Paradigm Therapeutics

What Paradigm Therapeutics does

Paradigm Therapeutics, Inc. is a privately held, clinical-stage biopharmaceutical company headquartered in Mt. Pleasant, South Carolina, developing therapies for rare diseases with a singular focus on Epidermolysis Bullosa (EB). The company's lead and only investigational product, Zorblesia™ (SD-101), is a proprietary allantoin-based topical cream formulated for whole-body treatment of skin lesions, wounds, and blisters across all major EB subtypes (Simplex, Dystrophic, Junctional, and Kindler) in patients as young as 21 days old. SD-101 has received FDA Breakthrough Therapy designation — the first such designation for any EB treatment — along with FDA and European Commission Orphan Drug designation and FDA Rare Pediatric Disease designation. Phase II and Phase III trials have enrolled 217 patients, and a Phase 3 confirmatory study (SD-007) is enrolling approximately 80 pediatric patients across the US, Canada, Europe, South America, Mexico, Malaysia, and Australia in support of an NDA submission planned for the second half of 2025.

Paradigm acquired worldwide rights to SD-101 from Amicus Therapeutics in August 2023, and the company is led by Founder and CEO Dr. Robert Ryan, who previously co-founded Scioderm (an earlier EB topical therapy developer) and has 30+ years of pharmaceutical development experience with documented involvement in 20+ drug approvals. The leadership bench also includes veterans from Roche, Bristol-Myers Squibb, Pfizer, Quintiles, PPD, and ARIES ALLIANCE, covering regulatory affairs, clinical operations, finance, and business development. The company has 11–50 employees, reflecting a lean clinical-stage operating model.

The business model is prescription pharmaceutical sales upon FDA approval, with planned U.S. commercialization through specialty pharmacy channels and a parallel partner-enabled model for international distribution. The addressable patient population is estimated at 500,000 globally (20,000–50,000 in the U.S.), positioning SD-101 in orphan-drug economics where Priority Review Voucher eligibility further enhances value capture. As of 2025, Paradigm has raised $25.1 million from Eshelman Ventures, LLC ($12.5 million in May and $12.6 million in June), with Dr. Eshelman serving as strategic advisor; capital is earmarked for NDA preparation, manufacturing scale-up, and global commercial readiness.

Name

Paradigm Therapeutics

Legal name

Paradigm Therapeutics, Inc.

Website

https://paradigmtherapeutics.com

Company type

Private

Founded year

2023

Operating status

Operating

Headcount range

11–50 employees

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Dermatology Specialty Pharmaceuticals (HLAIACAL)

akta.pro secondary industries

Clinical Development (Phase I–III Trial Design & Execution) (HLAIALAE), Regulatory Strategy & IND/NDA/BLA Submissions Support (HLAIALAH)

Keywords

• Rare disease therapeutics
• Orphan drug development
• Topical dermatological therapy
• Biopharmaceutical clinical development
• Epidermolysis Bullosa treatment

Headquarters

HQ city

Mt Pleasant

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Others

Revenue model

1. Pharmaceutical Product Sales: Paradigm Therapeutics is a clinical-stage biopharmaceutical company preparing for revenue generation through sales of SD-101 (Zorblesia™) upon FDA approval. The company plans commercial launch in the U.S. and is working with potential global partners for international commercialization. Revenue will be generated through prescription drug sales, likely through specialty pharmacy channels typical for orphan/rare disease therapeutics.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

Paradigm Therapeutics is a clinical-stage biopharmaceutical company developing Zorblesia™ (SD-101), an investigational topical cream with allantoin as the active pharmaceutical ingredient, for whole-body treatment of skin lesions across all subtypes of Epidermolysis Bullosa (EB) in patients from 21 days of age and older. The company acquired worldwide rights to SD-101 from Amicus Therapeutics in August 2023 and is preparing an NDA submission planned for the second half of 2025 to support U.S. commercial launch.

Product overview

Paradigm Therapeutics is a clinical-stage biopharmaceutical company developing a single investigational product, Zorblesia™ (also known as SD-101). Zorblesia™ is a proprietary topical whole-body therapy for Epidermolysis Bullosa (EB), a rare genetic skin disorder. The product is applied as a cream across the entire skin surface and has completed Phase II and Phase III clinical trials, with an NDA submission planned for late 2025.

Differentiator

Problem solved

Functional benefit

Brands

• Zorblesia™: Investigational topical therapy being developed for treating whole body skin lesions (blisters, tears, sores) associated with all subtypes of Epidermolysis Bullosa (EB). Active ingredient is allantoin.

Products and services

• Zorblesia™ (SD-101) Zorblesia™ (SD-101) is an investigational proprietary topical cream with allantoin as the active pharmaceutical ingredient, developed for whole-body treatment of skin lesions (blisters, tears, wounds) across all subtypes of Epidermolysis Bullosa (EB) in patients from 1 month of age and older. The therapy has completed Phase II and Phase III clinical trials enrolling 217 patients with demonstrated efficacy in wound healing and a favorable long-term safety profile.

Quantifiable outcome

• 217 patients treated across Phase II and Phase III clinical trials demonstrating improvements in wound healing with excellent long-term safety profile
• +1 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Three partnerships are on record, tiered core and minor.

• Amicus TherapeuticscoreTechnology or Integration · 22 August 2023Paradigm Therapeutics completed acquisition of worldwide rights of SD-101, a topical whole-body treatment for all subtypes of Epidermolysis Bullosa, from Amicus Therapeutics. Terms of the deal were not disclosed. SD-101 had received Breakthrough Therapy designation and was a late-stage development asset.
• DEBRA InternationalminorOthersDEBRA International is a worldwide network of national groups working on behalf of those affected by epidermolysis bullosa. Paradigm Therapeutics supports this organization as part of its patient community engagement strategy.
• EB Research PartnershipminorOthersEB Research Partnership is the largest global organization dedicated to funding research to treat and cure Epidermolysis Bullosa. Paradigm Therapeutics supports this organization as part of its commitment to the EB community.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• BridgeBio Pharma: BridgeBio is a larger rare disease biotech with multiple late-stage assets across genetic conditions. Provides a comparable business model (rare disease, specialty commercialization) at greater scale.
• Ultragenyx Pharmaceutical: Ultragenyx is an established rare disease biotech with multiple approved and late-stage assets across genetic conditions. Comparable as a commercial-stage rare-disease specialist.
• Alexion Pharmaceuticals (AstraZeneca Rare Disease): Alexion operates the AstraZeneca Rare Disease unit with a portfolio of specialty/rare-disease therapeutics. Useful comparator for orphan-drug commercialization strategy and rare-disease market dynamics.

Direct peers

• Krystal Biotech: Krystal Biotech developed and commercialized Vyjuvek, the first FDA-approved topical gene therapy for dystrophic Epidermolysis Bullosa. Most direct commercial and clinical comparator to Paradigm's SD-101 in the EB topical therapy category.
• Amicus Therapeutics: Amicus Therapeutics is the company that divested worldwide rights to SD-101 to Paradigm in 2023. Operates in the rare disease space and is the originating developer of Paradigm's lead asset.
• Abeona Therapeutics: Abeona is developing pz-cel (prademagene zamikeracel), an autologous cell therapy for recessive dystrophic EB. Direct peer in the EB therapeutic space targeting the same patient population.
• Castle Creek Biosciences: Castle Creek developed dabocemagene autoficel, a cell therapy for EB. Direct peer in EB with a late-stage development asset targeting the same indication.
• Scioderm (acquired by Amicus Therapeutics): Scioderm was co-founded by Paradigm's CEO Dr. Robert Ryan and developed a topical therapy for EB that was later acquired by Amicus Therapeutics. Closely linked historical comparator in the EB topical therapy space.

Others

• Chiesi Farmaceutici: Chiesi acquired EB-related assets from Amicus Therapeutics. Relevant as a potential strategic acquirer / partner in the EB space and as a global specialty rare-disease commercializer.
• DEBRA International: Worldwide network of national EB patient advocacy organizations, including DEBRA of America. Already a partner of Paradigm; central stakeholder in patient access, reimbursement, and disease awareness for any EB therapy.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/paradigm-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds1 record

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Paradigm Therapeutics