Precision Medicine Online’s Post

This review provides an overview of current knowledge in prenatal gene therapy and potential future research, discussing its scientific, ethical, legal, and sociological implications, and outlines the necessary parameters for future clinical application. #clinicalappilication #fetalmedicine #fetaltherapeutics #genetherapy #prenatalgenetherapy #prenatalgenetictesting

#Commentary now available! Comparative analysis of #nucleicacid delivery systems for #genetherapy: assessing viral and non-viral approaches with emphasis on #extracellularvesicles The potential of #nucleicacidtherapeutics lies in their ability to address genetic disorders by directly targeting and manipulating the underlying genetic material. However, the translation of nucleic acid therapeutic concepts into effective clinical interventions faces challenges. This Commentary #article navigates the complexities associated with nucleic acid therapeutics, with a particular focus on gene therapy, and explores the recent emergence of extracellular vesicles as a potential solution to overcome the hurdles in nucleic acid delivery. Access below:

Nice summary of the recent approvals in the Cell and Gene Therapy space and why they matter from a practical standpoint. Excited to see what 2024 brings to the industry! https://lnkd.in/e3piaV6Y

The first project in this program to advance gene therapies for very rare conditions will focus on CHAT-mutated congenital myasthenic syndrome.

Thanks for choosing to publish in #PharmacooEconomics

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

Intravenous targeting of airway basal cells for cystic fibrosis gene therapy overcomes lung barriers. 🫁 More than 27 clinical trials for cystic fibrosis gene therapy have failed to find a way past the airway epithelial barrier On page 1196 of this issue, Sun et al. report intravenous airway targeting using lung-targeted lipid nanoparticles (LNPs) containing CRISPR gene editing tools in a mouse model of cystic fibrosis. This basolateral route might provide a breach through the airway barriers to treat cystic fibrosis and possibly other lung diseases. And who is CRISPR's ever diligent Uber? LNPs! https://lnkd.in/exQAvHe8 #CRISPR #Pulmonary #LNP #DDS #mRNA #RNA #DNA #cysticfibrosis

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Exclusive: Ultragenyx secures first funds for Alzheimer's gene therapy spinout: Rare disease specialist Ultragenyx reeled in a $14 million seed round for its Alzheimer’s gene therapy spinout Amlogenyx, the company exclusively told Endpoints News. The California biotech’s subsidiary stapled together the raise, led by GordonMD Global Investments, after initially seeking a $50 million Series A earlier this year. Rather than go with the Series A at this time, Ultragenyx opted for a seed round that will get Amlogenyx to an IND filing in “a couple years,” CEO Emil Kakkis said in an interview. Emil Kakkis “The financing environment can be pretty tough for early-stage, non-clinical stage research at this point in time, and we had a few very committed investors who wanted to move it forward,” Kakkis said. “Instead of trying to close a larger round with more people, we decided just to work with a group that was committed and move the ball a little further down the road. By getting it closer to an IND, we would be in a better position to do the Series A.” Amlogenyx aims to create a one-time Alzheimer’s gene therapy based on a protease developed with Alessandra d’Azzo at St. Jude Children’s Research Hospital, Kakkis said. The 14-year-old Ultragenyx’s expansion into a common disease — after snagging multiple approvals for rare disease treatments — derived from insights into a one-in-a-million-person disease known as galactosialidosis. Work in that area drummed up gleanings of how the lysosomal compound known as protective protein/cathepsin A, or PPCA, had an impact on amyloid beta, Kakkis previously told Endpoints. A study found that the enzyme degrades Aβ42, a protein central to the devastating effects of the memory-robbing disease. Kakkis thinks Amlogenyx’s experimental medicine could potentially offer more benefit to patients than the existing anti-amyloid monoclonal antibodies from Eisai-allied Biogen and Eli Lilly. “What we’re showing is we can put a lysosomal enzyme in the lysosome, and that can reduce the plaque outside the cell, which tells us mechanistically that if you could clear the amyloid inside the lysosomal pathway, you would stop dumping plaque outside and would stop, we think, rupturing cells,” Kakkis said. But it will be years before the biotech’s offshoot will study the investigational gene therapy in humans. By that point, there could be additional Alzheimer’s drugs on the market that target amyloid, other nasty proteins like tau or different angles to the tricky disease. Either way, Kakkis sees room for Amlogenyx’s approach. An intracellular attack, he said, might have a more profound effect on neuronal function than current anti-amyloid methods and it could potentially work “in concert” with the existing monoclonal antibodies. “They… #lucidquest #genetherapy #celltherapy

Gene&Cell Therapy >> Pfizer’s Duchenne muscular dystrophy gene therapy fails Phase 3 trial: Pfizer’s Duchenne muscular dystrophy gene therapy failed to meet the primary and key secondary endpoints in a Phase 3 study, pointing to a bleak future for the program. The gene therapy failed to significantly improve motor function at one year in boys with Duchenne, measured using a 17-item scale that evaluates various aspects of mobility called the North Star Ambulatory Assessment. The therapy also did not significantly improve secondary endpoints such as 10-meter run/walk velocity and time-to-rise-from-floor velocity compared to placebo. “Pfizer will continue to closely monitor all participants enrolled in the study and is evaluating appropriate next steps for the program,” the company said Wednesday in a statement. Known as CIFFREO, the study enrolled 99 boys aged 4 through 7 with Duchenne muscular dystrophy, according to the federal clinical trials database. The participants received Pfizer’s fordadistrogene movaparvovec or placebo. There have been two patient deaths in Pfizer’s Duchenne gene therapy clinical studies. In May, Pfizer disclosed that a boy enrolled in a Phase 2 study for the therapy passed away, and it was investigating the patient’s death. As a result, the company paused the administration of the gene therapy to boys who had originally been in the placebo arm of the Phase 3 study. In 2021, Pfizer disclosed a boy in a Phase 1b study of the gene therapy died and later said he “had more advanced disease with underlying cardiac dysfunction.” The FDA granted accelerated approval last June to Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy for boys aged 4 to 5. Within the narrow group of patients eligible for Elevidys, demand has been high. The FDA is expected to decide by June 21 on whether to expand the eligible population based on mixed results from a confirmatory study. Last year, Pfizer took a step back from developing early-stage gene therapies, selling a suite of preclinical programs to AstraZeneca. #lucidquest #genetherapy #celltherapy

Presentation of the first clinical results for GNT0004 gene therapy for Duchenne Muscular Dystrophy: These results obtained with our drug candidate GNT0004 for Duchenne Muscular Dystrophy are very encouraging, particularly in patients treated at the highest dose, both in terms of microdystrophin expression and functional improvement. They enable us to prepare for the pivotal phase of the trial, and demonstrate how this innovative technology can provide solutions to one of the most complex genetic diseases. I would like to pay tribute to the expertise of our researchers and experts who have made these initial results possible.