SynaptixBio Ltd.

We’ve come a long way in just three years; here are some highlights of the journey so far:   • Founded in April 2021 to find therapies for TUBB4A leukodystrophies. Raised £2m in initial investment. • September 2022 - Signed a worldwide exclusive license to intellectual property from the Children’s Hospital of Philadelphia (CHOP), the world’s leading centre for leukodystrophy research, enabling commercialisation of a treatment. • February 2023 - Gained a Rare Paediatric Disease designation from the US FDA for a drug to treat H-ABC, the most severe form of TUBB4A leukodystrophy. • March 2023 – Achieved Orphan Drug Designation (ODD) from the FDA for H-ABC therapy. • April 2023 - Raised further £11M to drive clinical research and development through to the start of clinical trials. • February 2024 – Achieved second ODD for a treatment for Isolated Hypomyelination, a less well-known form of the disease. • March 2024 – Awarded prestigious BioMedical Catalyst Grant from Innovate UK • April 2024 – Appointed Dr Uwe Meya as Chief Medical Officer to define clinical strategy for the first in-human trials.   Read more about our journey at: https://lnkd.in/dHpRFKPS   #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech

We have come so far, but we have never been more focussed on developing therapies to transform the quality of life for families all over the world who are living with the devastation caused by TUBB4A leukodystrophy.   It is the thought of changing so many lives that drives us, and meeting regularly with the team at the H-ABC Foundation is a constant reminder of how important our work is.   Read more about our work at: https://lnkd.in/dHpRFKPS #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech

We are delighted to welcome Dr Uwe Meya as Chief Medical Officer (CMO).   In a career spanning almost 40 years, Dr Meya has worked for major international biotech firms in clinical research and development for neurological and psychiatric indications.   He is a qualified neurologist and psychiatrist with five and a half years of clinical tenure in academia and has written or co-written over 30 peer-reviewed scientific and medical papers, which have been published in Lancet Neurology, Journal of Clinical Psychiatry, Movement Disorders and elsewhere.   We look forward to Dr Meya’s vital contributions to our mission to develop a therapy for TUBB4A-related leukodystrophies.   https://lnkd.in/etTbD4sG   #Leukodystrophies #EarlyDetection #Healthcare #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech #CommunitySupport #Hope

It is very exciting to be a part of a growing rare disease treatment market, which Global Market Insights Inc. predicts will more than double to $442.7bn in the next 8 years.   New technologies and business models can only support this growth.   Our virtual biotech model has enabled us to speed up the research and development process. This agility we hope will allow us to reach our goal and change the lives of families living with a devastating and life limiting disease. Read more about our work at: https://lnkd.in/dHpRFKPS #Leukodystrophies #EarlyDetection #Healthcare #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech #CommunitySupport #Hope

In the latest 'Beyond Biotech' podcast from Labiotech.eu, Dr Dan Williams discusses developing treatments for rare diseases and SynaptixBio Ltd.'s progress towards finding a therapy for H-ABC, a rare, deadly and currently incurable disease. The full interview is here https://lnkd.in/dDb63EJQ.

Diagnosis of leukodystrophies is based primarily on the analysis of brain imaging (MRI), which reveals myelin abnormalities and classifies leukodystrophies as either hypomyelinating or demyelinating.   As we move forward we will work closely with the broader medical community to support awareness and diagnosis of leukodystrophies.   Read more about our work at: https://lnkd.in/dHpRFKPS   #Leukodystrophies #EarlyDetection #Healthcare #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech #CommunitySupport #Hope

Many thanks to Betsy Goodfellow at Pharmafile.com for featuring an article by Dr Dan Williams. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. The full story is here: https://lnkd.in/e_58twVs

Every breakthrough brings us closer to a treatment! At SynaptixBio, we're committed to collaboration and innovation in the fight against leukodystrophies. Together, let's transform lives and bring hope to families worldwide. Read more about our work at: https://lnkd.in/dHpRFKPS #biomed #rarediseases #health #medicine #biology #education #treatment #TUBB4aleukodystrophy #leukodystrophy #biotech

Leukodystrophies don't discriminate – they affect individuals of all ages, backgrounds, and walks of life. Let's challenge stigma and misconceptions surrounding these conditions and promote inclusivity, acceptance, and support for everyone in the leukodystrophy community. Read about Aggie, 13, and her story with leukodystrophy at : https://lnkd.in/eGf5HgCD

A very interesting read in Bio Science Today from our CEO Dan Williams, discussing the importance of partnerships between small biotechs and Big Pharma in order to unlock faster drug development for rare diseases. Check out the full article at: https://lnkd.in/eneFNBvG