Corporate Sponsor Spotlight...

PTC Therapeutics

We extend our deepest gratitude to PTC Therapeutics for their generous support of INDConf LONDON 2025.

PTC is a global biopharma leader delivering transformative therapies for rare diseases, with over 25 years of dedication to science-driven solutions.

Notably, PTC's eladocagene exuparvovec (marketed as Kebilidi in the U.S. and Upstaza in the EU) is the first FDA- and EMA-approved gene therapy for AADC deficiency—an ultra-rare, life-limiting neurotransmitter disorder.

This pioneering one-time therapy delivers the missing AADC gene directly into the putamen, enabling children to produce dopamine and achieve vital motor milestones previously thought impossible.

PTC’s commitment to innovation and patient impact aligns perfectly with INDConf’s mission: bridging science and clinical care to transform the lives of those affected by rare neurological disorders.

Vitaflo International

We extend a warm thank you to Vitaflo, a Nestlé Health Science company, for their invaluable support of INDConf LONDON 2025.

Vitaflo has been a global leader in specialized medical nutrition for over 25 years, developing clinically backed medical foods for inherited metabolic disorders, pediatric kidney disease, and ketogenic diets

Their patient-centered approach includes innovations like single-dose amino acids and ready-to-use feeds—designed to make treatment convenient, effective, and integrated into daily life.

By ensuring consistent quality, accessibility, and education through resources like the VIA portal, Vitaflo empowers patients and healthcare providers across more than 70 countries.

Their clinical evidence-based solutions align perfectly with INDConf’s mission to bridge science and patient care.

Blackfin Bio

A heartfelt thank you to Blackfin Bio for their vital support of INDConf LONDON 2025.

Blackfin Bio is a clinical-stage UK-based gene therapy company focused on developing innovative treatments for rare neurological disorders .

Their lead program, BFB‑101, just received FDA clearance to start a Phase I/II clinical trial for Hereditary Spastic Paraplegia Type 47 – a breakthrough in treating progressive pediatric neurodegeneration.

With a growing pipeline—including advanced AP‑4 deficiency therapies—Blackfin Bio is pioneering scalable gene therapies that align deeply with INDConf’s mission to accelerate translation from bench to bedside.



Their work embodies innovation for rare CNS disorders, and we’re proud to have Blackfin Bio partnering with us on this journey toward real-world impact.

UCL - Neurometabolic Unit

A huge thank you to the Neurometabolic Unit at UCLH/National Hospital for Neurology and Neurosurgery (Queen Square) for their invaluable support of INDConf LONDON 2025.

Since 1995, this UKAS-accredited laboratory has provided clinically essential testing and translational research in mitochondrial, neurotransmitter, pterin, and amino acid disorders.

Their recent advances include:

• A multiplexed LC‑MS method for CSF neurotransmitter and 5-methyltetrahydrofolate analysis, validated by Dr Simon Pope and presented at BIMDG 2024.

• Innovative uHPLC‑Qda mass spectrometry for plasma amino acid profiling—advancing precision diagnostics even while seeking UKAS accreditation.

• Important partnerships, such as the 2025 collaboration with Guilford Street Labs, bringing AI-driven, next-gen diagnostics to neurotransmitter and mitochondrial disorders.

Based in the heart of Queen Square, the Unit exemplifies bench-to-bedside excellence, integrating top-tier science into patient care—a mission perfectly aligned with INDConf’s goals.

Chan Zukerberg Initiative

We’re honoured to acknowledge the Chan Zuckerberg Initiative (CZI) for their generous grant to Hrabriša – Lil’ Brave One through the “Rare as One” Network — marking one of only five patient-led initiatives outside North America to receive this global support.

CZI empowers parent-led rare disease groups to actively participate in scientific research and community-led projects. Thanks to their backing, Hrabriša is amplifying access to early diagnosis, care, and education in non-English-speaking communities affected by congenital neurotransmitter disorders.

This partnership fuels our shared mission to translate patient voices into scientific impact, advancing awareness, research, and global health equity.

We’re proud to be driving this movement alongside CZI and Hrabriša.

Guildford Street Labortory (GSL)

Guildford Street Laboratories (GSL), a University College London spin-out (founded in 2023), is transforming rare disease diagnostics with machine learning-enhanced mass spectrometry.

• Speed + Precision: Specialised protein, lipid, and metabolite assays deliver highly sensitive and accurate biomarker data for early disease detection and therapeutic monitoring.

• End-to-End Expertise: From academic research origins to a clinical-stage lab, GSL collaborates with hospitals, industry partners, and EU consortiums to bring advanced diagnostics to the frontline.

• Innovation Milestones: Their next-gen AI-enabled assays, like PD Predict and HCM Predict, have earned FDA Breakthrough Designation, marking a significant advance in blood-based diagnostics for Parkinson’s and cardiomyopathies.

Why it matters to INDConf delegates:

GSL represents the cutting edge of bench-to-bedside translation, empowering clinicians and researchers with tools that support early detection, patient stratification, and personalised intervention.