BioSpace’s Post

Using cell and gene therapy for a specific condition involves a complex assessment of the treatment's benefit-risk ratio. In this Cell & Gene article, Tome’s Chief Scientific Officer, Jonathan (John) Finn, discusses how real-world examples, evolving science, and long-term risks all influence the decision to use cell and gene therapy. Read more here: https://bit.ly/44QldTe #cellandgenetherapy

ICYMI: AMT-130 gene therapy (uniQure) slowed disease progression in patients with #Huntington disease (HD) treated in the high-dose cohort of a phase 1/2 clinical trial (NCT05243017). https://lnkd.in/et6S9-Ww

Primary mitochondrial diseases (PMDs) represent a highly heterogenous group of neurodegenerative disorders that is particularly difficult to treat utilizing conventional methods. Currently, there is no cure for PMDs, and the current standard of care focuses on symptomatic management and improving quality of life This large unmet need has created exciting opportunities for gene therapy programs to treat the underlying cause of PMDs for multiple reasons 1) acting directly on the origin of the problem, 2) allowing for personalized therapy 3) simply increasing the fraction of wild type mtDNA may be sufficient to mitigate the disease phenotype

We have made great strides at Muscular Dystrophy Association for the families we serve, with FDA-approved gene therapies for #SpinalMuscularAtrophy (#SMA) and #Duchenne #MuscularDystrophy (#DMD), however, challenges in development, manufacturing, and approval processes, particularly for #RareDiseases, must be addressed to make #GeneTherapies more accessible and sustainable. Read more in my article for Pharmacy Times, where I explore the latest advancements and challenges in #GeneTherapy for #neuromuscular diseases: https://lnkd.in/gzKiw_t6

National Hemophilia Foundation released a paper last week on Patient Safety in the Age of Gene Therapy: A Central Focus of Review article. The authors address fundamental considerations for people with hemophilia (PwH) patients, cognizant that each individual has their own personal health goals, lived experiences, and comfort level with open-ended questions of long-term risk vs. therapeutic benefits. Safety, in context of the hemophilia communities’ unique history, is a prominent through line in this paper. The authors stress the importance of high enrollment in the World Federation of Hemophilia / Fédération mondiale de l'hémophilie global gene therapy registry, ideally placed to collect adverse event data and other developments from patients who receive these products. Follow link below to read the full article: #geneediting #genetherapy #genetherapies #blood #bleeding #blooddisorders #bleedingdisorders #NHF #P2C #WFH https://lnkd.in/gNCYaaJW

Gene&Cell Therapy >> FDA widens Sarepta’s Duchenne gene therapy label to older boys in significant expansion of use: The FDA has broadened the label for Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy to patients ages 4 and older. The approval goes beyond the patient population in which the company reported data for its randomized clinical trials. It includes a full approval for Duchenne patients who are not dependent on a wheelchair, and an accelerated approval for those who are wheelchair-dependent. Marketed as Elevidys, the gene therapy was initially given accelerated approval last year for boys ages 4 and 5 who have Duchenne muscular dystrophy. It’s meant to slow progression of the aggressive muscle disease, and the expansion to older boys has long been awaited by families of patients who have few treatment options. It costs $3.2 million in the US and is not approved anywhere else in the world. There have been signs the FDA was going to expand the therapy’s label. Earlier this year, CBER Director Peter Marks made comments suggesting he was open to doing so. And when Elevidys was initially approved, Marks overruled FDA reviewers who had recommended rejecting it. Marks again overruled FDA staff in broadening the label of Sarepta’s gene therapy. Memos that were released Thursday with the agency’s decision showed that two top FDA officials, Lola Fashoyin-Aje and Nicole Verdun, recommended rejecting the gene therapy in favor of an additional confirmatory study but were overruled by CBER chief Peter Marks. It’s been debated whether the evidence has been strong enough to back the expansion. In October, the company reported that the Phase 3 EMBARK study failed to meet its primary endpoint. Intended as a confirmatory study, it showed that boys ages 4 to 7 who got Elevidys didn’t do better on a 17-item motor skill test than those who got placebo. However, the study met key secondary endpoints — measuring boys’ ability to stand and walk — that Sarepta cited when asking the FDA to approve the therapy for boys with Duchenne regardless of their wheelchair status or age. Continued approval for non-ambulatory Duchenne patients may depend on results from a confirmatory trial, which Sarepta said would be the Phase 3 ENVISION study with non-ambulatory and older ambulatory people with Duchenne. In its first six months on the market, Elevidys generated $200 million in revenue, reflecting strong demand for the therapy. The company’s stock $SRPT was up 35% in after-hours trading. It’s also been helped by the failure of a potential competitor: Last week, Pfizer announced that its Duchenne gene therapy did not succeed in a Phase 3 study. Pfizer’s therapy did not meet any of the secondary endpoints that Sarepta pointed to when asking for its label expansion. #lucidquest #genetherapy #celltherapy

Nanoscope Therapeutics Inc. is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations. #pharmaceuticalindustry #biotech #ophthalmology #eyedisease #retinitispigmentosa #genetherapy #blindness #neurodegeneration #clinicaltrials #geneticdiseases #drugdevelopment #regulatoryaffairs

#cellandgenetherapy continues to make a huge impact in the industry! Gene therapies offer a new hope for #sicklecell patients given the potential for increased availability and fewer side effect risks than previous treatments. #innovation #celltherapy #genetherapy #sicklecelldisease https://lnkd.in/etiMVhHy

💥 Exciting PRODYGY trial update! 👁️   We are delighted to announce positive initial safety data from the first cohort treated in our PRODYGY Phase I/II gene therapy trial of SPVN06, SparingVision’s lead gene-agnostic gene therapy for the treatment of retinitis pigmentosa (RP). RP one of the leading inherited causes of blindness, affecting an estimated two million patients worldwide. Safety data collected from the first cohort of the trial treated with a low dose showed that SPVN06 has been well-tolerated with a favorable safety profile. Following the review of the data, the Data Safety Monitoring Board concluded that it is safe to initiate the second cohort at a medium dose. https://lnkd.in/eQmpMzpd #Ophthalmology #GenomicMedicines #GeneTherapy #SPVN06 #Trial

ICYMI: CGTLive took a look for World Hemophilia Day at how the #hemophilia field has adapted to the introduction of gene therapy to the treatment landscape. https://lnkd.in/e3pfrkS2