Imagine a world without disease. Let's join forces to accelerate the development of advanced medicines and ensure access to cures for all patients, everywhere.
At age 41, Clevetta Drew no longer has to worry about severe pain crises and considers a 2019 gene therapy trial a rebirth. Learn about her #SickleCellDisease journey and advice for others: https://go.usa.gov/xFEAe #SCDFaces
Our @UCDavisGT Director @jan_nolta dreams about a time when #genetherapy is accessible to all. #immunotherapy #healthcare #affordable
At @fredhutch, @Jenome77 shows Michael Louella her "gene therapy in a box" technology that will make it possible bring #GeneTherapy research towards an #HIVcure to Uganda and India by 2024 #ADayWithHIV
"But let us be clear: fighting a pandemic with no vaccine and no cure is hard. With 1.5 million new infections and 680,000 deaths last year, AIDS remains an emergency. We must act with urgency if we are to save more lives."
- @Winnie_Byanyima at #UNGA76 #endAIDS
Claudin 18.2 specific CART cells are giving a hopeful positive signal in digestive system tumors- supporting the use of CART in solid tumors #ESMO21 @CARsgen_Ther #immunotherapy #CARTcell https://www.prnewswire.com/news-releases/appealing-data-of-carsgen-therapeutics-car-t-ct041-in-advanced-gastric-cancer-presented-at-esmo-301380054.html
Great article about recent advances toward sickle cell disease cure @nytimes @ginakolata @nih_nhlbi @GatesUS @gateshealth The time is now to develop low cost cures for all! @bluebirdbio @ScottDRamsey https://www.nytimes.com/2021/09/14/health/sickle-cell-cure.html
Wonderful summary from @PosAware of the @iasociety pre-conference on #HIV cure & #SickleCell disease https://www.positivelyaware.com/articles/highlights-2021-hiv-cure-and-gene-therapy-forum @globalgti
In 2020, the @globalgti was formed to tackle the barriers to #LMIC inclusion in #genetherapy development. We have set a goal of introducing two gene therapy Phase I #clinicaltrials in Uganda and India by 2024.
Thrilled to share our 1st collaborative publication as the @globalgti working group:"Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)" published in @GeneTherapy_SN. @cwru @CaseEngineer @fredhutch @caring_cross
The highest prevalence of #HIV/#AIDS, #SickleCell and #thalassemia, and thus the populations who could benefit the most from one-time curative treatments (#genetherapy), are in low- and middle-income countries.
Read more in our @GeneTherapy_SN publication: https://www.nature.com/articles/s41434-021-00284-4
Great article summarizing #sicklecell advances! Gene therapies close in on a cure for sickle-cell disease https://www.nature.com/articles/d41586-021-02138-w
Spain is leading the way here in a distributed POC (point of care) model @CChabannon
Pr Alvaro Urbano-Ispizua (BCN) presenting the Spanish experience and projects with locally manufactured autologous CAR-T Cells to treat r/r ALL, NHL and MM at the 6th edition of @TheEBMT ITC (virtual) @TheEBMT_CTIWP @TheEBMT_Nurses @JACIE_EBMT
Prof Julie Makani presents the @TheLancetHaem series on Hematological care priorities in sub-Saharan Africa. at the 13th conference of African Society of Human Genetics #AfSHG2021 and 18th @H3Africa Consortium meeting @juliemakani @humangeneticstz #AfSHG2021.
New pub highlighting efforts of Gates Fndtn & Novartis to bring curative cell/gene therapy for SCD & HIV @Novartis @gatesfoundation Collaborative science to advance gene therapies in resource-limited parts of the world: Molecular Therapy https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(21)00306-3?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1525001621003063%3Fshowall%3Dtrue
He is the first patient to participate in a clinical trial testing a novel #genetherapy approach for treating #SCD. After needing monthly blood transfusions for 17 years to control his disease, he is now symptom-free.
After a targeted #genetherapy operation at @OhioState to treat his #AADC, six-year-old Jamell no longer has seizures and is able to sit up and grab toys. #raredisease https://on.today.com/3zk28YL
The UK regulatory system is moving rapidly. Here is the link for “Consultation on Point of Care Manufacturing” This framework should accelerate point of care manufacturing of cell and gene therapy products world-wide https://www.gov.uk/government/consultations/point-of-care-consultation/consultation-on-point-of-care-manufacturing
Affordable, robust, local production is a bottleneck for #CARTcell treatments as front line therapies.
Just published in @BCD_AACR and free to read: a review on Scalable Manufacturing of CAR T Cells for Cancer #Immunotherapy: https://bit.ly/3CI0RwM
The UK is paving the way for “point of care manufacturing” to bring the cost of cell and gene therapy down-by proposing a new regulatory approach @MHRAgovuk - We fully support this visionary approach! @ASGCTherapy @sitcancer @CRUK_Policy @AACR