Beursonline.nl

Jinxter 23 oktober 2022 20:31

1

mldpfdd.org/

Op 21 oktober was er een meting waarbij o.a. FDA rechtstreeks in gesprek konden gaan met patiënten

Hoopvol dat er voor eind 2022 nog nieuws komt vanuit de FDA

In november (18e) staat er nog iets op de agenda.

Cheers

Mr. 3L 24 oktober 2022 12:17

0

Ben 'blij' te lezen dat het jullie ook enigszins heeft verbaasd. Bedankt voor jullie input...

dewarmehand 24 oktober 2022 12:39

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Ik mag dan wel staar hebben maar niet kleurenblind. Groen......
Succes alle positivo's.

The passenger 30 oktober 2022 10:57

1

quote:
Jinxter schreef op 23 oktober 2022 20:31:
mldpfdd.org/

Op 21 oktober was er een meting waarbij o.a. FDA rechtstreeks in gesprek konden gaan met patiënten

Hoopvol dat er voor eind 2022 nog nieuws komt vanuit de FDA

In november (18e) staat er nog iets op de agenda.

Cheers

Even weer tijd voor kleine positieve berichtgeving, zie onderstaand.

seekingalpha.com/article/4550783-phar...

Joseph Pantginis

Got it. And then just lastly, and this is really the part I alluded to where your hands might be tied with regard to how you answer it. So with 105, obviously, there's a bit of growing excitement for potential gene therapy around HAE, and this certainly came out at the recent HAE conference that we hosted. So I was just wondering, right now if you'll give us information as it comes out. Is there anything that we could look to even without timing that as the plan would be to release X type of preclinical data to be able to tease the profile of the asset?

Anurag Relan

So Joe, that's really the goal here, right? So I think, first of all, what got us into this position or why did we enter this partnership with Orchard was that we believe, again, around C1 inhibitor. We believe C1 inhibitor is the root cause of -- we know it's the root cause in these patients and we wanted to be able to provide these patients with C1 inhibitor in the same way that we do with RUCONEST to be able to do that with a gene therapy.

The other modalities that we looked at really didn't present themselves with a reliable way to do that. So the goal now in terms of these preclinical models is to be able to use a vector that now has been refined by the team at Orchard and to be able to show that we can actually increase the C1 inhibitor levels in these disease models. So these are disease models being knocked out mice that have -- that don't have the C1 inhibitor gene in place.

And now can you increase those levels to levels that we think would be meaningful in a clinical situation. And we're doing that testing now. So I'm hopeful to be able to provide an update on that soon. But those are the -- that's the type of information that we can look for is, say, can we show meaningful increases in C1 inhibitor expression levels in these preclinical disease models that could translate into the type of benefit that we would need to see in patients.

Zoals ik al vermoedde, bestaan de preclinical desease models, uit onder narcose gebrachte muisjes, die niet over het C1inh gen beschikken.

De vector waaraan gerefereerd wordt, lijkt aan kwaliteit te verbeteren, en Orchard heeft hiervoor een partnership gesloten met Oxford Biomedica, ook al staat het niet als zodanig op de website van Orchard, hetgeen mij enigszins bevreemdt, e doch lijkt het zijn vruchten af te werpen:

www.oxb.com/news-media/press-release/...

As part of the project, Oxford Biomedica’s LentiStable™ technology platform will be used to develop a producer cell line capable of stably expressing lentiviral vectors.

De koers blijft waarachtig te stabiliseren op 0.45. De kosten voor dit aandeel lijkt 0.017 te zijn, en de custodian lijkt voorlopig de lachende derde te zijn.

Salut

Mr. 3L 1 november 2022 15:12

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Volgens mij worden donderdag de 3e kwartaalcijfers bekend gemaakt..? Kunnen we nog geks verwachten of kabbelen we rustig richting eind 2022, waarin we nog wat meer nieuws mogen gaan verwachten..?

Groet,

Mr. 3L

Mr. 3L 3 november 2022 14:46

1

Press Releases

Orchard Therapeutics Announces First Neurocognitive Data from OTL-201 Study in MPS-IIIA Accepted for Oral Presentation at ASH 2022

Company to host KOL conference call and webcast on Monday, December 12 at 5:00 p.m. EST
BOSTON and LONDON, Nov. 03, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced early clinical findings from its ongoing proof-of-concept study of investigational OTL-201 in MPS-IIIA will be featured in an oral presentation at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 10-13, 2022, in New Orleans.

The oral presentation at ASH 2022 will showcase the first neurocognitive data for all patients and extend upon previously presented biochemical results, with a median follow-up of 1.5 years.

The presentation details are listed below. Abstracts are available on the ASH website.

Title: “Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA”
Date/Time: Monday, December 12, 2022, at 10:45 a.m. CST
Presenter: Professor Rob Wynn, consultant pediatric hematologist and director of Pediatric Bone Marrow Transplant Program, Royal Manchester Children’s Hospital (RMCH), part of Manchester University NHS Foundation Trust
Room: Ernest N. Morial Convention Center, 220-222
Orchard is planning to host a virtual KOL investor webcast on Monday, December 12, 2022, at 5:00 p.m. EST to review the data presented at the meeting. A live webcast will be available under “Events” in the Investors & Media section of the company’s website at www.orchard-tx.com and a replay of the webcast will be archived following the event.

Five patients with MPS-IIIA, aged 6 to 24 months, were treated with OTL-201 as part of this ongoing proof-of-concept trial, sponsored by The University of Manchester, conducted at Royal Manchester Children’s Hospital, and funded by Orchard Therapeutics. Primary study objectives include safety, tolerability and peripheral expression of SGSH activity in total leukocytes at 12 months. Secondary study objectives include overall survival and neurocognition as measured by the Bayley Scales of Infant and Toddler Development. Patients will be followed for a minimum of three years.

About MPS-IIIA
Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene which, when healthy, helps the body break down sugar molecules called mucopolysaccharides. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.

About OTL-201
OTL-201 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s hematopoietic stem cells. OTL-201 has received rare pediatric disease designation from the FDA and is currently being evaluated in an ongoing proof-of-concept clinical trial.

About Orchard Therapeutics
At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn.

About Manchester University NHS Foundation Trust (MFT)
Manchester University NHS Foundation Trust (MFT) is the largest NHS trust in England and a leading provider of specialist healthcare services. Its 10 hospitals are home to hundreds of world class clinicians and academic staff, committed to finding patients the best care and treatments. Its hospitals are Manchester Royal Infirmary, Saint Mary's Hospital, Royal Manchester Children's Hospital, Manchester Royal Eye Hospital, North Manchester General Hospital, University Dental Hospital of Manchester, Trafford General Hospital, Altrincham Hospital, Wythenshawe Hospital and Withington Hospital. More information is available at: www.mft.nhs.uk.

About The University of Manchester
The University of Manchester is a member of the prestigious Russell Group and one of the UK’s largest single-site universities. We have over 40,000 students, 12,000 staff and, with almost 480,000 former students from more than 190 countries, are home to the largest alumni community of any campus-based university in the UK. We are ranked in the top ten of the Times Higher Education (THE) Impact Rankings globally; are the top UK University for graduate employability according to The Graduate Market in 2022 and no fewer than 25 Nobel laureates have either worked or studied here. Visit www.manchester.ac.uk for further information or www.manchester.ac.uk/discover/vision/ for our latest strategic vision.

Mr. 3L 3 november 2022 14:46

1

(vervolg)

Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-looking Statements
This press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts Investors Renee Leck Senior Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

Bron: ir.orchard-tx.com/news-releases/news-...

Jinxter 3 november 2022 16:14

1

@ Mr.3L, Passenger, Iris,

Mooi vooruitzicht richting de kerstdagen en het nieuwe jaar (2023).

Cheers to a great future in gen-technologie!

The passenger 8 november 2022 06:52

1

quote:
Jinxter schreef op 3 november 2022 16:14:
@ Mr.3L, Passenger, Iris,

Mooi vooruitzicht richting de kerstdagen en het nieuwe jaar (2023).

Cheers to a great future in gen-technologie!

Nu maar eerst even kennis nemen van de Q3-cijfers. Ik vind ze wel laat vergeleken met voorgaande kwartalen, altijd 1e week van de nieuwe maand volgend op het kwartaal. Ik verwacht overigens dat het aantal behandelingen tegen MLD niet significant is gestegen. Verder hoop ik te horen of de reimbursement een keertje er doorheen is met Spanje, Benelux en Frankrijk. Pffff, dat blijft ook maar voortduren, terwijl de kinderen wel verder blijvend kunnen worden beschadigd door deze ziekte. Desalniettemin…

ir.orchard-tx.com/news-releases/news-...

14 Nov cijfers, 18 Nov 2e call, over MLD in de USA.

Salut

[verwijderd] 11 november 2022 18:37

0

Orchard Therapeutics Q3 2022 Inkomstenvoorbeeld

11 november 2022 12:17 ET Orchard Therapeutics plc (ORTX) Door: Manshi Mamtora , CFA

Orchard Therapeutics ( NASDAQ: ORTX ) zal naar verwachting op vrijdag 11 november, na sluiting van de markt, de resultaten over het derde kwartaal bekendmaken.

De consensus EPS-schatting is -$ 0,22 en de consensus-inkomstenschatting is $ 4,41 miljoen (+270,6% J/J).

In de afgelopen 3 maanden zijn de WPA-ramingen 1 naar boven bijgesteld en 2 naar beneden bijgesteld.

Inkomstenramingen hebben 3 opwaartse herzieningen en 0 neerwaartse bijstellingen gezien.

Jinxter 13 november 2022 21:36

2

Morgen de 14e voorbeurs nieuws vanuit Orchard Therapeutics.

Zou de 14 morgen iets speciaals hebben?

In de USA las ik een artikel op een forum wat positief was over een onderzoek.

ash.confex.com/ash/2022/webprogram/Pa...
Cheers to gen technologie

Drieklezoor 14 november 2022 09:18

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Knap hoor om deze gegevens op te sporen , dankjewel

[verwijderd] 14 november 2022 13:31

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Orchard Therapeutics GAAP EPS of -$0.37 misses by $0.13, revenue of $5.78M beats by $1.37M

Nov. 14, 2022 7:04 AM ETOrchard Therapeutics plc (ORTX)By: Meghavi Singh, SA News Editor
Orchard Therapeutics press release (NASDAQ:ORTX): Q3 GAAP EPS of -$0.37 misses by $0.13.
Revenue of $5.78M (+385.7% Y/Y) beats by $1.37M.

Cash, cash equivalents and investments as of September 30, 2022, were $146.6M, with $33.0M of debt outstanding, compared to $220.1M and the same debt figure as of December 31, 2021.

The company expects that its existing cash, cash equivalents and investments will now fund its anticipated operating, debt service and capital expenditure requirements into the second quarter of 2024.

[verwijderd] 14 november 2022 13:32

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Orchard Therapeutics GAAP WPA van -$ 0,37 mist $ 0,13, omzet van $ 5,78 miljoen verslaat $ 1,37 miljoen
14 november 2022 07:04 ET Orchard Therapeutics plc (ORTX) Door: Meghavi Singh , SA Nieuwsredacteur
Persbericht Orchard Therapeutics ( NASDAQ: ORTX ): Q3 GAAP WPA van -$0,37 mist $0,13 .

De omzet van $ 5,78 miljoen (+385,7% op jaarbasis) overtreft $ 1,37 miljoen .

Geldmiddelen, kasequivalenten en investeringen per 30 september 2022 bedroegen $ 146,6 miljoen, met een uitstaande schuld van $ 33,0 miljoen, vergeleken met $ 220,1 miljoen en hetzelfde schuldcijfer op 31 december 2021.

Het bedrijf verwacht dat de bestaande geldmiddelen, kasequivalenten en investeringen nu in het tweede kwartaal van 2024 de verwachte operationele, schuldendienst en kapitaaluitgaven zullen financieren.

[verwijderd] 14 november 2022 13:49

0

www.marketscreener.com/quote/stock/OR...

Jinxter 14 november 2022 13:56

0

nl.investing.com/news/orchard-winst-l...

Investing.com - Orchard rapporteerde op maandag voor het derde kwartaal een winst die lager is dan verwacht en een omzet die hoger is dan de voorspellingen.

Het bedrijf meldt een winst per aandeel van $-0,370 met een omzet van $5,78M. Analisten geraadpleegd door Investing.com voorzagen een winst per aandeel van $-0,230 met een omzet van $4,41M.

Dit jaar zijn Orchard aandelen vooralsnog 3.5% hoger uitgekomen, waardoor ze beter presteren dan de Nasdaq die 27% lager is uitgekomen in het lopende jaar.

Orchard in reeks belangrijke aandelen in de gezondheidszorgsector deze maand
Op 18 oktober rapporteerde J&J voor het derde kwartaal een winst per aandeel van $2,55 bij een omzet van $23,79B, in vergelijking met een voorspelde winst van $2,48 en een omzet van $23,36B.

Eli Lilly rapporteert op 1 november voor het derde kwartaal een winst die hoger is dan verwacht, namelijk een winst per aandeel van $1,98 bij een omzet van $6,94B. Investing.com analisten verwachtten een winst van $1,91 en een omzet van $6,91B

[verwijderd] 14 november 2022 14:22

2

seekingalpha.com/article/4557429-orch...

voda 15 november 2022 10:33

0

Met dank aan poster Holmes:

quote:
holmes schreef op 15 november 2022 09:17:
Uit:

Orchard Therapeutics Reports Third Quarter 2022 Financial Results and Reviews Recent Business Highlights

Research programs:
Report preclinical POC data for the OTL-104 program in NOD2 Crohn’s disease in early 2023 in advance of IND-enabling studies and an IND submission in 2024.
Continue to advance the OTL-105 program in hereditary angioedema (HAE), OTL-204 program for GRN-FTD and work in HSC-generated antigen-specific Tregs.

Collaboration revenue was $0.4 million for the three months ended September 30, 2022, resulting from the collaboration of OTL-105 in HAE with Pharming Group N.V. entered into in July 2021. This revenue represents reimbursements for research costs and preclinical studies incurred by the company and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which is being amortized into revenue over the expected duration of the agreement.

Net loss was $47.6 million for the three months ended September 30, 2022, compared to $36.4 million in the same period in 2021. The company had approximately 128.1 million ordinary shares outstanding as of September 30, 2022.

ir.orchard-tx.com/news-releases/news-...

Elzenbeurs 30 november 2022 15:56

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Het is stil hier, allemaal fingers crossed?
We hebben gisteren toch nog wat bijgekocht, klein beetje maar hoor. :-)
Wie niet wa.....

Jinxter 1 december 2022 14:13

3

News:

ir.orchard-tx.com/news-events/press-r...

Cheers today

Vertraagd	7 feb 2025 17:36
Koers	0,831
Verschil	-0,005 (-0,60%)
Hoog	0,843
Laag	0,826
Volume	3.502.719
Volume gemiddeld	5.488.752
Volume gisteren	2.518.302

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