Biopharma Spotlight: Neurogene and Rett Syndrome; Broken String and Francis Crick Institute and ALS; BioVersys and GSK; EyeDNA and Retinitis Pigmentosa
A look at some of today’s top life science stories
Neurogene Presents Favorable Rett Syndrome Gene Therapy Safety Data
Neurogene (New York) reported initial safety and tolerability data from its ongoing Phase I/II gene therapy clinical trial for Rett syndrome. NGN-401 was generally well-tolerated by the three patients dosed at about nine, six and three months after dosing. The gene therapy uses the company’s EXACT transgene regulation tech. Rett syndrome is a rare genetic neurologic disease that appears almost exclusively in girls. It causes severe impairments, including the ability to speak, walk, eat and even breathe. Constant repetitive hand movements are a hallmark of the syndrome. It is usually recognized in children between 6 to 18 months. Mutations in the MECP2 gene on the X chromosome cause the disease.
Broken String Biosciences and Francis Crick Institute Partner on ALS Research
Broken String Biosciences (Cambridge, England) entered a research partnership with the Francis Crick Institute (London) to develop novel applications for Broken String’s proprietary DNA break-mapping platform, INDUCE-seq. The partnership will focus on genomic instability in the development of amyotrophic lateral sclerosis (ALS). Prof. Simon Boulton and Dr. Nishita Parnandi at Crick are focused on genome stability and DNA double-strand break (DBS). Prof. Rickie Patani and Dr. Giulia Tyzack, at Crick, focus on the underlying mechanism of ALS.
Broken String’s R&D team, led by Professor Simon Reed, believes that the INDUCE-seq platform is well-suited for investigating ALS. Approximately 90% of ALS cases are sporadic, but why isn’t well understood. Boulton indicates their research is focused on how cells repair DNA damage and how “failures in this process lead to disease,” adding that they are “excited to leverage the INDUCE-seq platform’s unique capabilities in directly measuring and quantifying DNA double-strand breaks, and applying this to deepen our understanding of diseases that have genomic instability as a contributing factor, such as ALS.”
BioVersys Expands Collaboration with GSK and Extends its Series C Round by 12.3 Million CHF
BioVersys announced it has expanded its strategic collaboration with GlaxoSmithKline (GSK) to accelerate clinical development of alpibectir for the treatment of tuberculosis (TB). The small molecule was developed in a public-private collaboration with GSK, Pasteur Institute Lille and the University of Lille. It is currently in a Phase IIa trial in combination with ethionamide in pulmonary TB patients in South Africa. The expanded partnership will push development beyond the current Phase IIa trial to develop alpibectir in both TB pulmonary and TB meningitis. As part of the expansion, GSK is taking an equity stake in BioVersys’ latest investment round. This extends the Series C financing by CHF 12.3 million, ($13.56 million, US) bringing the total proceeds for the Series C to date to CHF 44.9 million ($49.51 million, US).
EyeDNA Announces Positive 24-Month Phase I/II Data for Retinitis Pigmentosa
Paris-based eyeDNA Therapeutics announced positive 24-month follow-up data from its Phase I/II trial of HORA-PDE6b for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6b gene. HORA-PDE6b is an AAV5-based gene replacement therapy. eyeDNA is partnered with Thea Open Innovation for the therapy. eyeDNA is responsible for global development and holds commercial rights in the US, Japan, South Korea, China and other territories outside Europe. In Europe and certain other countries, the gene therapy is being co-developed by Coave Therapeutics (which owns eyeDNA) and Thea Open Innovation.
“The highly encouraging safety and efficacy data observed in patients two years after treatment with HORA-PDE6b continue to support our view that this novel gene therapy could provide an important clinical benefit for PDE6b RP patients,” said Rodolphe Clerval, CEO of eyeDNA.