Anagram Therapeutics

What Anagram Therapeutics does

Anagram Therapeutics is a clinical-stage biopharmaceutical company developing orally delivered enzyme replacement therapies for rare and life-threatening diseases driven by malabsorption syndromes and nutrient metabolism disorders. Founded in April 2023 and headquartered in Natick, Massachusetts, the company is led by co-founders Robert Gallotto (CEO) and Alexey Margolin (Chairman), supported by a leadership team drawn from Vertex Pharmaceuticals, Alcresta Therapeutics, Allena Pharmaceuticals, Alnara Pharmaceuticals, and other rare-disease biotechs. Its core technology is a proprietary enzyme stabilization platform designed to keep orally administered enzymes structurally intact and therapeutically active through the harsh gastric environment.

The company's lead candidate, ANG003, is a broad-spectrum, recombinant, non-porcine enzyme replacement therapy containing lipase, protease, and amylase, currently in a large international Phase 2 study for exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis, benchmarked against AbbVie's Creon. ANG003 is positioned to reduce the existing treatment burden of up to 40 daily capsules to a single tablet per meal and to address global supply shortages affecting porcine-derived PERT products. The broader pipeline includes ANG011 (congenital malabsorption syndrome), ANG021 (malabsorptive syndrome from pancreatic dysfunction), and ANG031 (congenital enzyme deficiencies causing toxic metabolite buildup).

Anagram is pre-revenue and operates as a privately held company whose economics are currently driven by equity capital rather than product sales, anchored by a $250 million investment from Blackstone Life Sciences in May 2026. Upon potential FDA approval, the company plans to commercialize through specialty pharmacy and PBM channels targeting rare-disease populations. Patient community engagement, including with the Cystic Fibrosis Foundation, medical conference presence (notably NACFC), and ClinicalTrials.gov listings form the core of its pre-commercial go-to-market motion.

Name

Anagram Therapeutics

Legal name

Anagram Therapeutics, Inc.

Website

https://anagramtx.com

Company type

Private

Founded year

2023

Operating status

Operating

Headcount range

11–50 employees

Short description

Anagram Therapeutics is a clinical-stage biopharmaceutical company developing orally delivered recombinant enzyme replacement therapies for rare malabsorption syndromes and nutrient metabolism disorders, with lead candidate ANG003 in Phase 2 for cystic fibrosis-related exocrine pancreatic insufficiency.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Enzyme Replacement Therapies

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Therapeutic Enzymes & Enzyme Replacement Therapies (ERT) (HLAAAAAK)

akta.pro secondary industry

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB)

Keywords

• Oral enzyme therapeutics
• Rare disease biopharmaceuticals
• Enzyme replacement therapy
• Malabsorption syndrome treatments
• Clinical-stage biotechnology

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Supply Chain, Marketing or Sales

Revenue model

1. Pharmaceutical Product Sales: As a clinical-stage biopharmaceutical company developing novel drugs, Anagram's primary revenue model will be through sales of prescription pharmaceutical products upon regulatory approval. The company is developing enzyme replacement therapies for rare diseases, which typically command premium pricing.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Anagram Therapeutics is a clinical-stage biopharmaceutical company developing orally delivered enzyme replacement therapies for rare and life-threatening diseases caused by malabsorption syndromes and nutrient metabolism disorders. Its lead candidate ANG003 is a broad-spectrum recombinant non-porcine enzyme replacement therapy containing lipase, protease, and amylase, currently in Phase 2 clinical trials for exocrine pancreatic insufficiency due to cystic fibrosis. The pipeline also includes ANG011, ANG021, and ANG031 targeting congenital malabsorption syndromes and related enzyme deficiencies.

Product overview

Anagram Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of orally delivered enzyme replacement therapies for rare and life-threatening diseases caused by malabsorption syndromes and nutrient metabolism disorders. The core product is ANG003 (Phase 2), a novel broad-spectrum recombinant non-porcine enzyme replacement therapy for exocrine pancreatic insufficiency. The pipeline includes ANG011 (congenital malabsorption syndrome), ANG021 (malabsorptive syndrome), and ANG031 (congenital enzyme deficiencies). All products leverage proprietary enzyme stabilization technologies designed to survive the harsh gastric environment and be immediately active in the gastrointestinal tract.

Differentiator

Problem solved

Functional benefit

Brands

• ANG003: Lead product candidate - a novel broad-spectrum recombinant non-porcine enzyme replacement therapy for exocrine pancreatic insufficiency in cystic fibrosis patients, currently in Phase 2 clinical trial.
• ANG011
• ANG021
• ANG031

Products and services

• ANG003 Lead product candidate — a novel broad-spectrum recombinant non-porcine enzyme replacement therapy containing lipase, protease, and amylase for exocrine pancreatic insufficiency (EPI) in cystic fibrosis patients. Currently in Phase 2 clinical trial. Designed to reduce treatment burden from up to 40 daily capsules to one tablet per meal.
• ANG011 Engineered recombinant enzyme candidate targeting congenital malabsorption syndrome, designed to address severe complications of congenital enzyme deficiencies.
• ANG021 Engineered enzyme candidate designed to break down specific macronutrient abnormalities due to pancreatic dysfunction, targeting malabsorptive syndrome.
• ANG031 Engineered recombinant enzyme candidate seeking to avoid the buildup of toxic metabolites in the body associated with congenital enzyme deficiencies.

Quantifiable outcome

• Reduces treatment burden from up to 40 daily capsules to just 1 tablet per meal
• +2 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core and minor.

• Cystic Fibrosis FoundationcoreStrategic or Co-development PartnerAnagram works with the Cystic Fibrosis Foundation to identify, develop, and deliver potentially life-changing therapies. The company engages with the rare disease patient community including the CF Foundation to understand patient perspectives and needs. The Foundation's Trial Finder provides information about Anagram's clinical trials.
• AbbVie (Creon)minorStrategic or Co-development PartnerAnagram's lead product ANG003 will be evaluated against AbbVie's Creon (a current standard of care pancreatic enzyme replacement therapy) in an upcoming Phase 2 study. This competitive benchmarking represents a form of partnership in clinical development.

Scale indicators3 records

Recent moves7 records

Expansion highlights6 records

Broad incumbents

• AbbVie: AbbVie markets Creon, the dominant pancreatic enzyme replacement therapy and ANG003's direct comparator in the Phase 2 trial. As the entrenched PERT leader with deep physician relationships and formulary positioning, AbbVie is the primary competitive benchmark for Anagram's lead asset.
• Nestlé Health Science: Nestlé Health Science markets Zenpep, a major porcine-derived PERT competing with Creon and directly relevant to the EPI/CF market Anagram is targeting. Operates within Nestlé's broader nutrition and consumer health portfolio.
• Genzyme (Sanofi): Genzyme pioneered the enzyme replacement therapy commercial model in rare diseases (Cerezyme, Fabrazyme, Aldurazyme) and offers a comparable rare-disease enzyme therapy commercial infrastructure and regulatory expertise that Anagram would need to build or partner to access.
• BioMarin Pharmaceutical: BioMarin is a rare-disease specialist commercializing multiple enzyme replacement therapies (Vimizim, Naglazyme, Aldurazyme) and represents the type of scaled, rare-disease-focused commercial organization Anagram could partner with or be benchmarked against for CF EPI commercialization.
• Vertex Pharmaceuticals: Vertex dominates the CF market with TRIKAFTA and the broader CFTR modulator franchise. Anagram's CMO led the TRIKAFTA launch there, and CF prescribers are concentrated among KOLs that Vertex already engages, making Vertex a critical account access consideration.
• Takeda Pharmaceutical: Takeda, through its Shire acquisition, operates a major rare disease franchise including enzyme replacement therapies (Replagal, Vpriv). Represents a comparable rare-disease commercial platform and a potential strategic acquirer for Anagram given its rare metabolic disease focus.

Regional players

• Chiesi Farmaceutici: Chiesi markets Pertzye, a third porcine-derived PERT approved in the U.S., making it a direct competitor in the EPI/CF therapeutic category. As a privately held mid-cap European pharma, it is a smaller but viable PERT incumbent.

Direct peers

• Alcresta Therapeutics: Alcresta developed RELiZORB, a non-porcine lipase cartridge for fat malabsorption in CF patients, and was co-founded by Robert Gallotto and Alexey Margolin, Anagram's co-founders. Direct overlap in CF malabsorption and shared founding team make it the most directly comparable prior venture.

Emerging players

• Protalix Biotherapeutics: Protalix develops plant-cell-based recombinant therapeutic proteins, including enzyme replacement therapies for rare diseases (e.g., Elelyso for Gaucher disease). Its plant-based expression platform represents an alternative non-mammalian approach to producing replacement enzymes.

Others

• Codexis: Codexis is a protein engineering company with a CodeEvolver platform for designing novel enzymes. It is thematically comparable as an enzyme engineering technology provider, though it operates primarily as a tool/ingredient supplier rather than a therapeutic developer.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/synspirapd
• LinkedInlinkedin.com/company/anagram-therapeutics-inc

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds4 records

Investors3 records

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 Anagram Therapeutics