{"id":142462,"date":"2018-12-10T15:30:34","date_gmt":"2018-12-10T20:30:34","guid":{"rendered":"https:\/\/patientworthy.wpengine.com\/2018\/12\/10\/pku-and-living-medicines-spotlight-synlogic\/"},"modified":"2018-12-10T15:30:34","modified_gmt":"2018-12-10T20:30:34","slug":"pku-and-living-medicines-spotlight-synlogic","status":"publish","type":"post","link":"https:\/\/patientworthy.com\/es\/2018\/12\/10\/pku-and-living-medicines-spotlight-synlogic\/","title":{"rendered":"PKU and\u00a0Living Medicines:\u00a0Spotlight\u00a0Synlogic\u00a0\u00a0"},"content":{"rendered":"

Press releases\u00a0earlier this year\u00a0on\u00a0biotech company, Synlogic, announced quite favorable results on both pre-clinical and clinical data on a unique application of synthetic biology for the rare disease,\u00a0phenylketonuria (PKU)<\/a>.<\/h4>\n

In just a short span of time, proof of concept on genetically modified probiotic bacteria (designed to perform\u00a0certain\u00a0enzymatic functions lacking in PKU individuals) progressed from\u00a0murine and primate models to\u00a0dose-related increases in specific\u00a0biomarkers of healthy volunteers\u00a0confirming metabolic activity. And now, with an identified dose for moving forward in PKU patients, investigators will be enrolling subjects across\u00a0several\u00a0clinical centers to better understand the pharmacodynamics of PKU\u00a0in single and multi-dose cohort studies with\u00a0this potential new therapy \u2013 a first in its class.\u00a0\u00a0\u00a0<\/span><\/p>\n

Harnessing the potential of a symbiotic relationship between the PKU prototype SYNB1618 and patients brings a\u00a0potential\u00a0wealth of rewards.<\/h4>\n

In addition to optimizing PKU management with\u00a0the promise of\u00a0new options and fulfilling great, unmet need, there is also another\u00a0\u2018symbiosis\u2019\u00a0to be\u00a0found in the approach to commercializing these therapies right from the start.\u00a0 Rare disease drug developer,\u00a0President,\u00a0CEO and\u00a0CMO, Dr. Aoife\u00a0Brennan explains that at the heart of this process\u00a0and company, which goes beyond\u00a0just\u00a0patient-centric care,\u00a0is\u00a0an\u00a0advisory board\u00a0made up of\u00a0patients and researchers as partners.\u00a0 Input from the primary beneficiaries and stakeholders\u00a0makes sense, especially when it comes to\u00a0identifying meaningful\u00a0outcomes\u00a0and\u00a0fulfilling the\u00a0mission and vision.\u00a0 And, for SYNB1618 that\u00a0vision will\u00a0hopefully translate to normalizing blood phenylalanine and moving a patient\u2019s\u00a0\u201cphe\u201d\u00a0level into the target range according to best treatment practices and guidelines.<\/p>\n

Although\u00a0the patient variability in dietary tolerance of phenylalanine (with patients falling on a\u00a0PKU\u00a0spectrum from mild to severe enzyme deficiency) is a challenge,\u00a0time will reveal the deliverables on\u00a0possibly eating a normal diet.\u00a0And, as a patient,\u00a0the end to dietary manipulation is\u00a0something I\u00a0would\u00a0love to hear\u00a0and experience in\u00a0my lifetime.<\/p>\n

But, what is even more impressive\u00a0to hear\u00a0and learn\u00a0is\u00a0the dedication and commitment\u00a0behind\u00a0reaching this shared\u00a0goal. This was modeled in both action and hard work by Dr. Brennan, who eloquently expressed, \u201cI love my job.\u201d\u00a0And, we love\u00a0and value\u00a0the work you do. The PKU community\u00a0and greater rare disease community\u00a0thanks you.\u00a0<\/span><\/p>\n

\n

Jennifer Payne,\u00a0(diagnosis PKU, 1973), pharmacist,\u00a0and mother to 3 nonPKU children (living\u00a0truth\u00a0to the power of the phe-restricted diet!) is\u00a0a health advocate on a personal and professional level.\u00a0\u00a0She grew up\u00a0as part of\u00a0Maryland\u2019s participation in a multi-state collaborative study on the efficacy of the PKU diet.\u00a0 And, as an early generation treated\u00a0PKU\u00a0adult, she\u00a0continues to\u00a0challenge\u00a0the federal government\u00a0on\u00a0health policy and\u00a0the necessity of medical foods in PKU clinical management\u00a0and\u00a0for other\u00a0FDA approved\u00a0PKU\u00a0pharmaceuticals\u00a0and emerging therapies. Jennifer\u00a0is passionate about\u00a0justice for PKU,\u00a0research\u00a0and rare disease,\u00a0and\u00a0continues to fight for\u00a0access to equitable and affordable treatment\u00a0for all in her work and advocacy efforts.\u00a0\u00a0\u00a0<\/span><\/p>\n<\/div>\n

What are your thoughts about the power of prayer when living with rare disease? Share your stories, thoughts, and hopes with the Patient Worthy community!<\/a><\/h4>\n","protected":false},"excerpt":{"rendered":"

Press releases\u00a0earlier this year\u00a0on\u00a0biotech company, Synlogic, announced quite favorable results on both pre-clinical and clinical data on a unique application of synthetic biology for the rare disease,\u00a0phenylketonuria (PKU). In just a short span of time, proof of concept on genetically modified probiotic bacteria (designed to perform\u00a0certain\u00a0enzymatic functions lacking in PKU individuals) progressed from\u00a0murine and primate […]<\/p>\n","protected":false},"author":1,"featured_media":124374,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":[],"categories":[12894,12007],"tags":[12067,12249,13742,14397],"_links":{"self":[{"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/posts\/142462"}],"collection":[{"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/comments?post=142462"}],"version-history":[{"count":0,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/posts\/142462\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/media\/124374"}],"wp:attachment":[{"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/media?parent=142462"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/categories?post=142462"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/patientworthy.com\/es\/wp-json\/wp\/v2\/tags?post=142462"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}