Eurobench.com

rationeel 23 november 2022 19:37

0

Gap dicht:)

rationeel 23 november 2022 19:41

0

De tijd die eens zou komen;)

rationeel 24 november 2022 09:34

0

13,52 de low van deze beweging?

rationeel 24 november 2022 10:34

0

Actionnaires
Nom Actions %
Point72 Asset Management LP 3 819 700................ 8,17%
Nantahala Capital Management LLC 2 901 929........ 6,21%
PFM Health Sciences LP 2 393 311........................ 5,12%
Bristol-Myers Squibb Company 2 388 108................ 5,11%
SSgA Funds Management, Inc. 2 365 144................. 5,06%
Fidelity Management & Research Co. LLC 2 293 015....... 4,91%
OppenheimerFunds, Inc. 1 817 534........................ 3,89%
Avoro Capital Advisor LLC 1 750 000......................... 3,74%
683 Capital Management LLC 1 355 000................ 2,90%
The Vanguard Group, Inc. 1 244 308......................... 2,66%

pim f 24 november 2022 12:00

0

uniQure (NASDAQ:QURE - Get Rating) had its target price hoisted by research analysts at The Goldman Sachs Group from $47.00 to $57.00 in a research note issued on Wednesday, Stock Target Advisor reports. The brokerage currently has a "buy" rating on the biotechnology company's stock. The Goldman Sachs Group's target price would suggest a potential upside of 127.54% from the stock's current price.

QURE has been the topic of a number of other reports. Cantor Fitzgerald restated an "overweight" rating on shares of uniQure in a research note on Thursday, November 3rd. Wells Fargo & Company reduced their price objective on shares of uniQure from $34.00 to $24.00 and set an "equal weight" rating for the company in a research note on Tuesday, August 9th. Chardan Capital lowered their target price on shares of uniQure from $65.00 to $60.00 and set a "buy" rating on the stock in a research note on Tuesday, August 9th. StockNews.com began coverage on uniQure in a research report on Wednesday, October 12th. They issued a "sell" rating on the stock. Finally, TheStreet cut uniQure from a "c-" rating to a "d" rating in a research report on Tuesday, August 9th. One investment analyst has rated the stock with a sell rating, one has given a hold rating and six have issued a buy rating to the company. According to data from MarketBeat.com, the company has an average rating of "Moderate Buy" and a consensus target price of $48.71.

www.marketbeat.com/instant-alerts/nas...

nb 24 november 2022 12:11

0

Er is een goedkeuring voor zijn hemofilie medicijn. Dat moet nog wel wat.meer kunnen triggeren

rationeel 27 november 2022 12:12

0

quote:
rationeel schreef op 24 november 2022 10:34:
Actionnaires
Nom Actions %
Point72 Asset Management LP 3 819 700....................... 8,17%
Nantahala Capital Management LLC 2 901 929............... 6,21%
PFM Health Sciences LP 2 393 311............................... 5,12%
Bristol-Myers Squibb Company 2 388 108....................... 5,11%
SSgA Funds Management, Inc. 2 365 144....................... 5,06%
Fidelity Management & Research Co. LLC 2 293 015....... 4,91%
OppenheimerFunds, Inc. 1 817 534............................... 3,89%
Avoro Capital Advisor LLC 1 750 000................................3,74%
683 Capital Management LLC 1 355 000....................... 2,90%
The Vanguard Group, Inc. 1 244 308............................... 2,66%

47,77%

rationeel 29 november 2022 18:05

0

uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event

November 29, 2022 11:00 AM EST
uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event
~ IND Submission and Clinical Development Expected to Begin in 2023 ~

~ Advancements in Manufacturing and Technology Platform Also Highlighted ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands , Nov. 29, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy (rTLE) and other focal epilepsies. The event featured a presentation from rTLE clinical expert Ellen Bubrick , MD, associate chair of Neurology at Harvard Medical School and director of the Epilepsy Surgery Program at Brigham and Women’s Hospital in Boston, MA on the unmet medical need of patients with rTLE. The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure’s miQURE™ and linQURE™ technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain. The Company also highlighted its progress in developing a commercial-scale AAV manufacturing platform. A replay of the investor event is available here.

“One week after the landmark FDA approval of the uniQure-developed world’s first gene therapy for hemophilia B, we are pleased to share the progress that we have made in developing AMT-260, a first-in-class gene therapy for temporal lobe epilepsy,” stated Ricardo Dolmetsch , Ph.D., president of research & development at uniQure. “Today’s event presented the preclinical data and the innovative technologies that we hope will allow us to develop AMT-260 and other gene therapies that can locally regulate neuronal activity for diseases of the central nervous system. We look forward to continuing to work toward submitting an investigational new drug application for AMT-260 and advancing it into the clinic in 2023.”

AMT-260 in refractory Temporal Lobe Epilepsy (rTLE)

AMT-260 is an AAV gene therapy that uses an AAV9 vector and combines uniQure’s miQURE and linQURE technologies to deliver multiple miRNAs that reduce the expression of the GRIK2 gene. The GRIK2 gene encodes a subunit of the kainate glutamate receptor, which plays a key role in neuronal excitability and in the pathogenesis of rTLE. It is the first example of uniQure’s broader CNS gene therapy platform that uses local delivery of an AAV to reduce the expression of neurotransmitter receptors and control the excitability of neurons. Pathological activation of neurons underlies diseases such as epilepsies, movement disorders and pain syndromes. AMT-260 is being developed initially for rTLE, but the platform is applicable to other focal epilepsies.

In preclinical mouse studies, AMT-260 has shown a dose-dependent increase in the expression of miRNAs in the mouse hippocampus as well as decreased expression of GRIK2 gene. AMT-260 has been shown to inhibit epileptic activity and improve health in a mouse model of TLE?. The Company has initiated an IND-enabling GLP toxicology study in non-human primates and to date, AMT-260 has been well tolerated and shown no relevant safety findings. uniQure is planning to conduct a Phase I/II study of AMT-260 starting in 2023.

?Advances in Manufacturing Platform Technology?

uniQure also provided an update on advances in its industry-leading gene therapy manufacturing platform and on innovations the Company has made to improve quality, decrease costs, and improve speed of manufacturing AAV therapies.

“We continue to capitalize on the knowledge of our platform, which is used to manufacture our recently-approved gene therapy for hemophilia B, while improving it for our new programs, enabling us to save precious time during development,” stated Pierre Caloz , chief operating officer at uniQure. “The recent development of our manufacturing modules allows us to achieve cost reductions typically seen in more mature biotech domains. We are very pleased that our platform may allow us to advance the exciting promise of AAV gene therapy into larger patient populations."

The full virtual research and development event program was webcast live under the Investors section of uniQure’s website at www.uniQure.com. A replay of the webcast will be available at uniQure’s website for 45 days following the event.

About Temporal Lobe Epilepsy
Temporal lobe epilepsy (TLE) is epilepsy that starts in the temporal lobe area of the brain. There are two temporal lobes, one on each side of the head located behind the temples. TLE is the most common localized, also called “focal,” type of epilepsy. About 60% of people with focal epilepsy have TLE.

About 80% of all temporal lobe seizures start in the mesial temporal lobe, with seizures often starting in or near the hippocampus which controls memory and learning. Mesial temporal lobe epilepsy is the most common form of epilepsy. In neocortical or lateral TLE, seizures start in the outer section of the temporal lobe. This type of TLE is very rare and mostly due to a genetic cause or lesions such as a tumor, birth defect, blood vessel abnormality or other abnormalities in the temporal lobe.

Temporal lobe epilepsy affects approximately 1.3 million people in the U.S. alone, of which approximately 800,000 patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory temporal lobe epilepsy (rTLE) experience increased morbidity, excess mortality, and poor quality of life.

About uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

Frontrunner 29 november 2022 22:22

2

seekingalpha.com/news/3911823-qure-st...

flosz 1 december 2022 12:09

1

Steven Pipe: If you get gene therapy treatment and are liberated from needing to remain on prophylaxis, that's an immediate cost savings every month, every year, and hopefully for decades afterwards www.managedhealthcareexecutive.com/vi...

Drie hematologische middelen als sluiskandidaat eerste helft 2023.
Incl. Etranacogene dezaparvovec www.mednet.nl/nieuws/drie-hematologis...

rationeel 12 december 2022 11:45

0

19,14 GAP...Hogere bodem?

14,44 nog een kleintje:(

Staat NU wel op steun.

rationeel 16 december 2022 14:08

0

uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B

December 16, 2022 7:15 AM EST

uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B

***If approved, etranacogene dezaparvovec would be the first licensed gene therapy in Europe for people living with hemophilia B***

LEXINGTON, Mass. and AMSTERDAM , Dec. 16, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received a positive opinion recommending conditional marketing authorization (CMA) for etranacogene dezaparvovec from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.1,2 uniQure conducted the multi-year clinical development program for etranacogene dezaparvovec and will leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization.

“We are very pleased with this positive opinion recommending conditional marketing authorization for etranacogene dezaparvovec,” said Matt Kapusta , chief executive officer of uniQure. “The program’s multi-year clinical journey began in Europe with our first-in-human Phase I/II study, and the favorable CHMP opinion moves etranacogene dezaparvovec one step closer to the European hemophilia B community. We look forward to continuing our partnership with CSL to help bring this potentially life-changing treatment option to people with hemophilia B.”

Etranacogene dezaparvovec was recently approved by the U.S. Food and Drug Administration . CSL licensed the exclusive global rights to etranacogene dezaparvovec from uniQure in May 2021 , and is now solely responsible for the further development, registration, and commercialization of the therapy. uniQure is responsible for the global commercial supply of etranacogene dezaparvovec.

The positive CHMP opinion is based on findings from the*** pivotal HOPE-B trial***, the largest gene therapy trial in hemophilia B to date.3,4 These findings showed that hemophilia B patients treated with*** etranacogene dezaparvovec*** demonstrated stable and durable increases in*** mean Factor IX (FIX) activity levels*** (with a mean FIX activity of 36.9%***) which led to an adjusted annualized ***bleed rate (ABR) reduction of 64%.1 Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.1***

***The HOPE-B study 24 months analysis*** showed a sustained and durable effect of etranacogene dezaparvovec.5,6 In a clinical setting, etranacogene dezaparvovec continued to be generally well-tolerated with*** no serious treatment-related adverse events.5,6***

The positive opinion from the CHMP will now be reviewed by the*** European Commission*** , which has the authority to approve medicines for the European Union member states.

About Hemophilia B

Hemophilia B is a life-threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.

About Etranacogene Dezaparvovec

Etranacogene dezaparvovec is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients. Etranacogene dezaparvovec (also known as CSL222, previously known as AMT-061) uses a specific type of AAV, called AAV5, as its vector. The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua), which generates FIX proteins that are 5x-8x more active than normal.

About the Pivotal HOPE-B Trial

The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult hemophilia B patients classified as having a diagnosis of moderately severe or severe hemophilia B and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5. A total of 54 patients received a single dose of etranacogene dezaparvovec in the pivotal trial, with 53 patients completing at least 18 months of follow-up. The primary endpoint in the pivotal HOPE-B study was 52-week ABR after achievement of stable FIX expression compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state FIX transgene expression.

Results from the pivotal HOPE-B study demonstrated that etranacogene dezaparvovec produced mean FIX activity of 36.9 IU/dL at 18 months post infusion. At 24 months follow-up, FIX activity remained stable at 36.7 IU/DL. After the six-month lead-in period post-infusion, the adjusted annualized bleeding rate (ABR) (1.51) for all bleeds was reduced by 64 percent (p=0.0002) and all FIX-treated bleeds was reduced by 77 percent (3.65 to 0.83; p<0.0001) over months seven to 18. 96 percent of subjects treated with a full dose of etranacogene dezaparvovec discontinued use of routine prophylaxis, with an overall 97 percent reduction in mean unadjusted annualized FIX consumption of 257338.8 IU/yr/participant to 8486.6 IU/yr/participant (from lead-in period to months 13-18).

Further analyses showed that there was no clinically meaningful correlation between patient AAV5 NAb levels at baseline and FIX activity.

No serious adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with etranacogene dezaparvovec by independent molecular tumor characterization and vector integration analysis. No inhibitors to FIX were reported.

About uniQure

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

rationeel 20 december 2022 14:38

0

uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B
If approved, etranacogene dezaparvovec would be the*** first licensed gene therapy*** in Europe for people living with hemophilia B***
LEXINGTON, Mass. and AMSTERDAM , Dec. 16, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received a positive opinion recommending conditional marketing authorization (CMA) for etranacogene dezaparvovec from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.1,2 uniQure conducted the multi-year clinical development program for etranacogene dezaparvovec and will leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization.

“We are very pleased with this positive opinion recommending conditional marketing authorization for etranacogene dezaparvovec,” said Matt Kapusta , chief executive officer of uniQure. “The program’s multi-year clinical journey began in Europe with our first-in-human Phase I/II study, and the favorable CHMP opinion moves etranacogene dezaparvovec one step closer to the European hemophilia B community. We look forward to continuing our partnership with CSL to help bring this potentially life-changing treatment option to people with hemophilia B.”

Etranacogene dezaparvovec was*** recently approved by the U.S. Food and Drug Administration . CSL*** licensed the exclusive global rights to etranacogene dezaparvovec from uniQure in May 2021 , and is now solely responsible for the further development, registration, and commercialization of the therapy. uniQure is responsible for the ***global commercial supply ***of etranacogene dezaparvovec.

The positive CHMP opinion is based on findings from the pivotal HOPE-B trial, the*** largest gene therapy trial in hemophilia B to date.3,4*** These findings showed that hemophilia B patients treated with etranacogene dezaparvovec demonstrated stable and durable increases in mean Factor IX (FIX) activity levels (with a mean FIX activity of 36.9%) which led to an adjusted annualized bleed rate (ABR) reduction of 64%.1 Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.1

The HOPE-B study 24 months analysis showed a sustained and durable effect of etranacogene dezaparvovec.5,6 In a clinical setting, etranacogene dezaparvovec continued to be generally well-tolerated with no serious treatment-related adverse events.5,6

The positive opinion from the CHMP will now be reviewed by the*** European Commission ***, which has the authority to approve medicines for the European Union member states.

About Hemophilia B
***Hemophilia B is a life-threatening rare disease***. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.

About Etranacogene Dezaparvovec
Etranacogene dezaparvovec is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients. Etranacogene dezaparvovec (also known as CSL222, previously known as AMT-061) uses a specific type of AAV, called AAV5, as its vector. The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua), which generates FIX proteins that are 5x-8x more active than normal.

About the Pivotal HOPE-B Trial
The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult hemophilia B patients classified as having a diagnosis of moderately severe or severe hemophilia B and requiring prophylactic FIX replacement therapy were enrolled in a prospective, six-month observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5. A total of 54 patients received a single dose of etranacogene dezaparvovec in the pivotal trial, with 53 patients completing at least 18 months of follow-up. The primary endpoint in the pivotal HOPE-B study was 52-week ABR after achievement of stable FIX expression compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state FIX transgene expression.

Results from the pivotal HOPE-B study demonstrated that etranacogene dezaparvovec produced mean FIX activity of 36.9 IU/dL at 18 months post infusion. At 24 months follow-up, FIX activity remained stable at 36.7 IU/DL. After the six-month lead-in period post-infusion, the adjusted annualized bleeding rate (ABR) (1.51) for all bleeds was reduced by 64 percent (p=0.0002) and all FIX-treated bleeds was reduced by 77 percent (3.65 to 0.83; p<0.0001) over months seven to 18. 96 percent of subjects treated with a full dose of etranacogene dezaparvovec discontinued use of routine prophylaxis, with an overall 97 percent reduction in mean unadjusted annualized FIX consumption of 257338.8 IU/yr/participant to 8486.6 IU/yr/participant (from lead-in period to months 13-18).

Further analyses showed that there was no clinically meaningful correlation between patient AAV5 NAb levels at baseline and FIX activity.

No serious adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with etranacogene dezaparvovec by independent molecular tumor characterization and vector integration analysis. No inhibitors to FIX were reported.

About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

colt 20 december 2022 17:02

0

UniQure Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

LEXINGTON, Mass. and AMSTERDAM, Dec. 20, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Company granted equity awards to employees as a material inducement to commencing their employment. The equity grants were approved by the Company’s board of directors on December 7, 2022, October 31, 2022, and September 27, 2022 (the “Grant Dates”) each in accordance with Nasdaq Listing Rule 5635(c)(4).

In the aggregate on December 7, 2022, 24 employees received 40,000 restricted stock units (“RSUs”) and options to purchase 26,000 ordinary shares of uniQure. Each option has an exercise price of $23.73 per share, the closing price per ordinary share as reported by Nasdaq on December 7, 2022. In the aggregate on October 31, 2022, 1 employee received 2,400 restricted stock units (“RSUs”) and options to purchase 1,500 ordinary shares of uniQure. Each option has an exercise price of $18.62 per share, the closing price per ordinary share as reported by Nasdaq on October 31, 2022. In the aggregate on September 27, 2022, 31 employees received 65,690 restricted stock units (“RSUs”) and options to purchase 55,970 ordinary shares of uniQure. Each option has an exercise price of $18.04 per share, the closing price per ordinary share as reported by Nasdaq on September 27, 2022.

Each option has a ten-year term and will vest over four years, with 25% of the original number of shares vesting on the first anniversary of the respective grant dates and an additional 6.25% of the shares vesting in approximately equal quarterly installments over the twelve successive quarters following the first anniversary. The RSUs will vest over three years, with one-third of the RSUs vesting annually on each successive anniversary of the Grant Date. The vesting of each grant of options and RSUs is subject to the employee’s continued service with the Company through the applicable vesting dates.

About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

rationeel 26 december 2022 14:35

0

Wij hopen dan maar dat de opties heel veel op gaan brengen:)

Prof. Dollar 26 december 2022 16:14

1

Mogelijk helpt het een overnamebod geaccepteerd te krijgen. Als er een overnamebod volgt en het management en personeel heeft geen aandelen of opties dan is er geen persoonlijke win en zullen zij het bod minder snel accepteren. Bovendien zullen ze het dan vast niet voor een appel en een ei van de hand doen.

Prof. Dollar 1 januari 2023 11:22

1

Gelukkig nieuwjaar!

Wat kunnen we dit jaar verwachten? Eerst een terugblik, dan de vooruitblik.

2022
- De eerder herdoseringsstudie is niet gestart
- Bekendmaking niet voortzetten van SCA3 programma
- Bekendmaking Hemofilie B marktgoedkeuringsaanvragen (US en EU)
- Update Huntington safety low dose (1 jaar; 10 patiënten)
- Update Huntington vrijwillige pauze (inmiddels voortgezet)
- Bekendmaking update intellectual property (IP) portfolio (patenten rondom Padua)
- Bekendmaking Hemofilie B goedkeuring US

2023
- Bekendmaking Hemofilie B goedkeuringen (EU, GB en mogelijk andere)
- Einde (21 februari) samenwerking BMY*
- Einde (16 april) regeling met Forbion (zijn dan niet meer aan banden gelegd)
- Bekendmaking eerste commerciële Hemofilie B behandeling en inkomsten
- Bekendmaking Epilepsie IND
- Update Huntington efficacy low dose (1,5 jaar) en safety high dose (1 jaar)
- Start Epilepsie
- Wellicht... R&D Day (mogelijk update Fabry) en bekendmaking nieuwe programma's

* Vorig jaar schreef ik erbij: van de BMY programma's verwacht ik he-le-maal niks. Als BMY wat wil dan doet ze het zelfstandig of ze nemen de tent over. Dit einde betekent ook nieuw perspectief op de pipeline. Mogelijk een nieuwe samenwerking voor bijvoorbeeld de programma's Alzheimer, Parkinson en ALS. Ik verwacht dat ze Huntington, Epilepsie en Fabry in eigen beheer houden, tenzij een beoogde partner een overnamebod doet.

Allen een gezond en fijn jaar toegewenst.

Prof. Dollar
www.twitter.com/Prof_Dollar

Frontrunner 16 januari 2023 19:33

1

www.linkedin.com/posts/dutch-health-h...

Izette 31 januari 2023 16:16

1

finance.yahoo.com/news/uniqure-apic-b...

flosz 8 februari 2023 18:21

1

Gene therapy can transform life for people with hemophilia. www.bostonglobe.com/2023/01/29/busine...

The firm plans to begin treating patients with severe hemophilia B in the United States by March 31. William Mezzanotte, CSL’s head of research and development, said that “maybe a third” of the roughly 6,000 hemophilia B patients will be eligible for Hemgenix. He said “multiple” insurers have pledged to cover the cost of the treatment but declined to name them. twitter.com/floszcrxl/status/16233661...

AEX 921,94 +2,94 +0,32% 31 jan


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