Alia Therapeutics’ Post

Save your spot for this informative webinar on December 4th to learn how regulatory overview and required analytical testing with qPCR and dPCR technologies aid in the development of cell and gene therapies. #thermofisheremp

How can a collaborative partnership between Veristat and a sponsor of a Phase I/II clinical study for a gene therapy help address critical patient, lab, and manufacturing logistics? Read our case study to learn how our full service clinical trial team delivered with tight coordination of the treatment’s dosing administration and data review 👇 https://bit.ly/48Mtrw4 #fullserviceclinicaltrials #raremetabolicdiseases

📝🎓(Bio) Analytics of Cell and Gene Therapies | 28 & 29 May 2024 | Short Online Course | £316 when you register before 2 May (20% saving) This training course will provide you with a deep-dive insight into how cell and gene therapy products are clinically developed and manufactured. Once completed you will be aware of the types of assays needed to assess pharmacokinetics, immunogenicity and potency. You will also examine the regulatory expectations around assay validation. #educolifesciences #cellandgene #CGT #ATMP #bioanalytics #PK #immunogenicity #celltherapy #training

The Phase Ib trial will evaluate the safety and efficacy of ATA-200, which is designed to deliver a functional copy of the SGCG gene.

dPCR offers precise gene expression analysis helping illuminate molecular pathways, track disease progression, and evaluate therapeutic responses. Ready to refine your research? Learn more in our dPCR guide here: https://lnkd.in/g7kzHx8S #dpcr #therapeutic #research #cellandgene #genetherapy

Considering Financial Perspectives With Gene Therapy Breakthroughs: This issue of Pharmacy Practice in Focus: Health Systems provides an overview of the considerations for the implementation of CRISPR and Cas9 cell-based gene therapies in sickle cell disease. #finance #pharmacy #lifesciences

Asimov and RevOpsis Therapeutics enter into strategic licensing agreement to advance the development of cutting-edge gene therapy solutions This collaboration combines Asimov’s state-of-the-art gene-editing platform with Revopsis’ expertise in therapeutic development, opening new doors for the treatment of genetic diseases and offering hope for patients worldwide. Read more here https://lnkd.in/eh3gTvXB #BiotechNews #GeneTherapy #HealthcareInnovation #LifeSciences #Therapeutics #MedicalAdvances #BiotechPartnerships #Asimov #Revopsis

https://lnkd.in/eD6TcFSs Gene therapies are becoming increasingly popular and offer great potential for treating diseases ranging from degenerations to congenital neurological disorders. Many DNA-based gene therapies use viral vectors to transfer the genetic material. The most common viral vector is the adeno-associated virus (AAV). Historically, this is not a new invention, it was already discovered in 1965. With AAV, as with other products, it is always a challenge to define appropriate CQAs. But what is the best way to do this without wasting time and money? This question and how to optimize analytics and manufacturing will be answered in this course by experts from USP, CDMOs and consultants. So feel free to click on the link and learn more on our website.

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> MeiraGTx's stock rises on small Parkinson's gene therapy dataset: MeiraGTx unveiled results from a small dataset for its Parkinson’s disease gene therapy on Tuesday morning, causing its shares $MGTX to jump by as much as 24%. In what the company called a “bridging study” featuring 14 patients, the high dose of MeiraGTx’s AAV-GAD showed a statistically significant improvement on a Parkinson’s disease measurement score known as MDS-UPDRS Part 3 after 26 weeks. Five patients in the high-dose group improved their “off” state scores by an average of 18 points from baseline, good for a p-value of 0.03. MeiraGTx CEO Alexandria Forbes said in a statement that the company expected a five- to 10-point improvement to represent a clinically meaningful change. The sham group (four patients) and low-dose group (five patients) saw no significant changes compared to baseline. The primary endpoint was safety and tolerability, and was met. In another exploratory endpoint, MeiraGTx said a quality-of-life measure called the PDQ-39, which uses a patient questionnaire, achieved statistical significance in both dosing groups when compared to patients’ baseline scores. There was only a “trend to significance” between the high-dose group and the sham group, MeiraGTx said. The company is still discussing its Phase 3 plans with regulators, but expects to launch a pivotal trial in the first half of 2025, according to a corporate slide deck updated Tuesday morning. Gene therapies for Parkinson’s disease have been attempted before, though none have been FDA-approved. MeiraGTx’s program is designed to reset the abnormal brain activity associated with the disease by targeting production of a brain chemical called gamma-aminobutyric acid, or GABA. AAV-GAD attempts to deliver a functional copy of a gene called GAD into the brain that would increase GABA production and alleviate motor symptoms. #lucidquest #genetherapy #celltherapy

The RNA editing system leverages the CRISPR/Cas technology to target and modify RNA molecules. Specifically, Cas13 proteins, such as Cas13b, Cas13c, and Cas13d, possess ribonuclease activity, allowing the CRISPR/Cas13 system to edit single-stranded RNA. This approach enables RNA manipulation, transcript labeling, splicing regulation, and the detection of viral RNA. Additionally, RNA editing offers the potential for reversible genetic modifications, mitigating the risks and ethical concerns associated with permanent gene editing. This method is known for its high specificity and efficiency.