Engineering the future of gene therapy

01 Overview

Gene therapy, built for the kidney.

Nionyx is developing next-generation AAV gene therapies for genetic kidney diseases.

Engineering the future of gene therapy

Gene therapy has transformed treatment in the eye, liver, muscle, and central nervous system. The kidney has been left behind — because until recently, most AAVs couldn't reach the cells inside the kidney where treatment needs to happen.

We built our platform human-first. By screening human whole-kidney explants directly, we identified our lead capsids which were able to target kidney cells.

02 Vision

Why kidney. Why now.

Gene therapy has reached therapeutic areas such as the eye, liver, muscle, hearing and CNS. The kidney is the next frontier.

0
Approved gene therapies for kidney disease
04 Investors

Backed by investors building the next generation of biotech.

$4M seed financing closed September 2025, led by 2048 Ventures.

01 Platform

Next gen AAV capsids

De-risked in human kidneys, validated in multiple models

Capsid
Next-gen AAVs
Cassette
Purpose-built payloads
Delivery
Direct to the kidney
02 Trifecta

Three parts, one system.

01
Capsid

Next-generation AAVs

NYX Capsids, identified by screening human whole-kidney tissue. Reach cells inside the kidney at higher multiples than other AAV.

02
Cassette

Purpose-built payloads

Designed for specific kidney diseases.

03
Delivery

Direct to the kidney

Our route of administration allows for smaller, more efficacious doses.

03 Validation

Validated across numerous models.

01 · Ex vivo

Whole human kidney explant

02 · In vitro

Cell lines

03 · In vivo

Healthy and diseased small and large animal models

04 Approach

Built human-first.

Most gene therapy platforms are built in mice. Nionyx was built the other way around. We screened human whole-kidney tissue first — then validated down the model chain, not up.

That philosophy matters because building "human-first" means the questions we most need to answer are answered first, not last.

05 Pipeline

A pipeline built for the kidney's most-underserved diseases.

Program DiscoveryPreclinicalIND EnablingPhase 1/2 Stage
ONYX-101
Alport syndrome
Preclinical
Early-stage programs
Additional kidney indications
Discovery
Back to Science
ONYX-201 Discovery Program

A gene therapy approach to APOL1 kidney disease.

Targeting one of the most common genetic causes of kidney failure, in a population historically overlooked in drug development.

IndicationAPOL1 kidney disease
ModalityAAV gene therapy
TargetAPOL1
StageDiscovery
01 The Disease

APOL1 kidney disease.

A common genetic driver of kidney failure — with no approved therapies that address the underlying cause.

APOL1 kidney disease is caused by specific variants of the APOL1 gene that dramatically increase the risk of progressive kidney failure. The variants are far more common in people of recent West African ancestry, and are a leading contributor to the disparity in kidney outcomes between Black and non-Black Americans.

Despite the size of the affected population, no approved therapy addresses the underlying APOL1 biology.

02 Our Approach

Silencing the risk. Restoring the baseline.

ONYX-201 combines two mechanisms: silencing the high-risk APOL1 variants that drive disease, and expressing a protective form of the protein. Built on the same NYX platform as ONYX-101.

03 Status

Where we are.

ONYX-201 is in discovery. The therapeutic construct has been built. In vivo proof-of-concept studies are planned.

Back to News
September 29, 2025

Nionyx Bio Launches with $4M Seed to Transform AAV Gene Therapy for Kidney Disease with Proprietary Capsid Platform and Pipeline

  • Raised $4M seed financing led by 2048 Ventures, with participation from New York Ventures (Empire State Development), White Mug Healthcare, AIN Ventures, and Pathway Bioventures
  • Proceeds will advance Nionyx’s proprietary capsid platform, AAV Kidney Atlas, to enable first-in-class, one-time gene therapies for renal diseases, and support early pipeline development
  • Led by industry veterans with proven expertise in gene therapy, kidney biology, and clinical development
“Gene therapy delivery is entering its next phase, defined by precision,” said Magdalena Tyrpien, Co-Founder, President & CEO of Nionyx. “Our mission is to set a new standard for how therapies are designed, built on efficiency, safety, and scale. By focusing on precision delivery, we aim to unlock better outcomes for patients while minimizing risk. At Nionyx, we are committed to translating these advances into potentially transformative treatments and we are proud to partner with 2048 Ventures and a syndicate that shares our urgency to expand therapeutic options.”

Nionyx is building the first AAV Kidney Atlas, a comprehensive reference of capsid tropism in human kidney tissue, and advancing a pipeline of targeted, potentially one-time gene therapies for patients with kidney disease. Our platform spans capsid, cassette, and delivery, all designed to engineer the future of gene therapy with greater precision and safety.

“We believe the most important breakthroughs come from founders tackling big problems with purpose and innovation. The Nionyx platform approach to kidney gene therapy is timely and ambitious, and we're proud to back their mission to deliver safer, one-time treatments to patients” said Julie Wolf, PhD, Partner at 2048 Ventures and member of the Nionyx Board.

Veteran Gene Therapy Leadership Team

Nionyx is launching with a veteran leadership team, bringing proven expertise in gene therapy, kidney biology, and clinical development:

  • Magdalena Tyrpien, MBA, Co-Founder, President & CEO – former Chief Business Officer at Forge Biologics, where she led the company’s $620M acquisition by Ajinomoto; seasoned gene therapy executive with senior leadership roles at PTC Therapeutics and Abeona Therapeutics, bringing extensive experience in business development, transactions, and capital formation across rare disease and gene therapy.
  • Prof. Leszek Lisowski, PhD, MBA, Co-Founder & CTO – internationally recognized AAV capsid engineer and co-founder of LogicBio Therapeutics; formerly at Stanford University and the Salk Institute, now Unit Head of the Translational Vectorology Research Unit at Children’s Medical Research Institute (CMRI), with over 20 years of leadership in gene therapy vector design, development, and translation.
  • Juan Ruiz, MD, PhD, MBA, Chief Medical Officer, Head of Preclinical Development – previously Senior Vice President of Medical Affairs at Forge Biologics and Chief Medical Officer at Abeona Therapeutics; seasoned rare disease executive with over 25 years of leadership spanning gene therapy development, regulatory filings, and global preclinical and clinical programs from IND to Phase II.

About Nionyx Bio

Nionyx Bio is redefining gene therapy for the kidney. Its proprietary AAV capsid platform is designed to deliver one-time, tissue-targeted therapies with improved safety, precision, and scalability. Backed by top-tier investors and led by a team of gene therapy pioneers, Nionyx is building the AAV Kidney Atlas to de-risk development and accelerate a new class of renal gene therapies.

About 2048 Ventures

2048 Ventures is a thesis-driven early stage venture capital firm based in NYC and Boston and is investing across US. 2048 ventures writes $500K - $2M checks and lead Pre-Seed or Seed rounds in Vertical AI, Deep Tech, Healthcare and Biotech. For more information, visit 2048.vc.

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