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In light of the clearances, Solid Biosciences plans to launch a phase 1b clinical trial in the final quarter of this year. #genetherapy #CPVT #CardiologyTwitter Read more: hubs.li/Q03wWsPg0

“I would say that gene therapy for monogenetic disease is validated now, and now I think we need to take gene therapy to the next level..." -Kilian Guse, PhD (Pacira BioSciences, Inc.) #osteoarthritis #genetherapy #ASGCT2025 Watch: hubs.li/Q03wXfwn0

"[T]here's a lot of potential out there, but there's still a lot of questions that remain." -Jeffrey Chamberlain, PhD (University of Washington) #MuscularDystrophy #genetherapy #Duchenne Read more: hubs.li/Q03wWwhw0

ICYMI: On Friday Capricor Therapeutics received a CRL from the FDA regarding its BLA for Deramiocel, an allogeneic cardiosphere-derived #celltherapy that was under review for the treatment of #Duchenne #MuscularDystrophy cardiomyopathy. Read more: hubs.li/Q03x7yw10

.Ultragenyx stated that it plans to work with the agency to address the CMC observations and that it plans to resubmit the BLA thereafter. #genetherapy #MPSIIIA #SanfilippoSyndrome Read more: hubs.li/Q03xczNb0

ICYMI: “I would say that gene therapy for monogenetic disease is validated now, and now I think we need to take gene therapy to the next level..." -Kilian Guse, PhD (Pacira BioSciences, Inc.) #osteoarthritis #genetherapy #ASGCT2025 Watch: hubs.li/Q03xcwLY0

“[If] you have an expert on a particular disease area, who may not be an expert in gene editing, we can pair those people up to develop a new therapy." -Nathan Yozwiak, PhD (Mass General Brigham) #ASGCT2025 #genetherapy #celltherapy Watch now: hubs.li/Q03xk6DR0

ICYMI: "[T]here's a lot of potential out there, but there's still a lot of questions that remain." -Jeffrey Chamberlain, PhD (University of Washington) #MuscularDystrophy #genetherapy #Duchenne Read more: hubs.li/Q03xkg5y0

.Nanoscope Therapeutics noted that the first modules of MCO-010's BLA have been submitted to the agency and that full submission is expected to be finished early next year. #genetherapy #RetinitisPigmentosa #Ophthalmology More: hubs.li/Q03xpmwp0

ICYMI: Ultragenyx stated that it plans to work with the agency to address the CMC observations and that it plans to resubmit the BLA thereafter. #genetherapy #MPSIIIA #SanfilippoSyndrome Read more: hubs.li/Q03xpn850

ICYMI: "[T]here's a lot of potential out there, but there's still a lot of questions that remain." -Jeffrey Chamberlain, PhD (University of Washington) #MuscularDystrophy #genetherapy #Duchenne Read more: hubs.li/Q03xyQPJ0

“At the end of the day, developing any gene therapy is a massive team sport, and to have that scale is a unique advantage." -Nathan Yozwiak, PhD (Mass General Brigham) #ASGCT2025 #genetherapy #celltherapy Read more: hubs.li/Q03xyV2N0

ICYMI: Last week Capricor Therapeutics received a CRL from the FDA regarding its BLA for Deramiocel, an allogeneic cardiosphere-derived #celltherapy that was under review for the treatment of #Duchenne #MuscularDystrophy cardiomyopathy. Read more: hubs.li/Q03xyTPB0

With 2025 having recently hit its midway point, the CGTLive® team has highlighted 5 notable therapies to keep an eye on as the remainder of the year unfolds. #celltherapy #genetherapy #FDA U.S. FDA Click here: hubs.li/Q03xM56f0

ICYMI: Nanoscope Therapeutics noted that the first modules of MCO-010's BLA have been submitted to the agency and that full submission is expected to be finished early next year. #genetherapy #RetinitisPigmentosa #Ophthalmology More: hubs.li/Q03xMRwt0

ICYMI: “[If] you have an expert on a particular disease area, who may not be an expert in gene editing, we can pair those people up to develop a new therapy." -Nathan Yozwiak, PhD (Mass General Brigham) #ASGCT2025 #genetherapy #celltherapy Watch now: hubs.ly/Q03xMRTw0

The FDA has cleared the path for ATSN-201’s pivotal phase 1/2/3 trial in X-linked retinoschisis (#XLRS), This subretinal gene therapy may become the first approved treatment for XLRS. Enrollment begins in 2026 👏 🔗Read more now: hubs.li/Q03xWpdV0

ICYMI: “At the end of the day, developing any gene therapy is a massive team sport, and to have that scale is a unique advantage." -Nathan Yozwiak, PhD (Mass General Brigham) #ASGCT2025 #genetherapy #celltherapy Read more: hubs.li/Q03xWZhk0

"I think really the main takeaway should be that that the HCAd vector platform is suitable for developing gene therapies for prevalent nongenetic disease." -Kilian Guse, PhD (Pacira BioSciences, Inc.) #genetherapy #ASGCT2025 #ASGCT #osteoarthritis Watch now: hubs.li/Q03xX68b0

ICYMI: With 2025 having recently hit its midway point, the CGTLive® team has highlighted 5 notable therapies to keep an eye on as the remainder of the year unfolds. #celltherapy #genetherapy #FDA U.S. FDA Click here: hubs.li/Q03xWZxK0