AlveoGene’s Post

bluebird bio Announces First Outcomes-Based Agreement with Medicaid for Sickle Cell Disease Gene Therapy Michigan Medicaid signed an OBA with bluebird bio. This is important because over 50% of Sickle Cell Disease patients are covered by Medicaid.   What isn't entirely clear from this morning's announcement is the level of specificity this agreement entails and whether it stands for one interested patient or is being built ahead of having specific folks in line.   bluebird is in ongoing discussion with 15 Medicaid agencies.   So when will we see the first patient apheresis?   When will we see the first SCD patient treated?   When will bluebird report YE2023Q423 earnings? When will the media's touted "floodgates" open in Sickle Cell Disease? Stay tuned... #bluebird #sicklecelldisease #oba #ATMP #vertex #lyfgenia #casgevy

The first ever gene-therapy approved for intraputaminal administration - how interesting! Read InpharmD's summary below for more information! #StayInformedWithInpharmD

A significant number of primary immunodeficiency diseases (PIDs) were first reported in patients living in the Middle East. Given the high rate of consanguinity in the region, PIDs are commonplace across the region. According to the Center of Arab Genomic Studies about 2.8 million patients are living with a rare disease in the Middle East. One such PID is the Wiskott-Aldrich syndrome - a rare genetic immunodeficiency which makes it difficult for the bone marrow to produce platelets, making a child prone to bleeding. Treatment options include immunoglobulin (antibody) infusions, platelet transfusions, topical creams for eczema, and steroids or similar medications to control autoimmunity. In severe cases a bone marrow transplant or gene therapy can also be considered. On the occasion of rare disease day, CSL Behring is working relentlessly to provide hope to several patients across the world. #MEA #RareDisease #DrivenbyOurPromise #WiskottAldrichSyndrome

This week, the FDA approved the first-ever gene therapy directly administered to the brain. KEBILIDI™ is indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. With only around 350 cases reported in the literature, AADC deficiency is an ultra rare genetic disease characterized by severe disability that begins during the first few months of life, ultimately leading to death. The FDA granted the application Priority Review, Orphan Drug designation, and a rare pediatric disease priority review voucher. Kebilidi was approved under the Accelerated Approval pathway, based on surrogate or intermediate clinical endpoints predicting clinical benefit for serious conditions. Read InpharmD's summary below! #StayInformedWithInpharmD

Community update from Taysha Gene Therapies: High Dose of TSHA-102 was Generally Well Tolerated. TSHA-102 was generally well tolerated with no serious adverse events or dose-limiting toxicities in the first two adolescent/adult patients up to 20 weeks and in the first pediatric patient up to six-weeks. The third high dose adult/adolescent patient was dosed, and the second high dose pediatric patient was enrolled. Continued Enrollment in High Dose Cohorts. The Independent Data Monitoring Committee reviewed available clinical data from the two high dose adult/adolescent participants and the first high dose pediatric participant and approved proceeding with enrollment in both adolescent/adult and pediatric studies at the high dose level. Full press release: https://lnkd.in/guNTWxcf

Today is World Parkinson's Day – a day dedicated to raising public awareness of, and supporting those affected by, Parkinson’s disease (PD). Parkinson's disease is the fastest growing and the second most prevalent neurological condition, affecting an estimated 10 million people worldwide. Despite significant advancements in treatment, there is still no known cure for the disease. Levodopa-induced dyskinesia (LID) is a prevalent and debilitating side effect of chronic levodopa treatment, the current gold-standard for managing PD motor symptoms. Around half of PD patients are affected by LID after 5 years of levodopa treatment and up to 90% after 10 years, with current treatment options providing only partial therapeutic benefit on LID.   We are developing BGT-PD, an investigational AAV2 gene therapy candidate for the treatment of LID in Parkinson’s disease. First preclinical data on our BGT-PD program will be presented at the upcoming American Society of Gene & Cell Therapy 27th Annual Meeting.    Learn more about BGT-PD here: https://lnkd.in/e2tZTHQm #WorldParkinsonsDay #ParkinsonsAwarenessMonth #ASGCT2024 #genetherapy #rarediseases #aav #NeurodegenerativeDisorders #advancedtherapies #Parkinsonsdisease 

ARM commends the U.S. House of Representatives for passing legislation to reauthorize the FDA's Rare Pediatric Disease Priority Review Voucher (RPD PRV) program for five years.  Genetic abnormalities are responsible for 80% of rare diseases, and nearly 70% of these conditions present during childhood. However, approximately 95% of those disorders lack an FDA-approved treatment. For many rare disease patients, the availability of cell and gene therapies offers hope for a cure. RPD PRVs enable biotechnology companies developing treatments for rare pediatric diseases to continue to invest in bringing cell and gene therapies to patients in need.  What’s next? The Senate HELP Committee will mark up the Creating Hope Reauthorization Act (S. 4583) on September 26. ARM continues to urge Congress to reauthorize the RPD PRV Program.  For more information, check out ARM’s one-pager on the impact of this program. https://lnkd.in/eVZCGGuu #cellandgenetherapy

More encouraging interim results in the CNS space this week from Lexeo Therapeutics and their LX2006 AAV gene therapy for Friedreich ataxia (FA) cardiomyopathy. Here are a few of my thoughts: 🌟 Similar to what we saw for Huntington’s disease last week, the FA pipeline is small with very few therapies specifically targeting cardiomyopathy. All positive data should be welcomed for the patients, caregivers, and families of those affected. 🌟 It's great to see regulatory incentives for development of rare disease therapies in action. The asset has been granted Rare Pediatric Disease designation, along with Fast Track designation, and Orphan Drug designation. 🌟 As with all severe progressive diseases, I support the use of natural history data for analyses, especially in the early phases of development. Lexeo's press release is attached below.

Many cell and gene therapies are in clinical development for autoimmune and other diseases beyond cancer. Diseases like multiple sclerosis, rheumatoid arthritis, Type 1 diabetes, Sjogren's syndrome, and systemic lupus erythematosus affect various organs and involve complex immune responses. Despite the excitement for potential new treatments, disease diagnosis remains slow and biomarkers of efficacy for new treatments are greatly needed. By identifying new biomarkers, we can develop more precise and personalized therapies, significantly improving patient outcomes and quality of life. #AutoimmuneDiseases #GeneTherapy #Biomarkers #HealthcareInnovation #ChronicIllness