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Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

Laatste koers (eur) Verschil Volume
0,881   +0,025   (+2,86%) Dagrange 0,843 - 0,882 8.243.635   Gem. (3M) 6,4M

Orchard ORTX

466 Posts
Pagina: «« 1 2 3 4 5 6 ... 24 »» | Laatste | Omlaag ↓
  1. forum rang 5 Jinxter 3 januari 2022 17:40
    [quot]

    Wij richten ons op de behandeling van immunologische, neurometabole en neurodegeneratieve ziekten, waarbij we verwachten dat een eenmalige behandeling volstaat.
    [/quote]

    Met name dit stukje is interessant, omdat Pharming aangegeven heeft dat men zoekt naar definitieve oplossingen. In een maand draad van Pharming is hier ook al een aantal keren over geschreven.

    Over een tijdje weten we meer……

  2. forum rang 8 Wilbar 3 januari 2022 21:20
    quote:

    Wijze man schreef op 3 januari 2022 13:28:

    [...]

    Mooi he.

    Een voordeel van inloggen bij debeurs is dat je niet al die rare strepen en tekentjes achter aliassen ziet staan.
    Die is majoor, die is luitenant die is sergeant, en terwijl ik dit type schiet ik in een stuip van het lachen.
    Dat IEX is toch compleet zijn doel voorbij geschoten.
    Hahahaahaaha.
    Nog 5 AB,s en ik ben sergeant majoor, als ze me dan nog niet serieus nemen............................

    hahahaahahaha
    u bent dan ook niet voor enig leiderschap in de wieg gelegd :)
  3. forum rang 7 LL 23 januari 2022 15:40
    Orchard Therapeutics Announces Publication in The Lancet of Long-term Clinical Outcomes with Libmeldy for the Treatment of Children with Early-onset MLD

    Administration of one-time hematopoietic stem cell (HSC) gene therapy resulted in sustained, clinically meaningful benefits by preserving cognitive function and motor development in most patients

    90% overall survival with up to 7.5 years of follow-up (median 3.2 years) in 29 patients

    BOSTON and LONDON, Jan. 21, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the publication in The Lancet of long-term clinical outcomes evaluating the safety and efficacy of Libmeldy® (atidarsagene autotemcel) for the treatment of early-onset metachromatic leukodystrophy (MLD). Libmeldy is the only approved one-time gene therapy intended to correct the underlying cause of MLD for eligible patients in the European Union, UK, Iceland, Liechtenstein and Norway. Also known as OTL-200, it is an investigational therapy in the U.S.

    “MLD is a cruel and ultimately fatal disease for which there were previously no approved treatment options beyond supportive care,” said Professor Alessandro Aiuti, deputy director of the San Raffaele Telethon Institute for Gene Therapy in Milan and full professor of pediatrics at the Vita-Salute San Raffaele University of Milan and a senior author of The Lancet manuscript. “Libmeldy represents a significant step forward in the treatment of MLD. These data highlight the potential long-term benefits of HSC gene therapy for these children, especially when intervention prior to symptom onset is possible.”

    Twenty-nine pediatric patients with early-onset MLD, enrolled in either a prospective non-randomized clinical study (n=20) or treated under expanded access frameworks (n=9), were administered Libmeldy and compared with an untreated natural history cohort of 31 patients adjusted for age and disease subtype. Most patients treated with Libmeldy developed motor skills within the predicted range of healthy children or maintained the ability to walk. Treatment with Libmeldy was well-tolerated and there was no evidence of abnormal clonal proliferation or replication-competent lentivirus over the follow up period. There were no treatment-related mortality or serious adverse events. Most adverse events were related to conditioning or background disease. Four patients developed transient anti-ARSA antibodies, which did not impact clinical outcomes.

    “Treatment with Libmeldy resulted in sustained, clinically relevant benefits in children with early-onset MLD by preserving motor development and cognitive function in most patients,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “These are compelling results that underscore the potential of our HSC gene therapy approach to end the devastation caused by severe genetic diseases with a single treatment. We are commercializing Libmeldy in Europe and intend to pursue future potential regulatory approvals.”

    Summary of Results Published in The Lancet

    An integrated analysis was performed on data from 29 pediatric patients with a molecular and biochemical diagnosis of MLD and with either pre-symptomatic late-infantile (typically ranging from six to 30 months old at symptom onset) or pre- or early-symptomatic early juvenile (typically between 30 months and less than seven years old at symptom onset) disease and treated with Libmeldy in a prospective non-randomized clinical study or under expanded access frameworks. These included 16 (55%) pre-symptomatic late-infantile patients (one pre-symptomatic at enrollment became symptomatic by the time of treatment) and 13 (45%) early juvenile patients, eight of whom were early-symptomatic at the time of treatment. Patients were treated and monitored at Ospedale San Raffaele, Milan, Italy. Treated patients were compared with a historical cohort of 31 age- and disease subtype-matched MLD patients from a non-interventional natural history study.

    At the time of analyses in 2018, results from all treated patients showed:

    Efficacy Results

    Total gross motor function measure (GMFM) scores were significantly improved in Libmeldy-treated patients compared to the natural history cohort at two years post-treatment (co-primary endpoint) for both late-infantile (66 percentage points [95% Confidence Interval (CI) 48.9–82.3], p<0.0001) and early juvenile patients (42 percentage points [95% CI 12.3–71.8], p=0.036). The difference was even larger at three years and remained statistically significant for both late-infantile and early juvenile patients.
    Most treated patients displayed normal cognitive development, as well as prevention or delay of central and peripheral demyelination and brain atrophy throughout follow-up. Treatment benefits were particularly apparent in patients treated before symptom onset.
    All treated patients had reconstituted ARSA activity in peripheral blood mononuclear cells (PBMCs) within or above normal range from three months post-treatment and onward with levels significantly increased above baseline at two years post-treatment (co-primary endpoint).
    Twenty-six of 29 patients (90%) were alive with median follow-up of 3.2 years (range 0.64 to 7.51 years) in all participants. Of the three deaths which occurred during the follow-up period, two were due to rapid disease progression in early symptomatic early juvenile patients (8- and 15-months post-treatment, respectively) and were considered unrelated to treatment. The third death was due to ischemic stroke following an infectious event 13.6 months post-treatment in a pre-symptomatic early juvenile patient, which was also determined by study investigators as unlikely related to treatment.

    Safety Data

    Treatment with Libmeldy was well-tolerated, with no treatment-related serious adverse events. Most adverse events were associated with busulfan conditioning or background disease. The most frequently reported grade =3 AEs were febrile neutropenia (n=23, 79%), gait disturbance (n=15, 52%), and stomatitis (n=12, 41%).
    Five treatment-related events of anti-ARSA antibodies were reported in four (14%) of patients, which resolved spontaneously or after B-cell depleting therapy, with no obvious impact on clinical outcome or safety profile. Antibody titers in all cases were generally low and no negative effects were observed in the engraftment of gene-corrected cells or in post-treatment ARSA activity.

    meer info:
    ir.orchard-tx.com/news-releases/news-...
  4. forum rang 5 Jinxter 28 januari 2022 16:53
    Orchard volgers:

    Onderstaande blijven volgen, veracht wel weer een hogere koers komende periode.... zie onderstaande link.
    Men zal de notering aan de Nasdaq niet kwijt willen.

    Zodra ik middelen vrij ter beschikking heb koop ik bij.

    pennywatch.nl/leren-beleggen/waarom-d...,wordt%20aan%20eerdergenoemde%20bovenstaande%20voorwaarden.

    Cheers to Orchard/Pharmnig
  5. forum rang 4 The passenger 29 januari 2022 19:51
    quote:

    Jinxter schreef op 28 januari 2022 16:53:

    Orchard volgers:

    Onderstaande blijven volgen, veracht wel weer een hogere koers komende periode.... zie onderstaande link.
    Men zal de notering aan de Nasdaq niet kwijt willen.

    Zodra ik middelen vrij ter beschikking heb koop ik bij.

    pennywatch.nl/leren-beleggen/waarom-d...,wordt%20aan%20eerdergenoemde%20bovenstaande%20voorwaarden.

    Cheers to Orchard/Pharmnig
    Hi Jinxter,

    Ze kunnen nog altijd een reverse stock split doorvoeren, 10:1.

    Heb er nu 3500, op gemiddeld 1,19, en ga volgende week mijn positie uitbreiden naar 6000.

    Daar hou ik het voorlopig bij, alhoewel de potentie van dit aandeel steeds meer boven het wateroppervlak uitstijgt.

    Salut
  6. forum rang 4 The passenger 29 januari 2022 20:16
    quote:

    The passenger schreef op 29 januari 2022 19:51:

    [...]

    Hi Jinxter,

    Ze kunnen nog altijd een reverse stock split doorvoeren, 10:1.

    Heb er nu 3500, op gemiddeld 1,19, en ga volgende week mijn positie uitbreiden naar 6000.

    Daar hou ik het voorlopig bij, alhoewel de potentie van dit aandeel steeds meer boven het wateroppervlak uitstijgt.

    Salut
    Nog even een overzichtelijke presentatie bijgevoegd.

    Zie link:

    ir.orchard-tx.com/static-files/498288...

    Salut

  7. forum rang 5 Jinxter 5 februari 2022 14:31
    In November 2020 is Orchard Therapeutics toegetreden tot de HollandBIO groep.
    In juli 2021 komt er een PB vanuit Pharming:

    Pharming Group en Orchard Therapeutics gaan samenwerken in de ontwikkeling en commercialisering van autologe HSC ex-vivo gentherapie tegen erfelijk angio-oedeem (HAE)

    Een stukje vanuit de tekst over toetreding van Orchard tot de HollandBIO group.

    “Wij maken gebruik van de eigen hemapoetische stamcellen (HSC) van patiënten. De stamcellen worden bij de patiënt afgenomen, genetisch aangepast en daarna weer aan dezelfde patiënt toegediend. De stamcellen zullen zich weer hechten in het beenmerg, om zich vervolgens blijvend te vermenigvuldigen en differentiëren in alle typen bloed en immuuncellen. Deze genetisch aangepaste cellen komen overall in het lichaam en passeren bijvoorbeeld ook de bloed hersenbarrière.

    Meer info in PB van Pharming en op de website van Orchard Therapeutics.

    Cheers to ORTX/PHAR

  8. forum rang 8 zjeeraar 5 februari 2022 15:28
    quote:

    The passenger schreef op 29 januari 2022 19:51:

    [...]

    Hi Jinxter,

    Ze kunnen nog altijd een reverse stock split doorvoeren, 10:1.

    Heb er nu 3500, op gemiddeld 1,19, en ga volgende week mijn positie uitbreiden naar 6000.

    Daar hou ik het voorlopig bij, alhoewel de potentie van dit aandeel steeds meer boven het wateroppervlak uitstijgt.

    Salut
    Mag ik weten via welke weg / waar / hoe jij die aandelen koopt....alvast bedankt.
466 Posts
Pagina: «« 1 2 3 4 5 6 ... 24 »» | Laatste |Omhoog ↑

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