Rare Disease Feasibility Intelligence

PREDICT
ENROLLMENT
FAILURE
BEFORE IT
COSTS YOU.

Zonra provides enrollment risk modeling for gene therapy trials in rare and ultra-rare disease — stress-testing site strategy before sponsors engage a CRO or activate a single site.

zonra.risk_model — v2.4
LIVE
$runfeasibility_scan--program PGND-401
Loading epidemiology model...done
Patient pool estimate:~340 diagnosed globally
Undiagnosed ratio:~65%
GT-capable sites (qualified):7 / 42 screened
Trial competition:HIGH — 3 active competitors
LTFU infrastructure:PARTIAL — 4 sites flagged
Africa site coverage:0 sites modeled
Patient pool densityLOW — 340 pts
Site qualification rate17% — 7/42
Trial competition indexHIGH — 0.82
LTFU readinessPARTIAL — 52%
Enrollment Risk Index
7.4
Assessment
⚠ HIGH RISK
Africa: 17% of world population
Africa's share of global trials: 1.3%
Average cost of enrollment delay: $1M+/month
Rare diseases affecting children: 72% are genetic
Gene therapy programs in Phase II/III: 400+ globally
Poor feasibility planning causes 85% of enrollment failures
Ultra-rare disease: fewer than 1 in 2,000 patients diagnosed
Africa: 17% of world population
Africa's share of global trials: 1.3%
Average cost of enrollment delay: $1M+/month
Rare diseases affecting children: 72% are genetic
Gene therapy programs in Phase II/III: 400+ globally
Poor feasibility planning causes 85% of enrollment failures
Ultra-rare disease: fewer than 1 in 2,000 patients diagnosed
1.3%
of global trials
conducted in Africa
$1M+
cost per month
of enrollment delay
85%
of failures linked to
poor feasibility planning
400+
gene therapy programs
in active development
01 The Problem

ENROLLMENT
FAILURE IS
PREDICTABLE.

It is rarely predicted. Sponsors spend millions activating sites before anyone has modeled whether the patients can actually be found.

01 / 04
FEASIBILITY HAPPENS TOO LATE

Most sponsors assess enrollment viability after CRO selection and site contracting. By then, reversing course costs $500K+. The problem was baked in from the start.

$2.4B
Lost annually to preventable enrollment delays
02 / 04
ULTRA-RARE POOLS ARE INVISIBLE

In disorders with fewer than 500 diagnosed patients globally, standard site metrics are useless. Genotype stratification, undiagnosed ratios, and geographic clustering are the real variables.

60–80%
Of ultra-rare trial patients are undiagnosed at trial open
03 / 04
GENE THERAPY ADDS INFRASTRUCTURE RISK

Long-term follow-up requirements, vector manufacturing constraints, and specialized administration capabilities filter the eligible site list from hundreds to single digits.

15 YRS
Minimum LTFU for most AAV gene therapy programs
04 / 04
AFRICA IS EXCLUDED BY DEFAULT

The most genetically diverse continent on earth — with the highest undiagnosed rare disease burden — is absent from every feasibility model built on Western site databases.

1.3%
Of global clinical trials conducted in Africa
02 The Approach

STRESS-TEST
BEFORE YOU
SPEND.

Our feasibility report models every variable that predicts enrollment failure — delivered before CRO engagement, before site activation, before the clock starts running.

01
Epidemiology & patient pool modeling

Diagnosed vs. undiagnosed ratio, genotype distribution, geographic clustering, and registry gap analysis. We find where the patients are before you look for sites.

02
Site capability scoring

Gene therapy infrastructure readiness, LTFU program capacity, investigator experience index, and competing trial load — scored across every candidate site globally.

03
Trial competition density

Active and planned trials competing for the same patient pool, with sponsor mapping and projected enrollment timeline pressure.

04
Enrollment Risk Index + recommendations

A scored summary with specific mitigation recommendations by site, region, and patient identification strategy. Actionable, not theoretical.

FEASIBILITY STRESS-TEST REPORT
Zonra Standard Deliverable · 3–4 week turnaround
Epidemiology & patient pool model
Included
Site capability scoring (global)
Included
Trial competition density map
Included
Enrollment Risk Index + recommendations
Included
Africa site & patient mapping
Add-on
LTFU infrastructure readiness module
Add-on
Regulatory pathway brief (FDORA / diversity)
Add-on
03 Why Africa

THE WORLD'S MOST
GENETICALLY DIVERSE
CONTINENT RUNS
1.3% OF GLOBAL TRIALS.

That is not a data gap. That is a structural failure — and it is costing sponsors the most scientifically valuable populations on earth. Africa's unmatched genetic diversity is a research asset that has been systematically ignored.

Zonra's long-term mission is to build the feasibility infrastructure that makes African sites investable for rare and genetic disease programs — from site identification and investigator mapping to registry development and regulatory pathway guidance.

Talk to us about Africa strategy →
17%
of world population
lives in Africa
1.3%
of global clinical trials
run in Africa
#1
most genetically
diverse continent
7K+
rare diseases, majority
with no approved therapy
04 Who We Serve

BUILT FOR
SPONSORS WHO
CAN'T GUESS.

Zonra works with organizations where enrollment failure is existential — not just expensive.

PRE-SERIES B BIOTECHS

Gene therapy and rare disease developers approaching pivotal studies who need to validate enrollment feasibility before investor scrutiny intensifies. Your runway depends on your trial timeline.

ACADEMIC SPINOUTS

University-originated rare disease programs transitioning to clinical development. You have the science. Zonra gives you the enrollment evidence base for IND submissions and grant applications.

AFRICA-ENTERING SPONSORS

Global programs exploring African sites for the first time. Zonra maps patient populations, qualified sites, and regulatory pathways — so your Africa expansion is evidence-based from day one.

READY TO
STRESS-TEST
YOUR TRIAL?

Tell us about your program. We'll scope a feasibility report that tells you exactly where your enrollment risk lives — before it becomes a crisis.

hello@zonra.bio → Connect on LinkedIn
ZONRA.BIO · RARE DISEASE FEASIBILITY INTELLIGENCE