PharmaVoice’s Post

Exciting news about frexalimab and CD40L inhibition in multiple sclerosis and the effects on MS biomarkers of neuroaxonal damage! More information below 👇 #MS #neurofilaments #biomarkers #frexalimab

From patient screening through cell product infusion and to LTFU, patient journey has numerous risks that can de rail the clinical progress of the cell therapy program. Learn how to build in the risk mitigation strategies in your clinical program

Must read CAR-T paper. I have been messaging this for a while: You DO NOT need the product to persist for durable remission. CD8+ expansion was correlated to response, not persistence. Should apply to allo as well. See Caribou Biosciences and their PD1 edit (extends time of CAR-T function, not for increased persistence).

Phenomenal results of #Lorlatinib therapy for #ALK positive advanced/ metastatic non small cell #lungcancer. Median progression free survival #PFS yet to reached! https://lnkd.in/ga4rFZzM

Wow! A new study shows clinical responses to Ide-cel (anti-BCMA CART) are correlated with CD8+ T cell expansion, even though #persistence was not essential for durable remission. This study challenges the current dogma that “persistence is king” and suggests that engineering attempts to increase persistence may not be as pivotal as once thought. This may also have implications for perfecting #allo persistence which has been the major drawback of allo cell therapy naysayers. MAJOR Question: should we be prioritizing more potent, effective CARs that may NOT persist over CARs that persist but are immunosuppressed or exhaust? 🤔 https://lnkd.in/e5TzKYTa #MultipleMyeloma #CART

PolTREG will present its vast expertise at a Treg cell therapy conference in Boston this week. Chief Executive Officer Prof Piotr Trzonkowski will join an impressive line-up of academic and business experts working at the cutting edge of Treg science. “All eyes are turning to the demonstration of positive clinical data to showcase true efficacy [of Treg therapies] and meet the unmet needs of thousands of autoimmune and inflammation patients,” the summit organisers said in a brochure. PolTREG expects several milestones this year. It will publish the results of a long-term safety study into type-1 diabetes (T1D) patients with lead asset PTG-007. And it will launch three new clinical studies for PTG-007: in presymptomatic T1D, in relapsing-remitting multiple sclerosis (RRMS) and the third in primary progressive multiple sclerosis (PPMS). #biotech #biotechnology #celltherapy #cellandgenetherapy #autoimmunedisease #diabetes #multiplesclerosis

Cell-based therapies have emerged as a promising tool for managing corneal disorders. In our latest publication, we highlighted the pivotal role of the local milieu in influencing the success of these therapeutic approaches. https://lnkd.in/dmSGs43M

The Ivy Center's Pharmacokinetics Lab has developed and validated a method to assess niraparib levels in human glioblastoma tissue, cerebrospinal fluid, and plasma to qualify patients for Phase in an ongoing Phase 0/2 trial in newly diagnosed glioblastoma patients (NCT05076513). The results reported in the recently published article indicate significant brain penetration ability of niraparib in glioblastoma patients. https://loom.ly/UrEnb-g

Gene&Cell Therapy >> First patient who received engineered B cell therapy sees early promise for MPS I: Immusoft gave a glimpse on Monday at how the first patient to ever receive an engineered B cell therapy is doing nine months after being dosed, suggesting that the treatment has been safe so far and is showing early signs that it improved his disease. The treatment is for mucopolysaccharidosis I, or MPS I, a genetic disease in which a key enzyme needed to break down sugars is missing. The adult patient in November received a low dose of Immusoft’s experimental treatment, ISP-001. He stayed on Sanofi’s Aldurazyme, an enzyme replacement therapy (ERT), for eight months and then was taken off ERT. Immusoft has said its ultimate goal is to replace ERT, which requires long weekly infusions, with higher doses of its engineered B cell therapy, which it hopes will act as a protein factory for the body to replace the missing enzyme. The company’s experimental treatment involves taking B cells from a patient and then programming them with non-viral gene delivery to make a key protein. The B cells are then returned to the patient in hopes that they will make that functional protein in the patient’s body. Notably, no preconditioning or immunosuppression was required for the B cell therapy. Sean Ainsworth The first patient, who was treated at the University of Minnesota Medical Center, in the Phase 1 trial saw measures of complex sugars called GAGs in his urine fall in the normal range between three and eight months after treatment, which is not typically achieved on Aldurazyme alone. In an interview last week, Immusoft CEO Sean Ainsworth noted that none of the MPS I patients in Aldurazyme’s clinical trial who received the ERT reached normal urinary GAG levels. The patient also saw a 25% decrease in heparan sulfate as measured in his cerebrospinal fluid at six months. Companies and patient advocacy groups have said the FDA has agreed that heparan sulfate is an acceptable surrogate measure of disease activity for MPS. Beyond biomarker results, the patient also saw a 30% improvement in a 6-minute walk test and increased range of shoulder motion at six months. Joint stiffness is a symptom of MPS I. Ainsworth described the functional improvements as “unexpected,” noting the patient was given “the first and our lowest dose that we’ll be giving. And this was in an adult patient, so we didn’t think that there was much opportunity to reverse some of these manifestations.” There have been no adverse events reported to date by the patient, according to Immusoft. At nine months, one month after the ERT treatment had ended, the patient still saw an improvement on their six-minute walk test, though it was less than at six months. Meanwhile, the shoulder flexion improvement persisted. The company is planning to dose a second… #lucidquest #genetherapy #celltherapy

How to cross the Blood Brain Barrier with functionalized #LNP to treat diseases like Parkinson's? Some scientist have used borneol to increase #permeability of nanoparticles to #brain parenchyma through transcytosis and paracellular pathway. Responsive release of exenatide in foci cells reduced α-synuclein formation, which mitigated Parkinson's symptoms in a mouse model. https://lnkd.in/et8zhuje