AFTX-201 / Affinia Therap - LARVOL DELTA

Development of an investigational AAV gene therapy utilizing a novel cardiotropic capsid for patients with BAG3-associated dilated cardiomyopathy: AFTX-201 preclinical data and clinical plan (ESGCT 2026) - No abstract available

Gene therapy • Preclinical • Cardiomyopathy • Cardiovascular • Gene Therapies

AFTX-201, A Low-dose, Investigational AAV Gene Therapy Using A Cardiotropic Capsid For The Treatment Of BAG3-associated Dilated Cardiomyopathy: Journey To The Clinic. (HFSA 2026) - "The primary end point is safety and tolerability through 52 weeks. Secondary endpoints include myocardial BAG3 expression and functional efficacy measures relevant to heart disease, as well as safety over 5 years."

Gene therapy • IO biomarker • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • BAG1 • BAG3 • BCL2

Dilated Cardiomyopathies and Gene Therapy: UPBEAT© Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid (HFSA 2026) - "Sponsored by Affinia Therapeutics"

Gene therapy • Cardiomyopathy • Gene Therapies

Dilated Cardiomyopathies and Gene Therapy: UPBEAT© Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid (HFSA 2026) - "Sponsored by Affinia Therapeutics"

Gene therapy • Cardiomyopathy • Gene Therapies

Dilated Cardiomyopathies and Gene Therapy: UPBEAT© Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid (HFSA 2026) - "Sponsored by Affinia Therapeutics"

Gene therapy • Cardiomyopathy • Gene Therapies

Dilated Cardiomyopathies and Gene Therapy: UPBEAT© Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid (HFSA 2026) - "Sponsored by Affinia Therapeutics"

Gene therapy • Cardiomyopathy • Gene Therapies

IIE 07: Industry Innovation Events (No CEUs) - Dilated Cardiomyopathies and Gene Therapy: UPBEAT© Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid (HFSA 2026) - "The study design protocol of the UPBEA"

Gene therapy • Cardiomyopathy • Cardiovascular • Gene Therapies

UPBEAT: An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) (clinicaltrials.gov) - P1/2 | N=22 | Recruiting | Sponsor: Affinia Therapeutics | Initiation date: Jun 2026 ➔ Sep 2026

IO biomarker • Trial initiation date • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

Affinia Therapeutics…announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy (DCM). (Yahoo Finance) - "AFTX-201 is being evaluated as a treatment for BAG3 DCM in the UPBEAT clinical trial (NCT07426419) in the U.S. and Canada."

Orphan drug • Cardiomyopathy

UPBEAT: An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) (clinicaltrials.gov) - P1/2 | N=22 | Recruiting | Sponsor: Affinia Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • IO biomarker • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

UPBEAT: An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) (clinicaltrials.gov) - P1/2 | N=22 | Not yet recruiting | Sponsor: Affinia Therapeutics | Initiation date: Mar 2026 ➔ Jun 2026

IO biomarker • Trial initiation date • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

Study design of a phase 1/2 multicenter, open-label, dose-exploration and dose-expansion trial of AFTX-201 in adults with BCL2-associated athanogene 3 (BAG3) mutation–associated dilated cardiomyopathy (UPBEAT trial) (ASGCT 2026) - "Long-term follow-up is included to assess long-term safety and efficacy outcomes. The study is expected to initiate in 1H2026 and results from this study are intended to support further clinical development and ultimately registration."

Clinical • IO biomarker • P1/2 data • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • BAG1 • BAG3 • BCL2

Development, Technology Transfer, and Qualification of Product-Specific Methods for AFTX-201, an Investigational New Medicine for the Treatment of BAG3-Associated Dilated Cardiomyopathy (ASGCT 2026) - "This approach supports the advancement of AFTX- 201 while demonstrating a scalable framework for analytical characterization of next- generation AAV gene therapies. The framework and assays can be readily leveraged for developing additional product candidates in cardiac or skeletal muscle indications using the ATC-0187 capsid."

Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

AFTX-201: A Novel Investigational AAV Gene Therapy for the Treatment of BAG3-Associated Dilated Cardiomyopathy (ASGCT 2026) - "The integrated nonclinical data support the therapeutic rationale and advancement of AFTX-201 into a first-in-human Phase 1/2 clinical trial (UPBEAT trial) at a starting dose 5-10 fold lower than investigational cardiac gene therapies utilizing conventional AAV capsids. The UPBEAT trial is a multicenter, open-label, dose-exploration and dose-expansion trial to evaluate safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201, administered as a one-time IV dose, in adult patients with symptomatic BAG3 associated DCM."

First-in-human • Gene therapy • IO biomarker • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Hematological Disorders • BAG1 • BAG3 • BCL2

Process Development, Technology Transfer, Clinical Manufacturing and Process Characterization of AFTX-201, an Investigational New Medicine for the Treatment of BAG3-Associated Dilated Cardiomyopathy (ASGCT 2026) - "Representative scale-down models were established for both bioreactor production and UC purification, enabling effective process characterization. These models demonstrated comparability with the 50 L at-scale process, providing a strong foundation for continued clinical development and future commercialization."

Clinical • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

Affinia’s high-yield manufacturing process and analytical characterization of high-quality AFTX-201 clinical product highlighted (Businesswire) - "Results presented showcased a robust and scalable, high-yield AAV manufacturing process that was successfully developed by Affinia and transferred to Forge Biologics for Good Manufacturing Practice (GMP) production of AFTX-201, supporting clinical development. Of note, the GMP run achieved exceptional harvest titers (> 6e15 vg/L) and quality attributes, exceeding typical industry benchmarks and providing confidence in being able to meet commercial demand of thousands of patients per year at a 50 – 250L scale after potential product approval."

Preclinical • Cardiomyopathy

New AFTX-201 preclinical and translational data for BAG3 DCM featured at ASGCT 2026 (Businesswire) - "New data show that in BAG3 haploinsufficient mice...a single intravenous dose of AFTX-201 at 9e11 vg/kg showed full correction of cardiac function and structure...durable transgene expression...Increase in AFTX-201 pharmacodynamics...in non-human primates (NHPs)...AFTX-201 is generally well tolerated...doses 5-10-fold lower than conventional capsids."

Preclinical • Cardiomyopathy

Affinia’s Phase 1/2 UPBEAT clinical trial design for AFTX-201 in BAG3 DCM presented (Businesswire) - "The trial includes a dose-exploration phase followed by a dose-expansion phase. All participants will receive a single intravenous infusion of AFTX-201 at a dose that has been deemed safe and efficacious based on preclinical studies. The primary objective of the trial is to evaluate safety and tolerability through 52 weeks following administration...Multiple trial sites across the U.S. and Canada are planned to be available."

Clinical protocol • Cardiomyopathy

Affinia Therapeutics…announced that it will present new preclinical data on the company’s lead program AFTX-201, as well as highlight the design of the Phase 1/2 UPBEAT clinical trial investigating AFTX-201 in patients with BAG3-associated dilated cardiomyopathy (DCM), and Affinia’s proprietary capsid engineering and high-yield manufacturing process, in several oral and poster sessions (Businesswire) - "Two oral presentations will showcase AFTX-201 preclinical data and Affinia’s proprietary manufacturing process for AFTX-201; Affinia will also hold four poster presentations. The first poster provides an overview of the design of the UPBEAT Phase 1/2 clinical trial evaluating AFTX-201 in patients with BAG3-associated DCM. The second poster describes the development, optimization, and qualification of a comprehensive product-specific analytical panel for AFTX-201 that also sets the foundation for the development of additional product candidates. The company will also present a poster showcasing new results that further validate the central nervous system (CNS) transduction potential of ATC-134, Affinia’s novel engineered AAV capsid which exhibits widespread CNS expression in more than 90% of neurons after IV dosing in nonhuman primates."

Clinical protocol • Preclinical • Cardiomyopathy

Affinia Therapeutics Receives Approval from Health Canada to Initiate the UPBEAT Trial, a Phase 1/2 Clinical Trial to Investigate AFTX-201 as a Treatment for BAG3-Associated Dilated Cardiomyopathy (DCM) (Businesswire) - "Affinia’s Investigational New Drug (IND) application for AFTX-201 has been accepted by the U.S. Food and Drug Administration (FDA)..The UPBEAT clinical trial is a multicenter, single-arm open-label Phase 1/2 clinical trial designed to evaluate the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults with genetically confirmed BAG3-associated dilated cardiomyopathy. The trial includes a dose-exploration phase followed by a dose-expansion phase."

IND • New P1/2 trial • Cardiomyopathy

Affinia Therapeutics Granted FDA Fast Track Designation for AFTX-201 as a Treatment for People Living with BAG3-Associated Dilated Cardiomyopathy (DCM) (Yahoo Finance)

Fast track • Cardiomyopathy

UPBEAT: An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM) (clinicaltrials.gov) - P1/2 | N=22 | Not yet recruiting | Sponsor: Affinia Therapeutics

IO biomarker • New P1/2 trial • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3

Affinia Therapeutics Receives European Medicines Agency Orphan Drug Designation for AFTX-201 in BAG3-Associated Dilated Cardiomyopathy (DCM) (Businesswire)

Orphan drug • Cardiomyopathy

Development of a Flexible High Yielding, High Performing Process for Manufacturing of AFTX-201, a Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy (ASGCT 2025) - "Additionally, the high yields enabled a reduction in Toxicology and Clinical lot production scale from 250L to 50L, resulting in significant cost savings without compromising quality. Disease Focus of Abstract:Cardiovascular/Heart Disease"

Gene therapy • B Cell Lymphoma • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Lymphoma • Oncology • BAG3 • BCL2

A Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy (ASGCT 2025) - "This trial aims to evaluate the safety, tolerability, and pharmacodynamics of AFTX-201, administered as a single intravenous dose to adult patients with symptomatic BAG3 mutation-associated dilated cardiomyopathy (DCM). Disease Focus of Abstract:Cardiovascular/Heart Disease"

Gene therapy • IO biomarker • B Cell Lymphoma • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Lymphoma • Oncology • BAG3 • BCL2