Eurobench.com

flosz 15 mei 2015 17:59

0

PIPER #ASGCT15 Day2 takeaways
$BIIB $AAVL $QURE $BLCM $CLLS $SGMO $LBIO $BLUE
"Gene Editing Safe, So Far"
t.co/Q5nXloGyCG

flosz 18 mei 2015 13:34

0

PIPER:
During the final 2 days of the ASGCT annual meeting, we continue to be impressed
by the speed at which the gene therapy and gene editing field continues to evolve and
iterate. In a particularly well attended presentation, additional academic data from the
ongoing St. Jude's / UCL trial for gene therapy in hemophilia B was presented. Overall,
we highlight several of our key takeaways from the conference, with companies including
BIIB, AAVL, QURE, BLCM, CLLS, LBIO, BMRN and BLUE all with exposure. We will
address many of these topics and findings at our GenomeRx Symposium this week in
NYC.
• Hemophilia B Data Continues To Impress: Data from the ongoing trial being
conducted at St. Jude's / UCL was presented disclosing additional FIX expression data
in treated patients, one of which goes beyond 5 years with many patients now 3-4 years
post infusion. Patients continue to see robust and durable FIX expression (2-7% in
high dose) with seemingly no sign of waning, in contrast to the recent NEJM paper
describing a waning of effect after 3 years in LCA patients. This continues to highlight
the curative potential of liver-directed gene therapy. Immunosuppression remains an
active topic of debate, and we note that 2 additional patients in this trial have been
treated with a vector preparation designed to remove empty capsids, with no sign of
immune response seen post infusion. In addition, work with modified FVIII variants
for hemophilia A was presented (for additional detail on the challenges of FVIII and
preliminary work in new FVIII variants, see our December deep dive) and is expected
to enter the clinic in 2016. We note that BMRN has licensed technology from UCL and
is entering the clinic imminently and we look forward to further updates and details
on these approaches. Interestingly, the hemophilia A gene therapy preclinical program
presented by UCL used AAV5, which could give BMRN freedom to operate if it kept
this vector. It also utilized a B-domain deleted construct with partial-add back of a
site which seems to meaningfully increase Factor production.
• Process improvements will come in all forms: One factor which we have
underestimated is the ongoing improvement to the manufacturing processes for viral
vectors and adoptive immunotherapy. Presentations at ASGCT focused on details all
the way down to the buffer selected. While vector selection and tropism are obvious
determinants of clinical success, we are also starting to appreciate that gains in
manufacturing may be equally impactful in furthering the clinical profiles of these
products.
• Key Advancements Drive The Field Forward: We believe several factors are
contributing to the rapid pace of innovation: advances in genetics and affordable gene
sequencing (which will also help identify patients for trials and therapies), creation
of new and improved animal models and better disease pathway elucidation, biology
techniques like cell-sorting and electroporation, and academic centers pivoting to be
more commercial and disease oriented (including advanced manufacturing facilities).
Companies also continue to learn from each other with respect to key variables,
including systemic administration and immune suppression, dosage, vector serotype
and evolution, etc.. All of this will allow earlier de-risking of clinical programs. We also
believe the field has learned from prior setbacks and is moving forward thoughtfully
and carefully.

Which All Bodes Well For The Commercial And Investment Potential: As gene therapy
continues to evolve as a platform, we therefore expect that access to capital will
remain robust and that return on investment will remain favorable. While we expect
an iterative path with both successes and failures along the way, we note that investors
are becoming more comfortable with gene therapy and increasingly understand which
variables are driving results, evaluating both positive and negative trials on their
own considerations. Overall, gene therapy has truly begun to unlock the vast orphan
disease market as well as make inroads in larger markets, and the potential benefits
of gene therapy are broadly applicable.

bridgetunnelinvestor.freeforums.net/t...

flosz 18 mei 2015 13:43

0

Suntrust:
ASGCT Days 3&4; Bright Future for Hemophilia,
CAR T Activity Remains Impressive
What's Incremental.
The final days of the ASGCT meeting featured updates from a Phase I trial of AAV-based gene therapy for hemophilia B (conducted by UCL/St. Jude). As BMRN in-licensed gene therapy technologies for hemophilia A from these academic centers, we believe the ~5 year persistence of hemophilia B gene therapy in this updated Phase I trial bodes well for BMRN's BMN 270 (data in Q4 or Q1 2016) - currently
upside to our estimates. We like BMRN ahead of BMN 111 results in achondroplasia (end-Q2) and other H2 catalysts. In addition, CAR T cells continue to impress on the ALL efficacy front.
Updates from a University College London/St. Jude's clinical trial in hemophilia B bode well for BMRN's program. Dr. Amit Nathwani (UCL, London, UK) discussed a Phase I study of AAV8-induced expression of clotting Factor IX (FIX) in 10 patients with hemophilia B (longer follow up to data previously published in November 2014 in the New England Journal of Medicine, NEJM). In line with previous disclosure, all patients achieved 1-5% FIX expression post gene therapy, sufficient to enable significant reduction in bleeds associated with hemophilia B. A key observation for the outlook of gene therapy approaches has been immunogenicity elicited by the AAV8 capsid, resulting in liver toxicity as evidenced by liver enzyme (ALT) elevations, and reduction in FIX levels post treatment. Management with immunosuppressants (i.e. prednisone) in patient 5, as soon as ALT elevations occurred, resulted in a less dramatic decrease in FIX levels. In addition, preventative prednisone dosing in patients 6-10
precluded ALT increases and loss of gene therapy-induced FIX production. Key updates to the NEJM article from patients 5-9 illustrate maintained FIX production at years 4, 4.5, 3, 2.5, and 2 (versus 3,
3.5, 2, 2.5, and 1.5 in the NEJM article, respectively). The sustained FIX production was associated with reduced need for FIX prophylaxis and significant improvement in the quality of life. In addition, Dr.Nathwani noted that in non-human primates, a single administration of FIX gene therapy is associated with persistence at ~10 years follow-up. We note BMRN's BMN 270 program for hemophilia A (to enter
the clinic in the summer) is based on technology licensed from UCL and St. Jude's Research Hospital, and believe the durability of response seen in the hemophilia B study bodes well for the outlook of BMN 270 (albeit a larger gene for hemophilia A).

Two presentations discussed approaches to overcome challenges to designing a successful hemophilia A gene therapy. Per Dr. Nathwani's presentation, several challenges render hemophilia more difficult to address compared to hemophilia A. A second presentation was delivered by Dr. Denise Sabatino (Children's Hospital of Philadelphia, collaboration with ONCE) and fully dedicated to this topic.
First, the defective protein in hemophilia A, factor VIII (FVIII), is encoded by a large gene, which exceeds the packaging capacity of AAV vectors. Thus, different investigator groups have generated truncation constructs of FVIII that appear to successfully recapitulate FVIII activity in animal models of disease (this
also appears to be the case with BMRN's FVIII construct entailed by BMN 270, per data presented at the company's R&D Day in December 2014). Second, compared to proteins of the same size, the production of FVIII is highly inefficient in animal/human cells or in vivo. Truncation of FVIII appears to also influence
production (as the deleted portion likely include genetic information for ineffective FVIII production), as well as the stability and the potency of FVIII. Thus, we believe that different optimization techniques for FVIII truncation are likely to differentiate the multiple programs in development.
A presentation of CD19-28z CAR T's highlights the efficacy of this approach in adult ALL, questions remain on toxicity. Updated results from a Phase I study of JUNO's (not covered) CD19-28 CAR T cells were presented by Dr. Jae Park (Memorial Sloan Kettering Cancer Center) as a "top
abstract". As of 03/03/15, 35 adult patients with highly refractory acute lymphoblastic leukemia (ALL) had been treated with CD19-28z CAR T cells (34 patients evaluable for a response with >1 month follow up).
30 (88%) patients achieved a complete response (CR), while 24 (83%) out of 29 patients evaluated for minimal residual disease achieved negativity. These data compare very well with historical trials, where 18-20% patients achieved a CR. The median overall survival was 8.5 months in all patients, and 10.5 months in the responders. T cells persisted 1-3 months following infusion. The main concern related to CAR T therapies appears to be safety, including cytokine release syndrome (CRS, fever, hypotension, respiratory insufficiency) and neurological toxicities (delirium, encephalopathy, seizures). 7 (20%) of the patients experienced CRS, and 10 (29%) experienced neurotoxicity (not correlated to CRS). CRS
was managed with IL-6 inhibitors while neurological toxicities were associated with disease burden and were reversible. We view these data as exciting, given the very low historical rates of response in this aggressive blood cancer, and look towards future optimization/improved management of the adverse events associated with CAR T therapy.
bridgetunnelinvestor.freeforums.net/t...
mobile.twitter.com/captainfuture__

flosz 19 mei 2015 14:13

0

Uit Griekenland.

Health: The challenge of the 21st century
As the population of the earth increasingly "aging" raised questions about the sustainability of health systems. What are the new challenges they face and how technology can be a solution?

Currently several efforts intensified in the direction of reducing the cost of research for the development of new drugs - which is currently estimated at an average of 2.5 billion. Dollars.A typical example is the debate has erupted on the case of Glybera, the first gene therapy approved in the western hemisphere. Essentially Glybera offers permanent cure rare disease lack lipoprotein lipase (LPLD) with patients suffering from the disease are prone to recurrent pancreatitis episodes.

The problem; The cost of treatment, which reaches 780,000 euros for each patient. The Dutch company uniQure that created justify the price by referring to the high cost of research and stressing that the respect Glybera gene intervention that will bring permanent cure. Of course this should be emphasized that the lack of disease lipoprotein lipase (LPLD) relates only 150-200 patients in Europe, so the company addresses a very small audience, which should cover the cost of developing the drug.

translate.google.com/translate?hl=en&...

flosz 19 mei 2015 15:00

0

Bluebird, Regulators Map Out Approval Plan For Gene Therapy

One of gene therapy’s major unanswered questions is just what it’ll take to convince U.S. regulators to approve one of these treatments. With a gene therapy that’s already produced promising results in a few patients in clinical trials, Bluebird Bio has a chance to pave the way. And today it’s cut a deal with the FDA to figure out how to get that treatment to the regulatory finish line.

Bluebird (NASDAQ: BLUE) said this morning that it’s come to an agreement with regulatory agencies both in the U.S. and in Europe on an approval path for LentiGlobin, its gene therapy for beta-thalassemia, a crippling blood disorder. The agreements map out potential approval on an accelerated basis for the therapy—based on less of a body of data than is typically required.

If Bluebird were to succeed, it would have the chance to own the first gene therapy approved in the U.S., a major milestone for the field. Only Europe has an approved gene therapy, Glybera, from Uniqure (NASDAQ: QURE) for a rare metabolic disorder, and that product hasn’t been launched yet.

Approval is still a ways away, of course. To this point, Bluebird has produced very promising data, but in just four patients. With past gene therapies falling short of expectations in clinical trials, a lot more proof is needed. And then there’s the question of how much LentiGlobin would cost (would it be paid for in one large, lump sum? On an annuity basis?).

Nonetheless, Bluebird has an unusual opportunity in front of it. Here’s what the Cambridge, MA-based company needs to do to get LentiGlobin to market:

In the U.S., Bluebird will have to run two additional trials, called HGB-207 and HGB-208. These are each 15-patient, open-label studies—not placebo-controlled, randomized trials. Adults and adolescents with beta-thalassemia will be enrolled in HGB-207, and children in HGB-208. Bluebird will track these patients’ results for two years, and the goal will be to free the subjects of the blood transfusions they normally require to prevent anemia for 12 months.

Bluebird says that based on its talks with the FDA, data from these two studies and the two existing trials underway (known as Northstar and HGB-205), will form the basis for a regulatory filing. Northstar and HGB-205 are enrolling patients in the U.S. and Europe, respectively, with either beta-thalassemia or sickle cell disease. If LentiGlobin hits its goal, Bluebird believes it could seek accelerated approval, and conduct post-approval long-term follow up studies of these patients.

In Europe, Bluebird has been working with the European Medicines Agency and others, and based on those discussions, believes that it can win conditional approval of LentiGlobin based on the data from HGB-205 and Northstar studies alone. That conditional nod could be converted to a full approval should Bluebird successfully complete HGB-207 and HGB-208, and obtain more long-term follow-up and post-approval monitoring data.

This feedback brings us closer to achieving our vision of delivering one-time, potentially transformative gene therapy to patients,” Bluebird chief medical officer David Davidson said in a statement.

Bluebird is holding a conference call this morning to discuss the news.

Patients with beta-thalassemia inherit a faulty gene that codes for beta globin, a subunit of hemoglobin, the protein in red blood cells that carries oxygen. Without the ability to make hemoglobin, these patients get severe anemia and need frequent blood transfusions to survive. They also need iron chelation therapy to deal with the iron overload those transfusions cause; that buildup of iron often kills patients.

About 15,000 people in the U.S. and Europe and 280,000 worldwide are estimated to be living with the disorder, and a majority of them have beta-thalassemia major, the severe form, according to Bluebird.

www.xconomy.com/boston/2015/05/19/blu...

Prof. Dollar 19 mei 2015 22:21

0

Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA - www.nasdaq.com/press-release/sarepta-...

flosz 19 mei 2015 22:22

0

Biotechnology Trends: What The Heck Are CAR-T And CRISPR?
May 19, 2015 12:29 PM

This blog won't attempt to do a deep dive into these emerging biotechnology trends. Frankly I'm still learning about them myself. As an alternative, I'll take the lead from a Quantum Physicist, Gemma Godfrey, and her refreshing TEDxWallStreet speech: How To Kiss. In short this blog will simply touch on the techniques and then put them in context. Seems like an appropriate first step to help investor's identify rather than avoid emerging opportunities.
CRISPR ("clustered regularly interspaced short palindromic repeats") is a form of gene editing. This Wikipedia image is confusing enough to show that a blog on the science would not be helpful to most investors.
CAR-T ("Chimeric Antigen Receptors") is also a form of gene manipulation which attempts to engineer a patient's own immune system (T-cells) to fight their cancer. Dr Renier Brentjens (Sloan Kettering Cancer Center) describes this type of adoptive cell transfer as "giving patients a living drug".
Both of these approaches are high-risk, early-stage approaches that have numerous hurdles yet to overcome. However they are rapidly gaining momentum and endorsement in the medical community.
Genomic alternation has been discussed for years as genomic sequencing has become a reality. Many view this in a very negative light, worrying the genetic equivalent of plastic surgery will become the norm. Consider a recent MIT Technology Review article well worth a read: Genome Surgery. I loved the title as it quickly framed the issue. Society has long accepted surgery as a medical option without reservation, even though cosmetic surgery is considered by some as questionable. The morality issues raised are not insignificant, but the potential benefits far outweigh them.
Another article well worth reading is: Human Ingenuity Takes on Cancer's Darwinian Ways, published in the New York Times. This article draws a parallel between evolutionary aspects of cancer and immune cells. It also states: "scientists have treated some patients by extracting their immune cells and re-engineering them to fight tumors. Sometimes, the cancer cells themselves are used to develop a custom vaccine, immunizing a body against a destructive subset of itself." Sounds consistent with the National Institute of Health Precision Medicine Initiative to me.
Analysts are actively publishing reports on the trends. Piper Jaffray issued a subscription-only report today: Gene Editing Primer: New Tools Emerging To Do Great Things. In this report they state:
Simply put, gene editing platforms are genetic scissors which can cut DNA and lead to knockout or correction or insertion of genetic sequences.
Progress is already being made deploying gene editing platforms to knock out: 1) the CCR5 receptor to treat HIV, 2) the BCL11a enhancer to treat hemoglobinopathies, and 3) T-cell receptors and other proteins to enable....enhanced CAT-T and TIL products.
The value of precisely correcting DNA is immeasurable if accomplished with precision and control...while debate rages (in utero germline correction of disease), the platforms continue to evolve and improve in a direction where someday this should be feasible.
Overall, we believe we are officially in an era of gene therapy 2.0 and the time has arrived for gene editing.
Leerink published their insights on What's Next in Gene Therapyfrom the American Society of Gene & Cell Therapy (5/13-5/13) annual meeting. Their takeaways:
There was significant interest in improving gene transfer technologies.
There was a major focus on gene editing platforms, with a keen interest in differentiating the various approaches.
Incremental color provided by the Dr Nathwani update on the hemophilia B rial at UCL can be interpreted positively for QURE and others.
We view the presentation of regulatory expectations for gene and cell therapies as optimistic.
In summary these approaches are a natural extension of genomic sequencing and have been anticipated for years. There are many obstacles and moral dilemmas still on the path to success. Yet the trends are clear: Genomic editing (aka surgery) is an exciting trend in biotechnology and well worth the effort to identify and invest in the best of breed companies leading the charge.
Disclosure: The author is long KITE, JUNO, QURE, BMRN, PFE, BIIB, XON.
seekingalpha.com/instablog/400846-mar...
twitter.com/mchilberg

flosz 9 juni 2015 19:24

0

arGEN-X: la petite soeur surdouée d'Ablynx et Galapagos ? - mon analyse à lire: t.co/RiKnWexUQ8
mobile.twitter.com/logribel

Bijlage:

flosz 10 juni 2015 09:59

0

ArGEN-X: Forging The Future Of Antibodies
seekingalpha.com/article/3243776-arge...

flosz 10 juni 2015 21:52

0

When your genome costs less than your iPhone: The beautiful, terrifying future of DNA sequencing
www.techrepublic.com/article/when-you...

flosz 12 juni 2015 19:48

0

17 reasons to ban wood burning

www.ehhi.org/woodsmoke/utah_17reasons...

flosz 13 juni 2015 08:26

0

Regels frustreren revolutie in farmacie
June 12, 2015

Risicovrees en cententellerij zijn fnuikend voor farmaceutische innovaties, betogen de hoogleraren Van Deventer, Girbes en Singer.
We staan aan de vooravond van grote innovaties op het gebied van medische behandelingen. Europa speelt een belangrijke rol bij de doorbraak van nieuwe medicijnen en de ontwikkeling van genen celtherapie. Helaas worden deze inspanningen gefrustreerd door hindernissen in de regelgeving. In plaats van het enorme potentieel van innoverende middelen in te zien, zijn regulerende autoriteiten, zorgverzekeraars en politici vooral bezorgd over de prijs van dergelijke vernieuwingen.
De huidige restrictieve protocollen, die door verzekeraars worden afgedwongen voor het gebruik van anti-TNF bij chronische darmontstekingen, vormen een voorbeeld van de negatieve invloed van hún kostenbesparing op het potentiële heil dat de individuele patiënt hiervan mag verwachten. Het is gemakkelijk om de besparing uit te rekenen die het gevolg is van het niet beschikbaar stellen van een effectief medicijn. Maar het is veel minder eenvoudig om te bepalen wat de opbrengst is van het ziektevrij houden van een patiënt, en diens bijdrage aan de samenleving. Deze ‘opbrengst’ wordt door zorgverzekeraars genegeerd.

Bijwerkingen
Het afgeven van een licentie voor een nieuw te vergoeden medicijn kan nu soms langer dan tien jaar duren door uitgebreide regelgeving en overheidsbeleid dat de zorgkosten wil verlagen. Natuurlijk moeten nieuwe medicijnen getest worden voordat die op de markt worden gebracht. Maar bij de ontwikkeling van geneesmiddelen gaat het om de balans tussen effectiviteit en bijwerkingen: een cholesterol verlagend middel mag natuurlijk niet veel bijwerkingen hebben, maar voor geneesmiddelen tegen ernstige en dodelijke ziekten zijn soms flinke bijwerkingen acceptabel.
Regelgevers lijken eerder bezig met het vermijden van alle eventuele bijverschijnselen dan met de positieve effecten. Als gevolg hiervan worden nog geen tien werkelijk innovatieve medicijnen per jaar in Europa en Amerika goedgekeurd. Als ontwikkelaars van mobiele telefoons net zo hadden gereageerd op de geruchten dat hun producten hersentumoren veroorzaken, dan was vernieuwing in die branche stilgevallen om te wachten op het ultieme bewijs dat mobieltjes veilig zijn.

Onnodig duur
Wij stellen de huidige toelatingsprocedure ter discussie, waarbij een positieve fase-II studie gevolgd moet worden door dure en langdurige fase-III studies, voordat een licentie afgegeven kan worden. Deze eis vertraagt de ontwikkeling van innovatieve medicijnen ernstig en maakt de ontwikkeling van innovatieve medicijnen het exclusieve terrein van een handvol grote farmaceutische bedrijven. Die kostbare trajecten maken medicijnen onnodig duur en laat beschikbaar voor patiënten. De gemiddelde kosten voor het ontwikkelen van een medicijn liggen nu op 1 miljard dollar.
Wij vinden dat het wikken en wegen van risico’s en voordelen niet uitsluitend in handen van de regelgevers mag liggen. Natuurlijk hebben zij professioneel advies nodig, maar patiënten met ernstige aandoeningen kunnen vaak even goed of zelfs beter geïnformeerd zijn dan de regelgevers die beslissen over de beschikbaarheid van dergelijke medicijnen. Pogingen om het EMA, het Europese regelgevend instituut, te reorganiseren, lopen vast in stroperige bureaucratie. Patiënten, instellingen en bedrijfsleven zijn hierbij onvoldoende betrokken. Patiënten verdienen een radicale herziening van het huidige regelgevende proces. Ze verdienen vergoedingsgaranties voor nieuwe medicijnen.
mytomorrows.com/blog/regels-frustrere...

flosz 13 juni 2015 08:33

0

Makelaar in medicijnen (Dutch)
April 28, 2015

Een nieuw medicijn ontwikkelen, duurt vijftien jaar en kost een miljard euro. De eerste pilletjes zijn peperduur. Maar dat hoeft niet. Het kan goedkoper en sneller. Alleen loopt de farmaceutische industrie hopeloos achter als je dat vergelijkt met moderne technologieën als de iPhone. Ronald Brus zet met myTomorrows een breekijzer in de medicijnkast.

Ronald Brus is arts en voormalig CEO van het Leidse biotechnologiebedrijf Crucell. Bij de verkoop van Crucell aan het Amerikaanse Johnson and Johnson maakte hij zijn miljoenen waarmee hij nu onbereikbare medicijnen bereikbaar maakt in myTomorrows. Inmiddels met steun van – volgens Brus – ’de beste Europese durfinvesteerders’ als Balderton Capital en Sofinnova Partners. ,,Toen mijn vader longkanker kreeg, kwam ik er achter dat veel dingen beter kunnen. Ik ontdekte als arts – en werkzaam midden in de wereld van de farmacie – dat het bijna onmogelijk was om voor hem de nieuwste medicijnen te krijgen. Dat kan niet de bedoeling zijn, dus ging ik kijken hoe het systeem werkt. Ik ontdekte dat de weg van reageerbuis naar patiënt veel te lang duurt.’’

Marktplaats
Met de huidige stand van de technologie kan dat anders. ,,De wetgeving is fair en biedt ruimte voor ’compassionate use’ (het bij wijze van uitzondering voorschrijven van niet-geregistreerde geneesmiddelen) voor schrijnende gevallen. Maar een arts kan niet eenvoudig aankloppen bij een bedrijf met een medicijn dat nog in experimentfase verkeert. Wij creëren met myTomorrows een soort marktplaats van firma’s die willen meewerken. We houden ons volledig aan de wet. De aanvragen van artsen zijn over het algemeen zo goed onderbouwd dat de inspectie ook toestemming geeft.’’

Wereldwijd
Zijn eerste 2,5 miljoen euro in myTomorrows ging op aan het onderzoeken en het begrijpen van alle juridische systemen op farmaceutisch gebied. Wereldwijd. myTomorrows is actief in heel Europa en heeft inmiddels ook voet aan de grond in Amerika waar de ’right to try’-beweging opkomt voor mensen die experimentele medicijnen willen gebruiken. ,,Nu weten we hoe het moet. Het begint met een deal sluiten met een farmabedrijf zodat artsen bij ons in de winkel kunnen kijken of we wat nieuws voor hun gereedschapskist hebben. En ze weten dan zeker dat ze het ook geleverd kunnen krijgen. Ons model is niet veel anders dan marktplaats of makelaar in medicijnen. Wij maken het mogelijk. We zijn geen patiënten aan het overhalen, we maken geen reclame. Dat mag niet eens. Alles gebeurt via de arts en dat moet ook zo blijven.’’

Gereedschapskist
Inmiddels heeft de gereedschapskist van myTomorrows zeven experimentele medicijnen. Het bedrijf richt zich op middelen die worden ingezet bij oncologie, ernstige depressie, aandoeningen aan het centraal zenuwstelsel, alzheimer, MS en ALS. Brus hoopt dat hierdoor het roer om gaat in medicijnland. ,,Er komen politieke en maatschappelijke discussies omdat wij die medicijnen aanbieden. Dat gaat werken. Ik voel me geen Messias als ik dat zeg, maar als er straks
weer een zoon is met een vader met kanker, vind ik dat ze een betere keuze moeten hebben en hoop ik dat de dokter de grootste gereedschapskist heeft die er is.’’

Alles in de wereld gaat sneller, behalve medicijnontwikkeling. Als het aan Brus ligt wordt er een tandje bij gezet. Voor we een medicijn aan mensen mogen geven, moet het goed zijn onderzocht in laboratoria en getest met dierproeven. ,,Dat heeft alles te maken met het medicijn Softenon tegen ochtendmisselijkheid bij zwangere vrouwen. Dat veroorzaakte in de jaren zestig misvormde kinderen. Daarom zijn er nu drie fases met veldproeven. In 1960 duurde hetzelfde proces drie tot zes jaar.’’
mytomorrows.com/blog/makelaar-in-medi...

Bijlage:

Prof. Dollar 13 juni 2015 09:20

0

’right to try’-beweging. Interessante artikelen flosz! Wederom dank voor het delen.

rationeel 14 juni 2015 13:09

0

wim brands...boeken...vpro:

Joris Luyendijk

Te gast is Joris Luyendijk. Hij kreeg van The Guardian de opdracht om te schrijven over The City, het financiële hart van Groot-Brittannië. Het resultaat is het boek 'Dit kan niet waar zijn'.Meer

flosz 16 juni 2015 18:57

0

Health~Holland Knowledge & Innovation Agenda www.health-holland.com/public/downloa...

Bijlage:

flosz 17 juni 2015 10:03

0

List in Europe or the U.S.....
Zie bijlage.
Art.via:
twitter.com/amitkjolly/status/6108794...

Bijlage:

flosz 17 juni 2015 10:04

0

Deel 2:
www.ey.com/Publication/vwLUAssets/EY-...

Bijlage:

flosz 17 juni 2015 10:22

0

Top European Ipo's 2014: ArGen-X, ProQR, $QURE

Bijlage:

flosz 19 juni 2015 14:23

0

Totaal OT: Gisteravond docu.film Keep On Keepin' On gezien (Via Uur vd Wolf,Ned.2).
Leven en vriendschap tussen Jazz-trompettist Clark Terry en Pianist Justin Kauflin, prachtig imho.

AEX 878,83 -3,44 -0,39% 30 apr


Germany40^	17.905,20	-0,15%
BEL 20	3.883,26	-0,09%
Europe50^	4.901,63	-0,40%
US30^	38.043,99	0,00%
Nasd100^	17.570,06	0,00%
US500^	5.063,57	0,00%
Japan225^	38.318,54	0,00%
Gold spot	2.290,37	-1,95%
EUR/USD	1,0667	-0,51%
WTI	81,58	-1,35%

Corbion	+2,70%
B&S Group SA	+1,20%
Ahold Delhaize	+1,17%
ForFarmers	+1,04%
OCI	+1,00%

VIVORYON THER...	-13,94%
EBUSCO HOLDING	-5,50%
ACOMO	-4,69%
Air France-KLM	-4,29%
Arcadis	-3,88%

uniQure « Terug naar discussie overzicht

Draadje OT, bijzaken & geleuter in de marge!

Meedoen aan de discussie?

Direct naar Forum

Markt vandaag

Aandelenadviezen van IEX.nl

Stijgers

Dalers