguish inside from outside, just like in a real kidney. Moreover, the biologically important barrier function of the kidney tubes is intact. We show that transporter activity is functional, which is a hallmark of kidney tissue, responsible for pumping of nutrients and toxicants across the kidney barrier. We can grow 40 of such tubules in one single OrganoPlate®. The kidney tubes can be used to create disease models that allow the development of novel therapies. This novel technology offers a powerful human alternative to animal testing for toxic side effects of new medicines.”
A mini kidney from the lab doesn’t look like a normal kidney, but the simple cell structures share many of the characteristics of real kidneys. Researchers can use them to study certain kidney diseases. “We can use these mini kidneys to model various disorders: hereditary kidney diseases, infections, and cancer. This allows us to study in detail what exactly is going wrong”, says Hans Clevers, Professor of Molecular Genetics at Utrecht University and the University Medical Center Utrecht, and group leader at the Hubrecht Institute.“This helps us to understand the workings of healthy kidneys better, and hopefully, in the future, we will be able to develop treatments for kidney disorders.”
Bioinformatics software engineering company ENPICOM has closed a series A investment round. BOM Brabant Ventures, Nextgen Ventures and Arches Capital have jointly invested 1.2 million euros to support the company’s repertoire sequencing data analysis platform. ENPICOM will use these proceeds to finalize development of its ImmunoGenomiX (IGX) platform and introduce it in the marketplace.
ENPICOM BV was founded in 2017 by Dr. Alvise Trevisan (CTO), Dr. Nicola Bonzanni (CSO) and Jos Lunenberg, MSc, MBA (CEO). Mid 2017 the company started the development of a unique platform to manage, store, analyze, visualize and interpret clone sequencing data from T and B cell receptor repertoires. ENPICOM is currently in the final stage of preparations for the global launch of the first full version of its ImmunoGenomiX (IGX) platform. This festive event will take place in Vienna at the 6th Immunotherapy of Cancer Conference (ITOC6) from April 11 to 13.
Dr. Bonzanni commented: “When studying immune system related diseases or developing drugs influencing this system, it is extremely important to be able to directly measure and closely monitor its status. Repertoire sequencing is a powerful way to analyze hundreds of thousands to millions of T or B cell receptors swiftly and in a cost-effective way.” “While there are different companies offering kits and service for this specific form of targeted sequencing, there is no commercial platform to manage the rapidly growing amounts of data generated in the lab in an intuitive, user-friendly way,” added Dr. Trevisan. He continued: “To get from data to powerful insights from this exciting technology, smart new analysis methods and rock-solid software were needed. We have built a team of experts, bringing together a wealth of knowledge and expertise in this field, to develop the IGX platform.”
“I am very pleased we have attracted this excellent syndicate of investors,” said CEO Lunenberg. “With the completion of this Series A financing, we are well funded to execute on our IGX development and market introduction programs.”
Quotes from the investors
Sjoerd van Gorp (BOM): “The ambitions of the ENPICOM team in the field of personalized immunotherapy fits perfectly with our investment goals. By providing innovative life sciences companies and healthcare providers with the tools to measure the status of the patients’ immune system we believe that ENPICOM can facilitate efficient drug development and ensure effective treatment for patients.”
Matthijs Blokhuis (NextGen Ventures): “The ambition of ENPICOM to personalize immunotherapy resonates extremely well with the objective of NextGen Ventures to provide meaningful results for patients and providers. An increased understanding of immune repertoire sequencing data significantly helps to improve outcomes in an efficient manner.”
Eric van der Maten (Arches Capital): “ENPICOM is a perfect example of what we look for in a portfolio company: a strong, highly skilled, motivated and dedicated team, that is showing the ability to combine innovative emerging technologies like data science, AI and medical science into an exhilarating and fast-growing Deep Tech company, eventually even with a real impact on society. We are very pleased to add ENPICOM to our investment portfolio”.
Met een interview in haar nieuwsbrief Graadmeter mengt ook de Gezondheidsraad (GR) zich in het maatschappelijk en politiek debat over vaccineren. Prof. Gerhard Zielhuis, voorzitter van de Commissie Vaccinaties, licht in dit interview onder andere het beoordelingskader en de rol van de GR omtrent de dalende vaccinatiegraad toe. HollandBIO vindt het top dat ook de GR, als wetenschappelijke hoeder van onder meer het Rijksvaccinatieprogramma, zich nu in de discussie mengt.
Het interview sluit mooi aan op het plenaire debat over vaccinatie in de Tweede Kamer, twee weken geleden. Middels een motie droeg de Kamer Staatssecretaris Blokhuis op om geregistreerde vaccins direct te voorzien van een advies over opname in een programma. Een oproep die bij HollandBIO met gejuich ontvangen werd. De Staatssecretaris is nu aan zet.
HollandBIO is van mening dat ook vaccins die (nog) niet voldoen
aan de criteria voor opname in een vaccinatieprogramma of het verzekerd pakket,
een route op maat verdienen. Nu belanden die vaccins vaak ongebruikt op de
plank, al voegen ze op individueel niveau nog zoveel waarde toe. Hierdoor
blijft gezondheidswinst liggen. HollandBIO is pas tevreden als hier een
oplossing voor komt en we vaccins optimaal benutten in de strijd tegen
infectieziekten.
Het gericht veranderen van het plantengenoom is niet
makkelijk. Het moeilijkste is om het DNA of RNA in de plantencel te krijgen.
Daar wordt vaak letterlijk grof geschut voor gebruikt, een methode die
‘shotgun’ heet. Het DNA met behulp van bacteriën in de cel brengen is
subtieler, maar ook aan deze methode kleven verschillende nadelen. Zo blijven
er altijd ‘sporen’ van het bacterieel DNA aanwezig. In Europa worden beide methoden
als genetische modificatie beschouwd. Twee Amerikaanse onderzoeksgroepen hebben
nu – onafhankelijk van elkaar – een briljant alternatief gevonden.
De onderzoekers brengen het DNA in de cel via nanobuisjes
gemaakt van koolstof. Deze buisjes zijn zo klein dat de plantencel ze met het
water opnemen. Om het genoom van de plantencel te kunnen veranderen moeten de
buisjes vervolgens nog doordringen in de celkern. De onderzoekers hebben er
vertrouwen in dat dat ook gaat lukken.
De echte doorbraak voor de plantenveredeling zal komen als
de onderzoekers er in slagen om de onderdelen van het CRISPR-Cas systeem via
deze nanobuisjes de cel in te krijgen. Dan kun je DNA gericht aanpassen terwijl
je de plant bewatert. Diverse landen zien het aanpassen van DNA via CRISPR-Cas
niet als genetische modificatie, waarmee de methode daar laagdrempelig en breed
te benutten is. Helaas is dit in Europa niet het geval. Gelukkig laat de
Nederlandse regering het hier niet bij zitten en zet zij zich in om ook in
Europa CRISPR-Cas vrij te stellen van de GMO-richtlijn en breed toegankelijk te
maken voor de duurzame productie van gezond voedsel.
Afgelopen week publiceerde de Volkskrant een verfrissend gebalanceerd artikel over de totstandkoming van geneesmiddelprijzen. Zeven argumenten van de sector om die prijzen te verklaren passeren hierin de revue. Biogen en Roche grepen deze kans aan en gingen open en eerlijk het gesprek aan over de complexiteit van en uitdagingen in de ontwikkeling en prijsstelling van geneesmiddelen. Een dikke pluim voor Biogen, Roche én de Volkskrant. HollandBIO hoopt dat dit goede voorbeeld breed navolging krijgt. Voor nu: een absolute must-read!
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-03-05 18:32:322019-03-05 18:32:34Dure medicijnen: wat klopt er van de argumenten van de farmaceutische industrie?← #nieuws
Fantastisch nieuws: de hele Tweede Kamer roept de regering
op te laten onderzoeken hoe de werkwijze van het Zorginstituut beter kan
aansluiten op de nieuwe regeling voor weesgeneesmiddelen, voorwaardelijk
toegelaten geneesmiddelen en op de beweging naar personalised medicine. Vandaag
stemde de Tweede Kamer unaniem voor een
motie van de leden Carla Dik-Faber en Pia Dijkstra. De methodiek om
de effectiviteit van de nieuwste generatie geneesmiddelen vast te stellen is achterhaald
en één van de knelpunten die hoog op de ‘te tackelen’ prioriteitenlijst van
HollandBIO staat. We zijn dan ook erg blij dat het opheffen van deze blokkade
op de weg van lab naar patiënt nu op de Haagse agenda staat.
De methodologie en de criteria die het Zorginstituut
gebruikt om te bepalen of een geneesmiddel tot “de Stand der Wetenschap en
Praktijk” (SW&P) behoort, GRADE genaamd, plaatsen de gouden standaard voor
klinisch onderzoek, gerandomiseerde klinische trials (RCT’s) met grote groepen
patiënten, op een voetstuk. En dat systeem loopt steeds verder vast nu biotech
bedrijven steeds vaker geneesmiddelen of therapieën ontwikkelen tegen zeldzame
ziekten, voor subpopulaties of zelfs voor individuele patiënten. Het is
namelijk onmogelijk om aan de oude gouden standaard te voldoen. Allereerst zijn
er simpelweg onvoldoende patiënten om in een trial te includeren. Ten tweede kan
het langdurig continueren van dubbelblind gerandomiseerd onderzoek onethisch
zijn, omdat je een groep ernstig zieke patiënten de enige mogelijke behandeling
onthoudt.
Het gevolg: op het moment dat het Zorginstituut zo’n
geneesmiddel met GRADE beoordeelt aan de hand van de criteria van het verzekerd
pakket, krijgt de onderliggende dataset het stempel ‘niet robuust’. Dit stempel
werkt negatief door in de hele beoordeling, zowel voor het vaststellen van de
therapeutische waarde als voor kosteneffectiviteit. Na het jarenlange, kostbare
en risicovolle ontwikkelingstraject van een geneesmiddel, bereikt de
behandeling de patiënt in het zicht van de haven niet. Niet omdat het niet
effectief is, maar omdat de methodiek het innovatietempo van de biotech sector
niet bij kan benen.
Kortom: de oproep van de Kamer om het Zorginstituut indien
nodig de 21ste eeuw in te slepen, is weliswaar een eerste stap, maar
desalniettemin een mijlpaal. Het erkennen en aanpakken van dit methodologische
probleem is niet alleen cruciaal voor de realisatie van zorg op maat, maar kan
van Nederland een innovatieve en internationaal toonaangevende proeftuin voor
personalised medicine maken.
HollandBIO ziet de lopende evaluatie van het Besluit GGO als een uitgelezen moment om een prangend knelpunt in het Nederlandse biotech beleid weg te nemen: de inschaling voor cel- en gentherapie. Het Besluit GGO ziet toepassing van cel- en gentherapie op het moment als introductie in het milieu. HollandBIO pleit voor inschaling van deze therapieën als ingeperkt gebruik. Alleen dan kan Nederland de vruchten plukken van deze aanstormende nieuwste generatie therapieën.
Naast dit knelpunt bracht HollandBIO een aantal andere verbeterpunten onder de aandacht om het Besluit en de uitvoering daarvan te verbeteren. Deze reiken van de introductie van een zelflerend vermogen, waarbij opgedane kennis en ervaring wordt meegenomen in de uitvoering tot de verbetering van de informatievoorziening op de website van bureau GGO. Lees hier onze volledige inbreng.
https://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svg00hollandbiohttps://www.hollandbio.nl/wp-content/uploads/2023/08/HollandBIO_logo.svghollandbio2019-03-05 18:04:412019-03-05 18:22:37Evaluatie Besluit GGO: maak Nederland koploper in gen- en celtherapie
Alnylam Reports Positive Topline Results from Phase 3 Study in Acute Hepatic Porphyria
GezondheidAlnylam, the leading RNAi therapeutics company, announced today that the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), met its primary efficacy endpoint and the majority of secondary endpoints. Specifically, givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks relative to placebo (p less than 0.00000001) and achieved statistically significant results for five of nine secondary endpoints (p less than 0.0001), with a safety and tolerability profile that the Company believes is encouraging, especially in this high unmet disease. Based on these results, the Company plans to complete its rolling submission of a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) in mid-2019.
“Patients living with AHP experience debilitating and sometimes life-threatening neurovisceral attacks as well as chronic disease manifestations which negatively impact their quality of life. We believe the ENVISION results demonstrate a robust therapeutic benefit of givosiran treatment on the debilitating aspects of this disease. Based on these results, we believe givosiran has the potential, if approved, to be a transformative medicine for AHP patients and their families,” said Akshay Vaishnaw, M.D., Ph.D., President, R&D at Alnylam. “We extend our profound thanks to all the patients, investigators, and study staff who participated in the ENVISION study.”
“These positive ENVISION results represent another landmark event in Alnylam’s pioneering efforts to advance RNAi therapeutics as a whole new class of medicines. Notably, givosiran is the first GalNAc-conjugate siRNA to achieve positive Phase 3 results, and the second example of Alnylam’s R&D strategy bearing fruit due to our focus on genetically validated targets for the advancement of potential high impact medicines,” said John Maraganore, Ph.D., CEO of Alnylam. “Assuming favorable regulatory review, we very much look forward to adding givosiran as the second product in our global commercialization efforts. Indeed, we believe today’s news brings us one important step closer to meeting our Alnylam 2020 goals of building a multi-product, global commercial company with a deep clinical pipeline for continued growth and a robust product engine for sustainable innovation.”
Givosiran has received Breakthrough Therapy and Prime designation by the FDA and EMA, respectively, and has Orphan Drug status in the U.S. and EU for the treatment of AHP. The Phase 1 results of givosiran were recently published in The New England Journal of Medicine1. FullENVISION study results will be presented in an oral plenary session on Saturday, April 13 at EASL in Vienna, Austria. The ENVISION results have not yet been reviewed by regulatory authorities.
ENVISION Study Results
ENVISION, a randomized, double-blind, placebo-controlled trial, enrolled 94 patients with AHP (including 89 with genetically-confirmed acute intermittent porphyria [AIP], the most common subtype of AHP), at 36 study sites in 18 countries around the world, and is the largest ever interventional study conducted in AHP. Patients were randomized 1:1 to givosiran or placebo, with givosiran administered subcutaneously at 2.5 mg/kg monthly. The primary endpoint for the study was reduction relative to placebo in the annualized rate of composite porphyria attacks, defined as those requiring hospitalization, urgent healthcare visit, or hemin administration, in patients with AIP over six months.
At six months, givosiran met the primary endpoint in patients with AIP (p less than 0.00000001). All the components of the composite primary endpoint and all subgroup analyses for the primary endpoint favored givosiran. Secondary endpoints listed below also demonstrated statistically significant favorable differences in the givosiran arm compared to placebo (p less than 0.0001):
The remaining four secondary endpoints did not meet the hierarchical threshold for significance and included: daily worst pain (p equal to 0.053), daily worst fatigue (p equal to 0.29), daily worst nausea (p equal to 0.25), and the physical component summary of the SF-12 health survey in AIP patients (p equal to 0.022).
Adverse events (AEs) were reported in 43/48 (89.6 percent) of givosiran and 37/46 (80.4 percent) of placebo patients and serious adverse events (SAEs) were reported in 10/48 (20.8 percent) of givosiran and 4/46 (8.7 percent) of placebo patients. There were no deaths in the study. One patient, described below, in the givosiran arm (2.1 percent) discontinued treatment due to an AE. AEs reported in greater than 10 percent of givosiran patients and seen more frequently compared to placebo were: nausea, injection site reactions, chronic kidney disease and fatigue; those reported in greater than 10 percent of placebo patients and seen more frequently than in givosiran-treated patients were headache, urinary tract infection, vomiting and pyrexia. The AEs of chronic kidney disease were reported in five givosiran-treated patients (10.4 percent) and no placebo patients; these events were all in patients with renal dysfunction at baseline and patients continued dosing throughout the study. Liver transaminase increases greater than three times upper limit of normal (ULN) were observed in 7/48 (14.6 percent) patients on givosiran and 1/46 (2.2 percent) patients on placebo; all had evidence of iron overload or liver disease at baseline. As previously reported and as noted above, one patient on givosiran discontinued treatment due to an increase in alanine aminotransferase (ALT) levels greater than 8 times ULN, a protocol-defined stopping rule; this elevation did not meet Hy’s Law and subsequently resolved. Peak ALT levels in the other six givosiran-treated patients ranged from 3.0-5.4 times ULN and were not accompanied by bilirubin increases. The patients were asymptomatic, and all events resolved with continued dosing (n=5) or after a brief pause in dosing (n=1).
Upon completion of dosing in the ENVISION double-blind period, all eligible patients (93/94 or 99 percent) enrolled in the ENVISION open-label extension (OLE) study, receiving monthly givosiran administration. In addition, patients continue dosing in the Phase 1/2 OLE study with over two years of exposure to givosiran.
Source: Alnylam
Virtuvax Reports Positive First-in-Human Results of Nasal Spray RSV Vaccine
GezondheidVirtuvax and the trustee of biotech company Mucosis, Mr. Holtz LLM announced the results of a first-in-human study of the intranasal Respiratory Syncytial Virus (RSV) vaccine candidate, SynGEM. The study was executed by Imperial College London.
Results of the study have been posted (in press) on the website of the American Journal of Respiratory and Critical Care Medicine. The vaccine was found to be safe and immunogenic in healthy adults.
“Virus-specific immunogenicity induced after SynGEM vaccination was demonstrated by systemic IgG+ and IgA+ responses in terms of antibody-secreting cells (ASCs)” indicated prof. Openshaw, professor of Experimental Medicine at Imperial College London. Professor Openshaw added that “SynGEM induced some RSV-specific mucosal IgA in most individuals, particularly in those with low pre-existing F-specific IgA titers. Indeed, higher doses of the vaccine induced prolonged IgA-producing ASC responses in some volunteers.” Prof. Openshaw further explained that “The publication includes in vitro data supporting the clinical observations, showing that SynGEM induces a dose-dependent antibody response associated with cytokine production from T-helper cells.”
Dr. Chiu, principal investigator of the clinical trial, commented: “Previously, we showed that antibodies induced by natural RSV infection were only short-lived and we hypothesized that RSV-induced immunomodulatory mechanisms impaired the antibody responses against the virus. In this study, SynGEM not only induced immune responses but also boosted long-term systemic RSV-specific antibodies for at least 6 months. Vaccination with the SynGEM RSV vaccine therefore was not associated with evidence of immune modulation, as seen after infection with live RSV.”
In another publication also in AJRCCM, senior author Prof. Louis Bont of University Medical Center Utrecht, The Netherlands commented on the SynGEM trial. Prof. Bont argued that in the absence of a correlate of protection to RSV, vaccine developers are working in the dark. In the case of SynGEM, preclinical data had shown protection against infection suggesting evidence of the vaccine providing protective immunity. However, despite the induction of broad immune responses, neutralizing antibodies were not detected in the clinical trial. In their comment, the authors suggested that testing of the SynGEM vaccine in a human challenge model might be a quick way to finally determine proof of concept of efficacy.
The investigators of Imperial College were surprised that no detectable neutralizing antibody responses were found, since extensive stability tests had shown stable binding of such antibodies by SynGEM. Openshaw said: “the system needs further optimization, but establishes proof of principal that the nasal vaccine approach can work. It was a shame that the programme was ended before the infection challenge component could be performed”. Trustee of Mucosis, Mr. Holtz, commented: “During the clinical trial, Mucosis was confronted with a sudden shortage of finances and the company had to apply for bankruptcy before the final results were known as published today. Since these long-term data have become available as of now, any further development of the vaccine may be taken forward by other companies interested in doing so.”
SynGEM is based on a unique pre-fusion version of the F subunit of RSV, shown to raise more potent serum neutralizing antibodies against RSV compared with the post-fusion F antigen approach others have used. The pre-fusion conformation (pre-F), which predominates on infectious virions, displays a
distinct antigenic site (site Ø) preferentially targeted by the most potent neutralizing antibodies. Preclinical studies using SynGEM showed induction of high levels of both systemic and mucosal antibodies.
Delivered via the mucous membranes in the nose, the vaccine candidate is also able to recruit antibodies produced in the mucosal linings of the body, with the potential to effectively stop the virus from entering the body via the mucosal pathways, where over 90% of pathogens enter the body.
SynGEM is based on Mucosis’ patented Mimopath technology, which uses bacterium-like particles (BLP) derived from food-grade bacteria, to deliver the antigen in a more natural conformation and boost the body’s immune response to the virus.
RSV is a common contagious infection of the lungs and respiratory pathways, eliciting cold-like symptoms in adults. Whilst RSV infection is normally mild in healthy adults, the infection can lead to serious and sometimes fatal diseases such as pneumonia and bronchiolitis, most often in susceptible populations such as the young and elderly. Every year in the US, RSV infections lead to 57,527 hospitalizations of children under 5 year’s old (2)and are responsible for up to 200,000 deaths annually worldwide (3). The annual global burden of RSV illness is significant, with 33.8 million estimated new episodes of RSV-associated acute lower respiratory infection (ALRI) worldwide in children under 5, and over 3.4 million hospital admissions associated with severe RSV disease. Global mortality was estimated at 253,500 deaths in 2010.
The Wellcome Trust translational fund supported the clinical trial of SynGEM with a staged award of up to €3.44 million (£2.77 million).
Source Virtuvax
BiosanaPharma gets approval to start phase I clinical trial for a biosimilar version of omalizumab
GezondheidBiosanaPharma has received permission from the Australian Bellberry Human Research Ethics Committee HREC to start a phase I clinical trial of their first pipeline product. The trial consists of a bioequivalence double-blind, randomized, two-parallel-group phase I study of a humanized anti-immunoglobulin E monoclonal antibody ‘BP001’ as lyophilized powder formulation compared with the standard omalizumab (Xolair®) lyophilized powder formulation in healthy male volunteers. Prior to the submission of the Investigative Brochure to the HREC an extensive comparability exercise was performed between BP001 and Xolair®, indicating functional, binding and structural similarity between the two molecules. This extensive laboratory exercise was also sufficient for Biosana to obtain a waiver for preclinical studies in animal models from the EMA.
The Phase I trial is expected to be concluded and fully reported in Q4 2019.
Ard Tijsterman, CEO of BiosanaPharma: “We are extremely happy and proud that we’ve been able to develop our first biosimilar product, produced by our innovative ‘3C process’, the first fully continuous process to deliver a product entering phase I. We will start negotiations with potential marketing & sales partners now to perform phase III studies and secure future global distribution of the product.”
Omalizumab
Omalizumab is marketed by Roche/Genentech (USA) and Novartis (ROW) as Xolair® since 2006. It is used to treat moderate to severe allergic asthma and chronic idiopathic urticaria in patients aged 12 years and above in the US and the EU. Additionally, it is approved for paediatric patients aged 6 to 11 years in the EU. Xolair® is delivered via subcutaneous injections. Total sales for Xolair® in 2018 were US$ 2.9M with a consistent growth in both indications of 11-13% annually. The US market accounted for roughly 2/3 of total Xolair® sales. The patent on Xolair® has expired in 2017 in the US and Europe (Novartis, 2016).
BiosanaPharma
BiosanaPharma is a Dutch/Australian biotechnology company with a disruptive approach to the manufacture and delivery of monoclonal antibody therapeutics (mAbs). BiosanaPharma’s “3C process” offers a 90% reduction in the cost of developing and manufacturing EMA/FDA quality mAbs. The 3C process is a fully-continuous, small footprint platform and produced BiosanaPharma’s first pipeline drug BP001, now approved to enter Phase I (see above).
BiosanaPharma is also developing a platform for oral delivery of biologicals, which has delivered successful preclinical results. The current pipeline consists of biosimilars and oral versions of blockbuster biologics.
Source: BiosanaPharma
Kidney-on-a-Chip in Nature Biotechnology
GezondheidScientists from the Hubrecht Institute, the University of Utrecht, and MIMETAS have successfully grown kidney tubules derived from human kidney adult stem cells in microfluidic chips. This research has been published in Nature Biotechnology by the world-leading research group from Professor Hans Clevers.
Henriette Lanz, Ph.D., Director of Biology at MIMETAS and co-author on the paper explains: “Scientists at the Hubrecht Institute managed to reprogram stem cells found in the human kidney cortex to cells that are similar to tubular cells of the kidney. From these cells, my team has grown three-dimensional perfused kidney tubules. We have discovered that these tubules are fully polarized, meaning that they distinguish inside from outside, just like in a real kidney. Moreover, the biologically important barrier function of the kidney tubes is intact. We show that transporter activity is functional, which is a hallmark of kidney tissue, responsible for pumping of nutrients and toxicants across the kidney barrier. We can grow 40 of such tubules in one single OrganoPlate®. The kidney tubes can be used to create disease models that allow the development of novel therapies. This novel technology offers a powerful human alternative to animal testing for toxic side effects of new medicines.”
A mini kidney from the lab doesn’t look like a normal kidney, but the simple cell structures share many of the characteristics of real kidneys. Researchers can use them to study certain kidney diseases. “We can use these mini kidneys to model various disorders: hereditary kidney diseases, infections, and cancer. This allows us to study in detail what exactly is going wrong”, says Hans Clevers, Professor of Molecular Genetics at Utrecht University and the University Medical Center Utrecht, and group leader at the Hubrecht Institute.“This helps us to understand the workings of healthy kidneys better, and hopefully, in the future, we will be able to develop treatments for kidney disorders.”
Read the full Nature paper here: https://www.nature.com/articles/s41587-019-0048-8
Source: MIMETASand Hubrecht institute
Enpicom secures funding round to finalize igx platform development
InnovatieklimaatBioinformatics software engineering company ENPICOM has closed a series A investment round. BOM Brabant Ventures, Nextgen Ventures and Arches Capital have jointly invested 1.2 million euros to support the company’s repertoire sequencing data analysis platform. ENPICOM will use these proceeds to finalize development of its ImmunoGenomiX (IGX) platform and introduce it in the marketplace.
ENPICOM BV was founded in 2017 by Dr. Alvise Trevisan (CTO), Dr. Nicola Bonzanni (CSO) and Jos Lunenberg, MSc, MBA (CEO). Mid 2017 the company started the development of a unique platform to manage, store, analyze, visualize and interpret clone sequencing data from T and B cell receptor repertoires. ENPICOM is currently in the final stage of preparations for the global launch of the first full version of its ImmunoGenomiX (IGX) platform. This festive event will take place in Vienna at the 6th Immunotherapy of Cancer Conference (ITOC6) from April 11 to 13.
Dr. Bonzanni commented: “When studying immune system related diseases or developing drugs influencing this system, it is extremely important to be able to directly measure and closely monitor its status. Repertoire sequencing is a powerful way to analyze hundreds of thousands to millions of T or B cell receptors swiftly and in a cost-effective way.” “While there are different companies offering kits and service for this specific form of targeted sequencing, there is no commercial platform to manage the rapidly growing amounts of data generated in the lab in an intuitive, user-friendly way,” added Dr. Trevisan. He continued: “To get from data to powerful insights from this exciting technology, smart new analysis methods and rock-solid software were needed. We have built a team of experts, bringing together a wealth of knowledge and expertise in this field, to develop the IGX platform.”
“I am very pleased we have attracted this excellent syndicate of investors,” said CEO Lunenberg. “With the completion of this Series A financing, we are well funded to execute on our IGX development and market introduction programs.”
Quotes from the investors
Sjoerd van Gorp (BOM): “The ambitions of the ENPICOM team in the field of personalized immunotherapy fits perfectly with our investment goals. By providing innovative life sciences companies and healthcare providers with the tools to measure the status of the patients’ immune system we believe that ENPICOM can facilitate efficient drug development and ensure effective treatment for patients.”
Matthijs Blokhuis (NextGen Ventures): “The ambition of ENPICOM to personalize immunotherapy resonates extremely well with the objective of NextGen Ventures to provide meaningful results for patients and providers. An increased understanding of immune repertoire sequencing data significantly helps to improve outcomes in an efficient manner.”
Eric van der Maten (Arches Capital): “ENPICOM is a perfect example of what we look for in a portfolio company: a strong, highly skilled, motivated and dedicated team, that is showing the ability to combine innovative emerging technologies like data science, AI and medical science into an exhilarating and fast-growing Deep Tech company, eventually even with a real impact on society. We are very pleased to add ENPICOM to our investment portfolio”.
Source: ENPICOM
Gezondheidsraad: varen op wetenschap
Gezondheid op maat, Hollandbio, GezondheidMet een interview in haar nieuwsbrief Graadmeter mengt ook de Gezondheidsraad (GR) zich in het maatschappelijk en politiek debat over vaccineren. Prof. Gerhard Zielhuis, voorzitter van de Commissie Vaccinaties, licht in dit interview onder andere het beoordelingskader en de rol van de GR omtrent de dalende vaccinatiegraad toe. HollandBIO vindt het top dat ook de GR, als wetenschappelijke hoeder van onder meer het Rijksvaccinatieprogramma, zich nu in de discussie mengt.
Het interview sluit mooi aan op het plenaire debat over vaccinatie in de Tweede Kamer, twee weken geleden. Middels een motie droeg de Kamer Staatssecretaris Blokhuis op om geregistreerde vaccins direct te voorzien van een advies over opname in een programma. Een oproep die bij HollandBIO met gejuich ontvangen werd. De Staatssecretaris is nu aan zet.
HollandBIO is van mening dat ook vaccins die (nog) niet voldoen aan de criteria voor opname in een vaccinatieprogramma of het verzekerd pakket, een route op maat verdienen. Nu belanden die vaccins vaak ongebruikt op de plank, al voegen ze op individueel niveau nog zoveel waarde toe. Hierdoor blijft gezondheidswinst liggen. HollandBIO is pas tevreden als hier een oplossing voor komt en we vaccins optimaal benutten in de strijd tegen infectieziekten.
Nanobuisjes: doorbraak in plantenveredeling
Wereld mét toekomst, Voedsel en materialenHet gericht veranderen van het plantengenoom is niet makkelijk. Het moeilijkste is om het DNA of RNA in de plantencel te krijgen. Daar wordt vaak letterlijk grof geschut voor gebruikt, een methode die ‘shotgun’ heet. Het DNA met behulp van bacteriën in de cel brengen is subtieler, maar ook aan deze methode kleven verschillende nadelen. Zo blijven er altijd ‘sporen’ van het bacterieel DNA aanwezig. In Europa worden beide methoden als genetische modificatie beschouwd. Twee Amerikaanse onderzoeksgroepen hebben nu – onafhankelijk van elkaar – een briljant alternatief gevonden.
De onderzoekers brengen het DNA in de cel via nanobuisjes gemaakt van koolstof. Deze buisjes zijn zo klein dat de plantencel ze met het water opnemen. Om het genoom van de plantencel te kunnen veranderen moeten de buisjes vervolgens nog doordringen in de celkern. De onderzoekers hebben er vertrouwen in dat dat ook gaat lukken.
De echte doorbraak voor de plantenveredeling zal komen als de onderzoekers er in slagen om de onderdelen van het CRISPR-Cas systeem via deze nanobuisjes de cel in te krijgen. Dan kun je DNA gericht aanpassen terwijl je de plant bewatert. Diverse landen zien het aanpassen van DNA via CRISPR-Cas niet als genetische modificatie, waarmee de methode daar laagdrempelig en breed te benutten is. Helaas is dit in Europa niet het geval. Gelukkig laat de Nederlandse regering het hier niet bij zitten en zet zij zich in om ook in Europa CRISPR-Cas vrij te stellen van de GMO-richtlijn en breed toegankelijk te maken voor de duurzame productie van gezond voedsel.
Bron: Trouw
Dure medicijnen: wat klopt er van de argumenten van de farmaceutische industrie?
Gezondheid op maat, GezondheidAfgelopen week publiceerde de Volkskrant een verfrissend gebalanceerd artikel over de totstandkoming van geneesmiddelprijzen. Zeven argumenten van de sector om die prijzen te verklaren passeren hierin de revue. Biogen en Roche grepen deze kans aan en gingen open en eerlijk het gesprek aan over de complexiteit van en uitdagingen in de ontwikkeling en prijsstelling van geneesmiddelen. Een dikke pluim voor Biogen, Roche én de Volkskrant. HollandBIO hoopt dat dit goede voorbeeld breed navolging krijgt. Voor nu: een absolute must-read!
Tweede Kamer: “Sleur Zorginstituut de 21ste eeuw in”
Gezondheid op maat, Hollandbio, GezondheidFantastisch nieuws: de hele Tweede Kamer roept de regering op te laten onderzoeken hoe de werkwijze van het Zorginstituut beter kan aansluiten op de nieuwe regeling voor weesgeneesmiddelen, voorwaardelijk toegelaten geneesmiddelen en op de beweging naar personalised medicine. Vandaag stemde de Tweede Kamer unaniem voor een motie van de leden Carla Dik-Faber en Pia Dijkstra. De methodiek om de effectiviteit van de nieuwste generatie geneesmiddelen vast te stellen is achterhaald en één van de knelpunten die hoog op de ‘te tackelen’ prioriteitenlijst van HollandBIO staat. We zijn dan ook erg blij dat het opheffen van deze blokkade op de weg van lab naar patiënt nu op de Haagse agenda staat.
De methodologie en de criteria die het Zorginstituut gebruikt om te bepalen of een geneesmiddel tot “de Stand der Wetenschap en Praktijk” (SW&P) behoort, GRADE genaamd, plaatsen de gouden standaard voor klinisch onderzoek, gerandomiseerde klinische trials (RCT’s) met grote groepen patiënten, op een voetstuk. En dat systeem loopt steeds verder vast nu biotech bedrijven steeds vaker geneesmiddelen of therapieën ontwikkelen tegen zeldzame ziekten, voor subpopulaties of zelfs voor individuele patiënten. Het is namelijk onmogelijk om aan de oude gouden standaard te voldoen. Allereerst zijn er simpelweg onvoldoende patiënten om in een trial te includeren. Ten tweede kan het langdurig continueren van dubbelblind gerandomiseerd onderzoek onethisch zijn, omdat je een groep ernstig zieke patiënten de enige mogelijke behandeling onthoudt.
Het gevolg: op het moment dat het Zorginstituut zo’n geneesmiddel met GRADE beoordeelt aan de hand van de criteria van het verzekerd pakket, krijgt de onderliggende dataset het stempel ‘niet robuust’. Dit stempel werkt negatief door in de hele beoordeling, zowel voor het vaststellen van de therapeutische waarde als voor kosteneffectiviteit. Na het jarenlange, kostbare en risicovolle ontwikkelingstraject van een geneesmiddel, bereikt de behandeling de patiënt in het zicht van de haven niet. Niet omdat het niet effectief is, maar omdat de methodiek het innovatietempo van de biotech sector niet bij kan benen.
Kortom: de oproep van de Kamer om het Zorginstituut indien nodig de 21ste eeuw in te slepen, is weliswaar een eerste stap, maar desalniettemin een mijlpaal. Het erkennen en aanpakken van dit methodologische probleem is niet alleen cruciaal voor de realisatie van zorg op maat, maar kan van Nederland een innovatieve en internationaal toonaangevende proeftuin voor personalised medicine maken.
Evaluatie Besluit GGO: maak Nederland koploper in gen- en celtherapie
Wereld mét toekomst, Hollandbio, GezondheidHollandBIO ziet de lopende evaluatie van het Besluit GGO als een uitgelezen moment om een prangend knelpunt in het Nederlandse biotech beleid weg te nemen: de inschaling voor cel- en gentherapie. Het Besluit GGO ziet toepassing van cel- en gentherapie op het moment als introductie in het milieu. HollandBIO pleit voor inschaling van deze therapieën als ingeperkt gebruik. Alleen dan kan Nederland de vruchten plukken van deze aanstormende nieuwste generatie therapieën.
Naast dit knelpunt bracht HollandBIO een aantal andere verbeterpunten onder de aandacht om het Besluit en de uitvoering daarvan te verbeteren. Deze reiken van de introductie van een zelflerend vermogen, waarbij opgedane kennis en ervaring wordt meegenomen in de uitvoering tot de verbetering van de informatievoorziening op de website van bureau GGO. Lees hier onze volledige inbreng.