Unravel Biosciences Begins Proof-of-Concept Trials in Colombia: RVL-001 for Rett Syndrome
Boston-based Unravel Biosciences brings the first-ever Rett syndrome and Pitt Hopkins syndrome clinical trials to Latin America — with INVIMA rare disease fast-track approval and first dosing in Medellin, Colombia.
April 10, 2026
12
min read
By
bioaccess® Team
**Key Takeaways**
- • Unravel Biosciences (Boston) is conducting proof-of-concept trials for RVL-001 in Rett syndrome and Pitt Hopkins syndrome
- • INVIMA approval December 2025 under rare disease fast-track pathway
- • First dosing January 2026 in Medellin, Colombia at PECET (Universidad de Antioquia)
- • First clinical trials for Rett and Pitt Hopkins syndromes ever conducted in Latin America
- • 20 patients (15 RTT + 5 PTHS) in placebo-controlled n-of-1 design
Trial overview: RVL-001 for Rett syndrome
Unravel Biosciences, a Boston-based AI-enabled therapeutics company, is conducting proof-of-concept clinical trials in Colombia for RVL-001, a repurposed formulation of vorinostat, for the treatment of Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS). These represent the first clinical trials for either condition ever conducted in Latin America.
RVL-001 was identified through Unravel's proprietary BioNAV AI discovery platform, which uses computational biology to identify existing drugs that can be repurposed for rare genetic disorders. Vorinostat is an FDA-approved HDAC inhibitor currently used in oncology — Unravel's innovation lies in recognizing its potential therapeutic benefit in neurodevelopmental conditions.
RVL-001: repurposed vorinostat for neurodevelopmental disorders
Rett syndrome is a rare genetic neurological disorder that primarily affects girls, causing severe cognitive and physical disabilities. Pitt Hopkins syndrome is an even rarer condition with similar characteristics. Both conditions currently have no FDA-approved disease-modifying treatments.
- RVL-001 is a repurposed formulation of vorinostat (FDA-approved HDAC inhibitor)
- Identified via Unravel's BioNAV AI drug discovery platform
- Targets both Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS)
- No disease-modifying treatments currently available for either condition
INVIMA rare disease fast-track approval
INVIMA, Colombia's national regulatory authority, granted approval for the Unravel trial in December 2025 under its rare disease fast-track pathway. This accelerated regulatory mechanism is designed to expedite clinical trials for conditions affecting small patient populations — exactly the kind of innovative regulatory framework that makes Latin America attractive for rare disease drug development.
Clinical site: PECET at Universidad de Antioquia, Medellin
The trial is being conducted at PECET (Programa de Estudio y Control de Enfermedades Tropicales) at the Universidad de Antioquia in Medellin, Colombia. PECET is one of Latin America's most respected clinical research organizations, with extensive experience in complex clinical trials.
First dosing occurred in January 2026, marking a historic milestone — the first time patients with Rett syndrome or Pitt Hopkins syndrome have received an experimental treatment in a controlled clinical trial setting in Latin America.
Placebo-controlled n-of-1 trial design
The trial employs an innovative placebo-controlled n-of-1 design, enrolling 15 RTT patients and 5 PTHS patients. In this design, each patient alternates between treatment and placebo periods, serving as their own control — an approach ideally suited for ultra-rare diseases where large-scale randomized trials are impractical.
| Parameter | Details |
|---|---|
| Sponsor | Unravel Biosciences (Boston, MA) |
| Drug | RVL-001 (repurposed vorinostat) |
| Indications | Rett syndrome (RTT) + Pitt Hopkins syndrome (PTHS) |
| Design | Placebo-controlled n-of-1 |
| Enrollment | 15 RTT + 5 PTHS patients |
| INVIMA approval | December 2025 (rare disease fast-track) |
| First dosing | January 2026 |
| Site | PECET, Universidad de Antioquia, Medellin |
Why Colombia for rare disease clinical trials
Colombia — and INVIMA's regulatory framework — has emerged as a strategic destination for rare disease clinical trials in Latin America:
- INVIMA rare disease fast-track pathway for expedited trial approvals
- World-class clinical research infrastructure (PECET, leading universities)
- Patient populations with limited prior access to experimental therapies
- Cost advantages: 40–60% lower trial costs than US or European studies
- Ethical framework supportive of innovative trial designs (n-of-1)
- Geographic accessibility from the US (direct flights to Medellin from major cities)
bioaccess® and rare disease clinical trials in Latin America
The Unravel Biosciences trial demonstrates Colombia's growing importance as a clinical trial destination — not just for medical devices, but for innovative therapeutics targeting rare diseases. bioaccess® supports sponsors with first-in-human trials, proof-of-concept studies, and market access across Latin America.
**Planning a clinical trial in Colombia?**
bioaccess® has deep expertise in INVIMA regulatory submissions and clinical operations in Colombia. Contact bioaccess® today to discuss your development program, or explore our FIH FastTrack process.
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