MarketVIEW · Gene therapy intelligence
18Approved products audited
436Registered trials tracked
14Approved indications covered
10Major markets forecast to 2040
Premium gene therapy market intelligence: from the rare/orphan indications to the broader opportunities.
Gene and cell therapy is moving fast. Winning requires better intelligence.
Gene Scope Intelligence delivers actionable insights for biotech and pharma BD, strategy, and competitive intelligence teams — powered by our trusted analytics platform and deep vaccine market expertise.
First area of focus
Franchise 01 / 04
Retinal diseases
From orphan wins in LCA to the Wet AMD and emerging GA opportunities that could reshape retinal commercial dynamics — plus an RP sub-cluster of multiple parallel AAV programs.
Jump to Wet AMD, RP & GA →Deal tape
Featured collection
Five current titles. One commercial narrative.
Our collection is sequenced for the client journey: we start with an audit of the approved landscape, move into the broader R&D pipeline, then deep-dive into currently 3 ophthalmology opportunities that anchor our retinal sub-cluster. We believe in the broader application of gene therapies in common indications.
Approved landscape · hematology-led
01 · Foundation
Approved gene therapies: situation review
Detailed audit of the approved gene therapy landscape — 18 product audits across 11 indications, with commercial performance, competitive dynamics, and total market assessment. Ideal opportunity-scanning entry point.
Pipeline whitespace · rare disease
02 · R&D pipeline overview
Future gene therapies: R&D targets review
Pipeline analysis across eight non-oncology rare / orphan indications — beta thalassemia, DMD, retinitis pigmentosa, ALS, scleroderma, lupus, myasthenia gravis, and hemophilia A. 436 registered trials, 25 countries, with competitive and platform intensity scored by indication.
Wet AMD · forecast to 2040
03 · Broader ophthalmic opportunity
Future gene therapies: Wet AMD (nAMD)
Patient-based forecasts through 2040 across 10 major high-income developed markets. Interactive model and ~165-slide executive deck. Deep coverage of late-stage AAV candidates (4D-150, ABBV-RGX-314, ADVM-022) and the anti-VEGF injection-burden reduction thesis.
Retinitis pigmentosa · specialist title
04 · Rare retinal focus
Future gene therapies: retinitis pigmentosa
Dedicated specialist report on the RP gene therapy opportunity — inherited retinal disease with a heterogeneous genetic landscape and a growing stable of subretinal and intravitreal AAV programs. Perpetuates our ophthalmology sub-cluster.
Coming soon
Geographic atrophy · forthcoming
05 · Forthcoming retinal title
Future gene therapies: geographic atrophy (GA)
Geographic atrophy — the advanced form of dry AMD — represents the largest unmet retinal need by addressable patient pool. With complement inhibitors (Syfovre, Izervay) defining the first wave, a small set of AAV gene therapy entrants stake a single-dose, long-duration value claim against complement-injection burden.
Patient-based forecasts through 2040 across 10 major developed markets, with construct-level coverage of OCU410, JNJ-1887, and other emerging programs.
Forecast preview
Scrub the Wet AMD model, 2025–2040.
Illustrative share erosion: anti-VEGF maintains dominance through the late 2020s; AAV gene therapy penetration ramps from first launches in 2028, reaching the steady-state switch-in share we model by the mid-2030s. Full assumption set and geographic splits in the workbook.
Wet AMD treated-market revenue, 10 major markets (USD B)
2025
Anti-VEGF$12.2B
Gene therapy$0.0B
GT share0%
Illustrative preview only — full scenario set (base, upside, downside), geographic splits, and asset-level penetration curves live in the interactive workbook.
Regulatory history
Every approved gene therapy, mapped from Luxturna onward.
Eighteen approved products across more than fifteen indications in less than a decade. The cadence accelerated sharply with five approvals in 2022 and another five in 2023, and 2025–2026 already has three confirmed launches. Hover any product for the full indication.
Source: Gene Scope Intelligence approved-product audit. Audit excludes cell-based and tissue-based products.
Whitespace map
Where the pipeline is thin and the need is loud.
The eight non-oncology rare / orphan indications covered in the pipeline report, plotted on competitive intensity versus unmet need. Bubble size scales with addressable patient pool. Hover any indication for program count.
How to read this
Upper-left quadrant flags indications with high unmet need and limited gene-therapy competition — the most actionable screening targets for in-licensing.
Bubble size
Scales with addressable patient pool in the ten major markets.
• <25K patients — small
• 25K–150K — medium
• >150K — large
Hover a bubble
Program count, lead asset, and notes appear here.
Methodology
Auditable sources. Transparent assumptions. Quarterly refresh.
The public tracker on this page is regenerated quarterly from a single master spreadsheet. Every number is sourced; every assumption is exposed in the client workbook. Team and enterprise subscribers get a live tracker and the working forecast models, not just the PDF.
436Clinical trial records ingested from CT.gov, EUCTR, jRCT, CTRI
25Countries with registered gene therapy trials
QuarterlyPublic tracker refresh from the master pipeline sheet
LiveWorkbook tracker for team and enterprise clients
Lead analyst · 20+ years
John Savopoulos
“The question BD teams keep asking isn’t how big is the market — it’s which assumptions am I willing to put my name on. MarketVIEW is built to answer that.”
Vaccines & gene therapy SME
Former sell-side and industry
Published forecaster
Access
Three ways clients work with MarketVIEW.
Single-user for analysts, team licences for BD and strategy groups, enterprise for platform-wide access including the underlying trackers and working forecast models. All tiers include email support from the analyst.
Single user
£6,995Per title, single-user licence
- PDF executive deck
- Interactive Excel workbook (view-only)
- One-year refresh cycle
- Named-user licence
Team licence
£8,995Per title, 5–15 seats
- Everything in Single user
- Editable workbook with assumption overrides
- Eligible for Quarterly analyst briefings (POA)
- Transferable seats within the team
Enterprise
Full platformUnlimited seats
- All current and forthcoming MarketVIEW titles
- Live pipeline & deal-tracker dashboards
- Data export (CSV / API on request)
- Bespoke deep-dives on subscription
- Named analyst lead
Get started
Get the TOC + sample exhibits.
An analyst will be in touch shortly with the TOC and sample exhibits. Three short questions — no eight-field form, no sales call unless you ask.
FAQ
What BD and strategy leads ask first.
If your question isn't here, email us — most enquiries get a same-day analyst response during UK business hours.
How are the reports licensed?
One-off subscription with tiered seat licences (single user, team 5–15 seats, enterprise unlimited). Enterprise includes full access to the underlying trackers and working forecast models. Reports are licensed on a non-exclusive, non-transferable basis according to our master terms and conditions of business. Reports can be updated with additional fees judged on a case-by-case basis.
How often are the data updated?
The public pipeline tracker on this page refreshes quarterly from the master spreadsheet — the most recent refresh date is shown on the explorer. Team and enterprise subscribers receive a live workbook tracker that's updated on disclosure. Forecasts are audited quarterly with a documented change-log.
Can we get the underlying data, not just the PDF?
Yes — team and enterprise clients receive Excel and CSV exports plus access to live dashboards. API access available on request at enterprise tier (under development).
Do you do custom work?
Targeted deep-dives for clients who already have purchased a single team licence — indication-specific competitive assessments, diligence support on in-licensing candidates, bespoke forecasts.
Who is the lead analyst?
John Savopoulos, with 25+ years in vaccine, infectious diseases, and now gene therapy market research across industry and sell-side. Full credentials and publication record available on request.
Is there a sample before I commit?
Yes. Submit the form above and an analyst will follow up personally with the TOC and selected sample exhibits. No credit card, no sales call unless you ask.