Next Generation Gene Therapeutics
- Company typePrivate
- Founded2020
- HeadquartersSuzhou, China
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
Next Generation Gene Therapeutics firmographics
Firmographics- Name
- Next Generation Gene Therapeutics
- Legal name
- NGGT Inc.
- Website
- https://nggtbio.com
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Ownership category
- akta.pro rank
Next Generation Gene Therapeutics industry classification
Industry- Product category
- Gene Therapy Pharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)
- SIC
- Services-Commercial Physical & Biological Research (8731)
- akta.pro primary industry
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)
- akta.pro secondary industries
- Gene Therapy Analytical Testing & QC (potency, vg/ddPCR, infectivity, RCL/RCA) (HLAAACAK), Gene Therapy Regulatory, Pharmacovigilance & Clinical Operations Services (HLAAACAM)
Keywords
Where Next Generation Gene Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Suzhou
- HQ country
- China
- HQ region
- Asia
Offices2 records
Markets served
Next Generation Gene Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales
Revenue model
- Gene Therapy Product Revenue: Pre-commercial clinical-stage biotech. Revenue will come from commercializing gene therapy products upon regulatory approval. Currently in Phase I/II clinical trials for NGGT001 (Bietti's Crystalline Dystrophy), NGGT002 (Phenylketonuria), and NGGT006 (Familial Hypercholesterolemia).
- Licensing and Partnership Revenue: Potential for licensing agreements and partnerships with pharmaceutical companies for co-development or commercialization rights to pipeline candidates.
Go-to-market motion2 records
Distribution channels1 record
Marketing channels4 records
Next Generation Gene Therapeutics product offering
Product offeringCore offering
Next Generation Gene Therapeutics (NGGT) is a clinical-stage biotechnology company that discovers, develops, and manufactures investigational recombinant adeno-associated virus (rAAV) gene therapies for rare genetic diseases, using its proprietary dual-functional vector platform. Its current pipeline consists of three investigational gene therapy drug products — NGGT001 for Bietti's Crystalline Dystrophy, NGGT002 for Phenylketonuria, and NGGT006 for Familial Hypercholesterolemia — produced at its 90,000-square-foot cGMP facility in Suzhou, China. NGGT is pre-commercial and monetizes through future product sales, licensing, and pharmaceutical partnerships.
Product overview
Next Generation Gene Therapeutics (NGGT) is a clinical-stage biotechnology company pioneering dual-functional vector gene therapies for genetic rare diseases. The company operates its proprietary 'One target, two bullets' technology platform, which uses dual-functional vectors to simultaneously silence harmful mutated genes and express correct protein versions. NGGT's product portfolio includes three clinical-stage gene therapy candidates: NGGT001 for Bietti's Crystalline Dystrophy (Phase I/II), NGGT002 for Phenylketonuria (Phase I/II), and NGGT006 for Familial Hypercholesterolemia (IND-enabling). The company maintains a 90,000-square-foot cGMP manufacturing facility for rAAV production.
Differentiator
Problem solved
Functional benefit
Products and services
- NGGT001 Investigational AAV2-based gene therapy for Bietti's Crystalline Dystrophy (BCD), a rare autosomal recessive ocular disease caused by CYP4V2 gene mutations, currently in Phase I/II clinical trial. Designed for patients with BCD for whom no approved treatments exist.
- NGGT002 Investigational gene therapy for Phenylketonuria (PKU), a rare genetic metabolic disorder caused by deficiency in phenylalanine hydroxylase, currently in Phase I/II clinical trial with demonstrated sustained plasma phenylalanine reduction. Aims to offer a single-treatment alternative to lifelong dietary management.
- NGGT006
Quantifiable outcome
- Visual acuity improved by 12.8 ± 3.7 ETDRS letters at 9 months in BCD patients (P = .0026)
- +3 more outcomes
Companies that use Next Generation Gene Therapeutics
Customer profileNamed customers3 records
Segments3 records
Ideal customer profiles2 records
Next Generation Gene Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Next Generation Gene Therapeutics partnerships and signals
Strategic signalScale indicators6 records
Recent moves6 records
Expansion highlights5 records
Next Generation Gene Therapeutics competitors and assessment
Company assessmentEmerging players
- Genprex: Clinical-stage gene therapy company developing treatments for cancer and diabetes using novel delivery systems. Comparable as a small clinical-stage biotech in gene therapy, though focused on different indications.
Broad incumbents
- BioMarin Pharmaceutical: Established rare disease biotech with a commercialized AAV gene therapy (Roctavian for hemophilia A) and active programs in PKU and other inherited metabolic disorders. Comparable as a larger player in the same gene therapy + rare disease space.
Direct peers
- Adverum Biotechnologies: Clinical-stage gene therapy company developing AAV-based treatments for ocular and rare diseases. Comparable on AAV platform, ocular disease focus (overlap with NGGT001 BCD), and clinical-stage status.
- Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments for neurological and rare diseases using proprietary capsid engineering. Comparable on AAV platform and clinical-stage pipeline.
- Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for rare genetic diseases. Comparable on AAV vector platform and clinical-stage status targeting rare monogenic disorders.
- REGENXBIO: Clinical-stage gene therapy company using proprietary AAV vectors (NAV Technology Platform) to treat rare diseases including metabolic and ocular disorders. Most directly comparable to NGGT on AAV platform technology and rare disease focus.
- uniQure: Gene therapy company developing AAV-based treatments for rare diseases including Huntington's disease and hemophilia. Comparable on AAV platform, rare disease strategy, and clinical-stage gene therapy pipeline.
- MeiraGTx Holdings: Clinical-stage gene therapy company developing AAV-based treatments for rare diseases including ocular and metabolic disorders. Comparable platform technology and rare disease therapeutic focus.
- Krystal Biotech: Commercial-stage gene therapy company with FDA-approved Vyjuvek for dystrophic epidermolysis bullosa, using a topical redosable gene therapy platform. Comparable on rare disease gene therapy focus and integrated manufacturing.
Regional players
- Terns Pharmaceuticals: China-headquartered clinical-stage biotech with a pipeline spanning oncology and rare diseases, including gene therapy programs. Comparable as a similarly-staged China-US biotech with rare disease ambitions.
Market position
Strengths4 records
Weaknesses5 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
Next Generation Gene Therapeutics social profiles
Digital presenceNext Generation Gene Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Next Generation Gene Therapeutics leadership team
Management profileNumber of profiles
Profiles2 records
Next Generation Gene Therapeutics funding detail
Funding detailFunding overview
Funding rounds2 records
Investors8 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Next Generation Gene Therapeutics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Next Generation Gene Therapeutics
What does Next Generation Gene Therapeutics do?
Next Generation Gene Therapeutics (NGGT) is a clinical-stage biotechnology company that discovers, develops, and manufactures investigational recombinant adeno-associated virus (rAAV) gene therapies for rare genetic diseases, using its proprietary dual-functional vector platform. Its current pipeline consists of three investigational gene therapy drug products — NGGT001 for Bietti's Crystalline Dystrophy, NGGT002 for Phenylketonuria, and NGGT006 for Familial Hypercholesterolemia — produced at its 90,000-square-foot cGMP facility in Suzhou, China. NGGT is pre-commercial and monetizes through future product sales, licensing, and pharmaceutical partnerships.
Is Next Generation Gene Therapeutics a public or private company?
Next Generation Gene Therapeutics is a private company. It is classified as venture growth investor backed and is currently operating.
When was Next Generation Gene Therapeutics founded?
Next Generation Gene Therapeutics was founded in 2020. It employs 1 to 10 people.
Where is Next Generation Gene Therapeutics based?
Next Generation Gene Therapeutics is headquartered in Suzhou, China, in the Asia region.
How does Next Generation Gene Therapeutics make money?
Two revenue lines are on record. Gene Therapy Product Revenue is the primary driver. The others are licensing and Partnership Revenue.
Who are Next Generation Gene Therapeutics's main competitors?
Genprex is listed as an emerging player. BioMarin Pharmaceutical is listed as a broad incumbent. Direct peers are Adverum Biotechnologies, Voyager Therapeutics, Solid Biosciences, REGENXBIO, uniQure, MeiraGTx Holdings and Krystal Biotech. Terns Pharmaceuticals is listed as a regional player.
Does Next Generation Gene Therapeutics have an API?
No public API is recorded for Next Generation Gene Therapeutics.
What industry is Next Generation Gene Therapeutics in?
Next Generation Gene Therapeutics's product category is Gene Therapy Pharmaceuticals. Its primary akta.pro industry code is HLAAACAE, Gene Therapy for Rare/Monogenic Diseases, with a secondary code of HLAAACAK, Gene Therapy Analytical Testing & QC (potency, vg/ddPCR, infectivity, RCL/RCA). Its NAICS code is 541714 and its SIC code is 8731.