Orchard Therapeutics @orchard_tx
Dedicated to transforming the lives of patients with inherited diseases through innovative gene therapy. orchard-tx.com London, England Joined January 2017-
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Today we outlined our U.S. launch plans for Lenmeldy™ (atidarsagene autotemcel), the only approved therapy for eligible children with early-onset metachromatic leukodystrophy. Read more: bit.ly/4940qMp
We are thrilled to announce that the @US_FDA has approved Lenmeldy™ (atidarsagene autotemcel), the only therapy for eligible children with early-onset metachromatic leukodystrophy in the U.S. Read more in our press release: bit.ly/4ceTmzr.
On #RareDiseaseDay please join us in helping raise awareness and #ShowYourColours in support of the millions of people living with rare diseases. Learn more at rarediseaseday.org
We look forward to participating in @WORLDSymposia 2024 and hosting a symposia featuring expert speakers sharing their knowledge and experience to establish new standards and advance #newbornscreening through interdisciplinary collaboration. Learn more: bit.ly/3Ume5uB
We are pleased to share that Kyowa Kirin has successfully completed the previously announced acquisition of Orchard Therapeutics. Learn more: bit.ly/3Hydjmq
Earlier this month, we hosted the 4th MLD NBS Alliance Summit in Vienna which brought together experts and advocates with deep experience across the diagnostic journey and care continuum to advance our efforts to ensure widespread newborn screening for this devastating disease.
Today is World Sanfilippo Awareness Day. Join us in spreading awareness about this rare and life-limiting neurometabolic disease. Visit @CureSanfilippoF for more information: bit.ly/3UIcrAy #WorldSanfilippoDay
We look forward to joining @RareDiseases at this week’s #NORDSummit where our Chief Medical Officer Leslie Meltzer, Ph.D., will participate in a panel on opportunities in cell and gene therapy. Learn more: nordsummit.org.
We are excited to announce that Kyowa Kirin and Orchard Therapeutics announced that our companies have entered into a definitive agreement under which Kyowa Kirin will acquire Orchard Therapeutics. Read more: bit.ly/45kK1RU
Today, we announced the acceptance of our Biologics License Application for OTL-200 in MLD under Priority Review. Read more: bit.ly/3rkvMyA
On #PRIDEday, we recognize inclusion and allyship are not confined to one day, week or month. At Orchard, we believe diversity is a key to our strength and vibrancy.
Tomorrow is International Neonatal Screening Day. We stand with the International Society of Neonatal Screening and like-minded organizations dedicated to advancing #NewbornScreening for genetic diseases. Learn more: neonatalscreeningday.org
Our team looks forward to participating in #epns2023! Click here for more information: epns-congress.com
Solidarity and allyship are key themes throughout the month of June and this #Juneteenth we are continuing the dialogue on diversity, equity and inclusion and encouraging all those in our Orchard to support causes dedicated to uplifting the Black community.
Orchard is excited to join the leukodystrophy community in taking strides to support @ELAOfficielle ‘Met tes baskets’ (wear your trainers) at work international challenge! Good luck to all involved! Learn more: bit.ly/3JeX8f4
Throughout #PRIDE month, and every day, we encourage our team to live our core values and always bring their authentic selves to our Orchard.
Earlier this week on #MPSAwarenessDay, we were honored to welcome Willow and her mother, Charlotte, to our global headquarters to hear their story of hope and resilience living with MPS-IH.
Today is #MPSAwarenessDay. Learn about this group of rare, life-limiting, inherited genetic diseases below and visit @MPSSocietyfor more information.
Our Chief Technical Officer Nicoletta Loggia, Ph.D. spoke at the @RE_Pharma #REpharmaUSA on the topic of innovation in cell and gene therapy manufacturing. Learn more: bit.ly/3nA8NNq
In recognition of #RareDiseaseDay, our global headquarters has been illuminated to help spread the word for all those affected by rare diseases as part of #LightUpforRare.
Beacon for Rare Disea.. @RareBeacon
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RARE Revolution Magaz.. @RareRevolutionM
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ASGCT @ASGCTherapy
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Giulia Massaro @DrGiuliaMassaro
504 Followers 523 Following Lecturer and Group Lead working on Gene Therapy Viral Vector Technologies @School_Pharmacy | Manager @UCL GTx Viral Synthesis Facility | @_BSGCT | @aisuk_info
Cambridge Rare Diseas.. @camraredisease
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Amy Gaviglio @agavig
515 Followers 350 Following Founder, Connetics Consulting | Genetic Counselor | Public Health Genetics/Rare Disease Consultant. Lover of challenging discourse. Tweets are mine.
Medics4RareDiseases @M4RareDiseases
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Cell and Gene Therapy.. @CGTCatapult
9K Followers 1K Following Enhancing productivity and growth in the cell and gene therapy industry 🧬🩺🌍
CGTRM @KCLstemcells
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Alliance for Regenera.. @alliancerm
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UCL Business Ltd @UCL_Business
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NORD @RareDiseases
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Dr Puja R. Mehta 🩺.. @DrPujaM
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Dr Jess Chadwick🧬 @Jess_Chadwick
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SavingDylan.com @SavingDylan_com
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Barbie for Congress (.. @BarbieHardnHall
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Helen Walker @helenmwalker44
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Newswatch @newswatchswizz
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EURORDIS-Rare Disease.. @eurordis
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Adam Feuerstein ✡�.. @adamfeuerstein
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Beacon for Rare Disea.. @RareBeacon
12K Followers 11K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
RARE Revolution Magaz.. @RareRevolutionM
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RDI @rarediseasesint
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Science Magazine @ScienceMagazine
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Great Ormond Street H.. @GreatOrmondSt
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ASGCT @ASGCTherapy
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National Human Genome.. @genome_gov
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Cambridge Rare Diseas.. @camraredisease
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Nature Biotechnology @NatureBiotech
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The Journal of Inheri.. @JIMD_Editors
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NEJM @NEJM
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EveryLife Foundation @EveryLifeOrg
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Genetic Alliance UK @GeneticAll_UK
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9K Followers 1K Following Enhancing productivity and growth in the cell and gene therapy industry 🧬🩺🌍
Alliance for Regenera.. @alliancerm
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6K Followers 625 Following The reference portal and knowledge base for information on rare diseases and orphan drugs. Orphanet nomenclature, ORPHA codes, Orphanet RD Ontology @inserm
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24K Followers 10K Following @nytimes reporter covering prescription drugs [email protected] 714-478-4224 for calls, texts, Signal or WhatsApp
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944 Followers 78 Following Brussels correspondent APM, Europe columnist ACT
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240 Followers 541 Following Biotech and Health Care Industry. Rare Diseases.
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3K Followers 2K Following Energy correspondent @Reuters; formerly focused on drugs & moonlights occasionally as footie reporter |[email protected] |ex @Guardian |Devoted to @Arsenal
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5K Followers 2K Following Health Science Editor at Reuters. Wife, Stepmom, Bubbe, Sister, Aunt. Any opinions are mine. https://t.co/95ehuJRHvO https://t.co/lB4R2VaQuP
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2K Followers 450 Following I write and talk about a wide range of things. IPOs, spins, SPACs and memes. @business @SyracuseU Alum. Views are my own [email protected] DM for Signal
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NYT Science @NYTScience
1.2M Followers 455 Following Science, Medicine, Environment, Space & Astronomy. Sign up for the Science Times email, in your inbox every Tuesday: https://t.co/kpdN2C1hDy
We congratulate our colleagues at Orchard Therapeutics for reaching this important milestone. Having a new treatment option for #MLD in the US means so much to #patient families affected by this disease. @orchard_tx
We are thrilled to announce that the @US_FDA has approved Lenmeldy™ (atidarsagene autotemcel), the only therapy for eligible children with early-onset metachromatic leukodystrophy in the U.S. Read more in our press release: bit.ly/4ceTmzr.
Coming up today at #CGMesa22! What are the current trends and challenges surrounding #genetherapy commercialization? Hear from our experts at @SatelliteBio, @CSLBehring, @NovartisNews, @PTCBio, @bluebirdbio, and @orchard_tx.
We are thrilled to welcome Amanda Malakoff to the RDCC as our new Executive Director! Read the press release here: buff.ly/3GKdSZj #OneRareVoice #publicpolicy #leadership #rarediseases
With @EMA_News set to approve between 10-20 ATMPs each year by 2025, how are payers preparing? Join us now for a panel of experts from @orchard_tx, @aok_plus, @HAS_sante & @eurordis as they discuss early dialogue with payers & cross-border access at #CGMED22!
It's almost time for our 1st panel at #CGMED22! To date, 7 ATMPs approved in Europe have been withdrawn from the market. In 15 minutes, speakers from @AntionBioSci, @orchard_tx, @novonordisk, @GenSightBio, & @KitePharma will discuss how to successfully commercialize #ATMPs.
We are thrilled to announce our 2022 Executive Committee! Thank you for your leadership and dedication to advancing greater awareness of the challenges and opportunities of discovering, developing and delivering treatments for those living with #rarediseases. #OneRareVoice
Will 2022 be a banner year for #genetherapy for #raredisease? We're excited to welcome our panelists from @pfizer, BioMarin, @bluebirdbio, @orchard_tx, & @KrystalBiotech to discuss upcoming milestones in our first panel of #CGSOTI22. Watch live: alliancerm.org/arm-event/soti… #JPM22
Cal’s Cupcake Gala is four months from today! Thank you to @orchard_tx and @CarlinosMarket for sponsoring this year’s event. Want to learn more about the sponsorship opportunities available for the 2022 Cupcake Gala? Learn more here: thecalliopejoyfoundation.org/events
Excited, only 1 day away! Join us tomorrow, for our Rare Storytellers Event: Women and Rare Disease. were we synthesize stories, science and songs. @MedicalCollege @mcwgspmc @harmonize4hope @childrenswi @orchard_tx Thursday, December 9, 12-2pm. Register Below
Happening in one week! We’re proud to partner with @childrenswi, @orchard_tx and @mcwgspmc to bring to you our next #RareStorytellers event–Women and Rare Disease at 12 pm on December 9. Register here: mcw-edu.zoom.us/webinar/regist…
Day 3 is here #WODC. Cell & Gene Therapy Track in Room 3 | 9:40 am. "Putting things into perspective: How well are GT’s performing?" @EUCOPE @RIZIV_INAMI @DAKGesundheit @NovartisGene @spark_tx @alliancerm @orchard_tx @AstellasGeneTx #WODC #orphandrugs #rarediseases
For the panel discussion "Evaluating cross border access to treatment and healthcare in Europe" head over to Room 3 | 11:40 AM in the Cell & Gene Therapy track. Hear from @eurordis @EMA_News & APAC, @PTCBio @EUCOPE @orchard_tx @Metab_ERN @Telethonitalia @EU_Commission #WODC
The last day of #WODC! But still a lot more to enjoy like “Putting things into perspective: How well are GT’s performing?” Moderator: @EUCOPE #RIZIVINAMI @DAKGesundheit @Novartis @spark_tx @orchard_tx @AstellasGeneTx #orphandrugs #rarediseases
The closing panel of the Science Strategy Track starts at 12:10 PM in Room 4. Hear "Creating successful business models for ultra-rare diseases" from @JanssenGlobal @GenethonFr @ForbionLifeSci @orchard_tx #WODC #orphandrugs #rarediseases
A fantastic panel on “Creating successful business models for ultra-rare diseases” #WODC #orphandrugs #rarediseases @JanssenUS @GenethonFr @orchard_tx
Our final session of #wodc 2021 #orphandrugs #rarediseases “How to expand and harmonize newborn screening programs across Europe?” @eurordis @orchard_tx #MeyerChildrensHospital @UniFerrara
It is so critical to better understand how health systems can support caregivers of children with #RareDiseases like MLD #KnowMLD #NationalCaregiversMonth
Caregivers for loved ones with devastating rare genetic diseases like MLD often have a substantial burden on their quality of life. #KnowMLD #NationalCaregiversMonth
Day 2 @AdTherapiesConf is well underway!See the panel discussion ‘Broadening the scope of gene therapies, expanding towards less rare conditions’ in the plenary hall #ExCeLLondon @MeiraGTx @uclchildhealth @ucl @GenSightBio @orchard_tx #ascendispharma #adtherapies #adtherapies2021
Coming up in 5 minutes — a panel on The Past, Present, and Future of Gene Delivery! Reps from @CTIClinical, @Ginkgo, @biogen, @orchard_tx, @mammothbiosci, and Arbor Biotechnologies will discuss novel #genetherapy delivery mechanisms and maximizing efficiency at #CGMesa21.
New on our Cell Lines Blog: Insights from @orchard_tx’s hematopoietic stem cell (HSC) gene therapy supply chain development. ms.spr.ly/6017naffB
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