Ocugen’s Post

At 4DMT, we are advancing enhanced therapeutic vectors with the potential to transform treatment of people with genetic diseases like #CysticFibrosis (CF). 4D-710 is our investigational genetic medicine candidate for the treatment of CF and is currently being evaluated in the AEROW clinical trial. Learn more about 4D-710.

Molecular glues hold significant promise for treating diseases that are currently difficult to manage, including various cancers and genetic disorders. Their unique ability to induce specific protein-protein interactions opens up new therapeutic avenues. A significant challenge for the discovery of new glue compounds is the complicated dynamics created by multi-component complexes of #proteins and #smallmolecules. The formation and stability of these complexes can be influenced by various factors, making the identification of effective molecular glues a challenging task. In the upcoming Cytiva webinar, ZoBio 's expert Pim de Vink will try to demystify glues and prove that working with them doesn’t have to be scary. He will also demonstrate that #fragments can serve as an excellent starting point for discovering #molecularglues. You can still register here: https://lnkd.in/e6jCmh67

For US clinicians. Individually, Inborn Errors of Immunity (IEIs) are considered rare, but over 450 have now been discovered, and collectively, they are reported to impact 1 in 1,200 people in the United States. APDS (activated PI3K delta syndrome) is an underdiagnosed, progressive IEI. Register for one of our non-CME speaker programs to hear more about IEIs, such as APDS. The presentation will cover a real-life case study of a patient with APDS, genetic testing, and the first FDA-approved treatment option for APDS. Click to see more information and times: https://lnkd.in/guMnNku8

Have you tried the International Prognostic Index for CLL-IPI on ONCOassist? - https://bit.ly/CLL-IPI Evaluate the prognosis of chronic lymphocytic leukaemia patients using validated genetic, clinical, and biochemical factors. #CLLIPI #ONCOassist #PrognosisAssessment #BloodCancerAwareness

Rhnull phenotype is a rare blood group characterized by the lack of expression of all Rh antigens (D, C, c, E and e) on the red cells. The phenotype is further classified into the regulator and amorph type based on underlying genetic defect. The clinical significance of its recognition is that such patients suffer from Rhnull syndrome associated with osmotically fragile red cells called stomatocytes with subsequent chronic haemolytic anaemia of varying degree. Another importance is that such subjects readily form alloantibodies on exposure to Rh antigens.

Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are both genetic disorders falling under the category of spinocerebellar ataxias (SCAs), a group of progressive, inherited conditions characterized by damage to the cerebellum and other parts of the nervous system. Antisense oligonucleotide (ASO) RNA modulating therapies are among the approaches being explored for their potential to address the genetic abnormalities associated with these conditions. Discover more: https://vicotx.com/ #PrecisionChemistry #SCA3 #SCA1

PacBio - Unravelling undiagnosed rare disease cases by HiFi long-read genome sequencing HiFi long-read sequencing (LRS) for detecting causative structural variants (SVs), single nucleotide variants (SNVs), insertion-deletions (InDels), and short tandem repeat (STR) expansions in extensively studied RD families without clear molecular diagnoses https://lnkd.in/d4c4zYik

Ever wondered how cracking the genetic code in blood and bone marrow samples could change the game for understanding blood cancers? 🤔🔬 🧬Isolation of nucleic acids from hematological samples is crucial for understanding and identifying cancer-causing genetic alterations. The new Applied Biosystems™ MagMAX™ Sequential DNA/RNA Kit offers an automated workflow solution to sequentially isolate both genomic DNA and total RNA in one protocol, allowing for a more comprehensive downstream analysis. Curious to dive deeper? 🧠 Check out the link: https://e.gv6.co/l3gfH4

👉 GGMC conducted the first Copy Number Variation study within Bangladeshi cohort using Chromosomal Microarray Analysis (CMA) for 212 NDD patients. 👉The study identified 1,581 CNVs that included 395 duplications, 658 deletions, and 528 LOH interpreted and classified according to the ACMG guidelines. 👉Our results show the utility of CMA for precise genetic diagnosis and its integration into the diagnosis, therapy and management of NDD patients. Full Paper link: https://lnkd.in/eTu7JyBJ #GenomicsBD #Clinicalgenomics #Genetictest #Chromosomalmicroarray #PrecisionmedicineBD 

Did you know that melanoma accounts for only about 1% of skin cancers but unfortunately causes a large majority of skin cancer deaths.¹   May is Melanoma Awareness Month 🎗️ and with that, we are highlighting how we can partner with oncology drug developers to discover new therapies to help realize a world where every patient gets the right treatment at the right time.    For our in vivo model spotlight we are featuring pretreated PDX model ME1154: ➡️Cancer Subtype: Malignant melanoma ➡️Matched organoid available ➡️SOC treatment data available (Sorafenib, Vemurafenib, Cisplatin) Log into HuBase™, our PDX database, to view the full data for this model! Learn more ➡️ https://hubs.la/Q02y7hFG0 #OncologyResearch #DrugDevelopment #SkinCancer #Melanoma #MelanomaAwarenessMonth ¹https://lnkd.in/eyKDTWj9.