DNAI1

ReCode Therapeutics Doses First Patient in Phase 1 Clinical Study of Novel mRNA-based Therapy for the Treatment of Primary Ciliary Dyskinesia

Retrieved on: 
Wednesday, January 3, 2024

“This is the first time a patient with PCD has received a targeted mRNA-based therapeutic,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics.

Key Points: 
  • “This is the first time a patient with PCD has received a targeted mRNA-based therapeutic,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics.
  • “This study will evaluate RCT1100 as a potential novel treatment option for patients who do not have any approved therapies.
  • The DNAI1 mRNA is intended to establish or restore proper ciliary function by addressing the underlying cause of the disease.
  • This study brings us one step closer to improving the respiratory health and quality of life of people living with this disease.”

ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 for the Treatment of Primary Ciliary Dyskinesia

Retrieved on: 
Wednesday, March 15, 2023

Patients with PCD have a high burden of morbidity with chronic respiratory infections and bronchiectasis, and often develop respiratory failure.

Key Points: 
  • Patients with PCD have a high burden of morbidity with chronic respiratory infections and bronchiectasis, and often develop respiratory failure.
  • RCT1100 is designed to be a disease-modifying mRNA-based therapeutic for PCD caused by pathogenic mutations in DNAI1, a gene that encodes a protein essential for ciliary movement.
  • The intent is for the mRNA delivery to lead to DNAI1 protein production in target cells and thereby rescue ciliary function.
  • The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT1100.

ReCode Therapeutics Presents Preclinical Data from Inhaled mRNA Therapeutic Program in Primary Ciliary Dyskinesia (PCD) in Three Posters at the ATS 2022 International Conference

Retrieved on: 
Sunday, May 15, 2022

Patients with PCD have a high burden of morbidity with chronic respiratory infections, bronchiectasis, and often develop respiratory failure.

Key Points: 
  • Patients with PCD have a high burden of morbidity with chronic respiratory infections, bronchiectasis, and often develop respiratory failure.
  • ReCode is developing a disease-modifying mRNA-based approach for the treatment of PCD caused by mutations in DNAI1, an essential gene for ciliary movement.
  • Full details from the data presented at ATS 2022 can be accessed via the Publications section on ReCodes website.
  • ReCode will also present new preclinical data from its mRNA-based therapeutic program for cystic fibrosis on Wednesday, May 18th.

ReCode Therapeutics Raises Oversubscribed Series B Financing Round of $80 Million

Retrieved on: 
Thursday, October 21, 2021

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.
  • Existing investors who participated included OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, L.P., and Osage University Partners (OUP).
  • Oleg Nodelman, founder and portfolio manager of EcoR1 Capital also joined ReCodes Board of Directors in connection with the financing.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver.

Translate Bio Announces Second Quarter 2021 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, August 5, 2021

LEXINGTON, Mass., Aug. 05, 2021 (GLOBE NEWSWIRE) -- Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat or prevent debilitating or life-threatening diseases, today announced financial results for the second quarter ended June 30, 2021 and reviewed recent corporate updates.

Key Points: 
  • This transaction is a validation of our mRNA platform and a testament to the talented Translate Bio team.
  • Translate Bio ended the second quarter of 2021 with $667.2 million in cash, cash equivalents and investments and 75,343,712 shares of common stock outstanding.
  • General and administrative expenses of $11.9 million during the second quarter of 2021, compared to $8.6 million for the same period in 2020.
  • Translate Bio is also pursuing the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur.