Port Therapeutics

What Port Therapeutics does

Port Therapeutics is a privately held, clinical-stage biotechnology company headquartered in Los Angeles, California, founded in 2021. The company is developing a category of therapeutics it terms "Spatiotemporal Therapeutics" — drugs designed to be activated at the right anatomical place and at the right time to address what it characterizes as locally presenting diseases that are difficult to treat with systemic interventions. Its lead asset, PORT-77, is an investigational drug candidate for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), rare genetic disorders causing severe photosensitivity due to accumulation of protoporphyrin IX (PPIX). In its Phase 2a GATEWAY trial, PORT-77 demonstrated 79% mean plasma PPIX reduction in the high-dose cohort and 63% in the low-dose cohort, with no serious adverse events or treatment discontinuations. The company plans to engage with the FDA via an End-of-Phase 2 meeting and initiate a Phase 2b/3 PATHWAY trial in Q3 2026, targeting an addressable population of over 25,000 EPP/XLP patients in the US and EU where no disease-modifying therapies are currently approved.

Port Therapeutics has 1-10 employees and operates as a venture-backed private company, with Catalio Capital Management and Cherubic Ventures identified as investors from a 2023 funding round (undisclosed amount). Scientific founders include Mikhail G. Shapiro (Caltech, HHMI Investigator, NIH Pioneer), Gabe A. Kwong (Georgia Tech & Emory, NIH New Innovator), and Sangeeta N. Bhatia (MIT, NAE/NAS/NAM/NAI). Leadership includes Steve Altschuler, MD as Executive Chair, João Siffert, MD as Consultant, and Lena Gamboa, PhD as Head of Cancer Immunotherapy — the latter signaling an organizational expansion into oncology beyond the EPP/XLP core indication. Port Therapeutics is pre-revenue, has no commercial distribution or marketing channels in operation, and has no disclosed partnerships, acquisitions, or collaborations in the provided material.

Name

Port Therapeutics

Legal name

Port Therapeutics

Website

https://porttherapeutics.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

1–10 employees

Short description

Port Therapeutics is a Los Angeles-based, clinical-stage biotechnology company developing spatiotemporal therapeutics, with lead candidate PORT-77 in Phase 2 development for erythropoietic protoporphyria and X-linked protoporphyria, rare genetic photosensitivity disorders affecting over 25,000 patients in the US and EU.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Rare disease therapeutics
• Spatiotemporal therapeutics
• Porphyria treatment
• Biopharmaceutical drugs
• Clinical stage biotech

Headquarters

HQ city

Los Angeles

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, Operations, Marketing or Sales, Others

Revenue model

1. Pharmaceutical Product Revenue: Pre-revenue clinical stage company developing PORT-77 for erythropoietic protoporphyria. Upon regulatory approval, the company expects to commercialize PORT-77 as a disease-modifying treatment for EPP and XLP, targeting a rare disease population of over 25,000 patients in the US and EU with no currently approved disease-modifying therapies.

Go-to-market motion1 record

Core offering

Port Therapeutics is a clinical-stage biotechnology company developing PORT-77, an investigational disease-modifying drug candidate for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). The candidate is built on a proprietary Spatiotemporal Therapeutics platform designed to activate drugs at the right place and time, targeting rare genetic disorders of the heme biosynthesis pathway that currently have no approved disease-modifying treatments.

Product overview

Port Therapeutics is pioneering a new category of spatiotemporal therapeutics that can be activated at the right place and for the right time to address local disease problems. The company's product portfolio includes PORT-77, an investigational drug candidate for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) that has demonstrated disease-modifying potential in Phase 2a trials. The company is led by scientific founders from Caltech, Georgia Tech, MIT, and Emory, and is developing therapeutics designed to overcome limitations of systemic interventions.

Differentiator

Problem solved

Functional benefit

Products and services

• PORT-77 Investigational disease-modifying drug candidate for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). Phase 2a GATEWAY trial demonstrated 79% plasma PPIX reduction in the high-dose cohort and 63% in the low-dose cohort, with a favorable safety profile showing no serious adverse events or treatment discontinuations. Targeted at the rare disease population of over 25,000 patients in the US and EU combined who currently have no approved disease-modifying treatment.

Quantifiable outcome

• 79% mean plasma PPIX reduction in high-dose cohort; 63% in low-dose cohort
• +1 more outcomes

Segments2 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Scale indicators1 record

Recent moves6 records

Expansion highlights5 records

Direct peers

• Mitsubishi Tanabe Pharma: Mitsubishi Tanabe developed dersimelagon (MT-7117), an oral melanocortin-1 receptor agonist approved in Japan for EPP, directly competing with Port Therapeutics in the same patient population with a marketed product.
• Disc Medicine: Disc Medicine (acquired by Eli Lilly in 2024/2025) developed bitopertin, a disease-modifying therapy for erythropoietic protoporphyria (EPP), making it the closest direct competitor to PORT-77 in the same indication and patient population.
• Clinuvel Pharmaceuticals: Clinuvel markets Scenesse (afamelanotide), the only approved EPP therapy in the US and EU, and represents both a direct competitor and the standard-of-care benchmark Port Therapeutics must beat or differentiate against.

Broad incumbents

• BioMarin Pharmaceutical: BioMarin is an established rare disease biotech with a portfolio of approved enzyme replacement and gene therapies for ultra-rare conditions, representing a benchmark for orphan drug commercialization, pricing, and acquisition comparables.
• Alnylam Pharmaceuticals: Alnylam developed givosiran (Givlaari) for acute hepatic porphyria and operates a leading RNAi therapeutics platform targeting rare genetic diseases — overlapping disease space (porphyrias) and platform strategy with Port Therapeutics' rare genetic disease focus.
• Ionis Pharmaceuticals: Ionis is an established antisense oligonucleotide platform company with multiple approved rare disease therapies (e.g., Spinraza, Tegsedi, Waylivra), representing a comparable rare genetic disease drug development and commercialization model.
• Ultragenyx Pharmaceutical: Ultragenyx is a commercial-stage rare disease biotech with multiple approved products across small patient populations, providing a comparable business model and commercialization playbook for orphan disease drug launches.
• Alexion Pharmaceuticals (AstraZeneca Rare Disease): Alexion is the rare disease arm of AstraZeneca and a leading acquirer/integrator of late-stage rare disease biotechs, making it directly comparable as both a potential partner/acquirer and as a benchmark for rare disease commercial economics.

Emerging players

• Intellia Therapeutics: Intellia is a clinical-stage in vivo gene editing company developing CRISPR/Cas9 therapies for rare genetic diseases such as transthyretin amyloidosis and hereditary angioedema, comparable as an emerging rare-disease modality company pursuing first-in-class therapies.
• CRISPR Therapeutics: CRISPR Therapeutics develops CRISPR/Cas9-based gene-editing therapies for rare genetic diseases including sickle cell disease and beta-thalassemia, sharing the same first-in-class genetic medicine playbook as Port Therapeutics.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/porttherapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds1 record

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 Port Therapeutics