'Our goal in doing this type of research is to try and push back on these higher prices to get them to a level that we consider to be cost-effective so that more patients can get these treatments...' -Amar Kelkar, MD, MPH, FACP

#Tandem24 #celltherapy

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PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

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John F. DiPersio, MD, PhD, director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine discussed novel innovations that lie on the horizon for cell therapy. Watch now:

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.4DMT has aligned on a pivotal phase 3 trial and registration path for developing 4D-710 gene therapy for the treatment of cystic fibrosis (CF). Read more now:

cgtlive.com/view/4dmt-alig…

Check out the latest news in #celltherapy and #genetherapy now at cgtlive.com.

“We've been working hard to understand the biology of CRS, and we've identified several key pathways that when interfered with reduce the amount of CRS.”

#Tandem24 #celltherapy #StemCellTransplant John F. DiPersio Lab ASTCT

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Data from the trial were published in an online paper for the American Association for Cancer Research ( #AACR24 ) Annual Meeting 2024, held on April 5-10, 2024, in San Diego, California.

#Xerostomia AACR

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Data from CAMPSIITE were presented by Paul Harmatz, MD, (UCSF School of Medicine) at the 2024 #WORLDSymposium .

#mucopolysaccharidosis

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Review top news and interview highlights from the week ending April 12, 2024.

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It was found that at 3 months posttreatment, the 8 patients who received a single 70mg dose showed a mean 43% reduction from baseline in convulsive seizure frequency.

#Dravet Syndrome #Dravet #AntisenseOligonucleotide Stoke Therapeutics

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Carlos Moraes, PhD, Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing with mtDNA mutations and its applications. #MDAconference

cgtlive.com/view/moraes-un…

The data are from 5 patients who were treated with P-BCMA-ALLO1 after having progressed on BCMA targeting CAR T-cell therapy, T-cell engagers, or both.

#MultipleMyeloma #mmsm #AACR24 AACR

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'I think long-term follow up is going to be a huge area of interest because you really want to know if all these therapies are equivalent in terms of organ damage, and whether one is better than the other?'

#Tandem24 #SickleCell #Hematology

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ICYMI:

'I think there's exciting new treatments coming down the pipeline for TCR therapy... I really think that with these modifications, there's a very good chance that we will be able to target this efficiently.'

#Tandem24 Dana-Farber

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ICYMI:

• Ginkgo Bioworks Scoops Up CAR-T Assets From Modulus

• Charles River Labs and Axovia Team Up to Tackle #Ciliopathy With Gene Therapy

Have a #celltherapy or #genetherapy update you’d like to share? Tag us with #AroundTheHelix !

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It was found that at 3 months posttreatment, the 8 patients who received a single 70mg dose showed a mean 43% reduction from baseline in convulsive seizure frequency.

#Dravet Syndrome #Dravet #AntisenseOligonucleotide Stoke Therapeutics

Read more:

cgtlive.com/view/stoke-the…

Allogeneic P-BCMA-ALLO1 CAR T-cell therapy yielded some responses in patients with relapsed/refractory multiple myeloma whose disease had progressed after prior BCMA-targeted therapy. #AACR24 Poseida Therapeutics

cgtlive.com/view/allogenei…

#ICYMI :

'Our goal in doing this type of research is to try and push back on these higher prices to get them to a level that we consider to be cost-effective so that more patients can get these treatments...” -Amar Kelkar, MD, MPH, FACP

#Tandem24 #celltherapy

Watch:

cgtlive.com/view/kelkar-as…

'I think there's exciting new treatments coming down the pipeline for TCR therapy... I really think that with these modifications, there's a very good chance that we will be able to target this efficiently.'

#Tandem24 Dana-Farber

Watch:

cgtlive.com/view/chapuis-i…

ICYMI:

New research out of @HarvardMed showed CAR TEAM cells modified tumor stroma and exhibited increased pancreatic ductal adenocarcinoma (PDAC) antitumor activity in multiple preclinical models. #AACR24

cgtlive.com/view/car-team-…