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    Home > Active Ingredient News > Endocrine System > Express improved microbial therapy obtains clinical proof-of-concept for the treatment of inherited metabolic diseases

    Express improved microbial therapy obtains clinical proof-of-concept for the treatment of inherited metabolic diseases

    • Last Update: 2021-10-11
    • Source: Internet
    • Author: User
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    ▎The content team editor of WuXi AppTec recently, Synlogic announced that its microbial therapies SYNB1618 and SYNB1934 modified by synthetic biology have obtained positive results in a phase 2 clinical trial for the treatment of patients with phenylketonuria (PKU).
    Phenylalanine levels have a clinically significant decrease

    .

    The press release states that this result is a proof-of-concept for the use of synthetic biology to modify microorganisms as "living therapies
    .
    "

    Phenylketonuria is an inherited metabolic disease.
    Because children cannot degrade the phenylalanine present in many foods, the level of phenylalanine in the blood is too high, causing toxicity, and causing serious neurological and neuropsychological problems.

    .

    Because these symptoms are very serious, babies in many countries need to be screened at birth to ensure that they are diagnosed and treated as soon as possible
    .

    Synlogic was co-founded by Dr.
    Timothy Lu, a well-known synthetic biology expert.
    The company's SYNB1618 and SYNB1934 are two bacterial strains modified by synthetic biology and have the ability to degrade phenylalanine

    .

    They are taken with food at three meals a day to reduce phenylalanine levels in patients with phenylketonuria
    .

    The results of the phase 2 clinical trial showed that the fasting plasma phenylalanine level of patients receiving SYNB1618 (dose of 1e12 live bacteria) decreased by 20% at 14 days.
    After stopping the treatment, the phenylalanine level rebounded, showing the therapeutic effect of SYNB1618

    .

    ▲The effect of SYNB1618 in reducing the level of phenylalanine (picture source: Synlogic's official website) SYNB1934 is an improved strain evolved from SYNB1618, which has a stronger ability to degrade phenylalanine
    .

    The results of the phase 1 clinical trial showed that SYNB1934 can dose-dependently increase the level of phenylalanine degradation product trans-cinnamic acid (TCA) in plasma
    .

    Based on the measurement of phenylalanine degradation biomarkers, the activity of SYNB1934 is twice that of SYNB1618
    .

    Image source: Synlogic's official website "This analysis provides a clear proof of concept for the phenylketonuria research and development project
    .

    " said Aoife Brennan, President and CEO of Synlogic.
    "These data indicate that synthetic biological drugs can bring patients
    Significant impact

    .

    This is an important milestone for Synlogic's synthetic biology platform
    .

    We look forward to advancing phenylketonuria drugs to the critical clinical research stage
    .

    "Reference: [1] Synlogic Announces Positive Phase 2 Data Demonstrating Reduction in Plasma Phenylalanine Levels in Patients with Phenylketonuria.
    Retrieved September 24, 2021, from https://investor.
    synlogictx.
    com/news-releases/news-release-details /synlogic-announces-positive-phase-2-data-demonstrating-reduction[2] Phenylketonuria Clinical Program Update 20 September 2021.
    Retrieved September 24, 2021, from https://assets.
    main.
    pro2.
    maf.
    wdc.
    west.
    com/fa0c96811bf64378a5a01a10915afdf3/PKU_Clinical_Program_Update_09_20_2021.
    pdf Disclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress

    .

    This article is for information exchange purposes only.
    The views in the article do not represent WuXi AppTec's position, nor does it represent WuXi AppTec's support or opposition Views

    in the
    article .
    This article is not a treatment plan recommendation

    .

    If you need treatment plan guidance, please go to a regular hospital for treatment
    .

    This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.
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