dirloctocogene samoparvovec (RG6797) / Roche - LARVOL DELTA

SPK-8011QQ-201: A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=5 | Recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Jul 2031 ➔ Apr 2032 | Trial primary completion date: Jul 2031 ➔ Apr 2032

Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Update on a Novel Investigational Adeno-associated Virus Gene Therapy, SPK-8011QQ: Rationale and Design of a Phase IIb Trial (THSNA 2026) - "Preclinical data support leveraging the low-dose SPK-8011 platform, while introducing the aPC-resistant enhanced-function FVIII-QQ variant. The Phase IIb trial will provide insights into the safety of the investigational AAV gene therapy, SPK-8011QQ. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Gene therapy • P2b data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

SPK-8011QQ-201: A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=5 | Recruiting | Sponsor: Hoffmann-La Roche | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4

SPK-8011QQ-201: A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=5 | Not yet recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Jan 2031 ➔ Jul 2031 | Trial primary completion date: Jan 2031 ➔ Jul 2031

Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4

SPK-8011QQ-201: A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A (clinicaltrials.gov) - P1/2 | N=5 | Not yet recruiting | Sponsor: Hoffmann-La Roche | Phase classification: P2 ➔ P1/2

Phase classification • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • CD4

Preclinical evaluation of SPK-8011QQ, an adeno-associated virus gene therapy for people with hemophilia A leveraging the dirloctocogene samoparvovec platform encoding an activated Protein C-resistant B-domain deleted factor VIII (ASH 2025) - "Surrogate SPK-8011QQ, which leverages the previously clinically evaluated SPK-8011(dirloctocogene samoparvovec) platform to introduce FVIII-QQ, demonstrates markedly enhancedhemostatic potency under APC-sensitive conditions in ex vivo and in vivo mouse models. Furthering thelearnings from the safety and durability of the SPK-8011 platform, our preclinical data collected to datesupport the ongoing evaluation of SPK-8011QQ, which aims to optimize FVIII potency and hemostaticpotential."

Gene therapy • Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A (clinicaltrials.gov) - P2 | N=5 | Not yet recruiting | Sponsor: Hoffmann-La Roche

New P2 trial • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Haemophilia A (Roche Press Release) - "Hemlibra (emicizumab): New post-marketing data from the Beyond ABR study show that, in the first year after switching to Hemlibra prophylaxis from factor VIII prophylaxis, people with various levels of baseline joint impairment had low bleeding rates, associated with overall improvements in joint health, and a shift towards higher activity levels...NXT007: Positive phase I/II results, including new data from a global study in people with haemophilia A with and without factor VIII inhibitors....SPK-8011QQ: Pre-clinical data on Roche’s next-generation investigational AAV gene therapy, show significantly enhanced haemostatic potency compared with SPK-8011 (dirloctocogene samoparvovec) in ex vivo and in vivo mouse models. Findings support the ongoing evaluation of SPK-8011QQ, furthering previous learnings on the safety and durability of SPK-8011, with phase IIb study initiation planned for 2026."

Clinical data • New P2b trial • Preclinical • Hemophilia A