Best in Biotech 3 Oct 2024 Six biotechs driving progress in Duchenne muscular dystrophy In this article, we take a look at six clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space. October 3, 2024 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 27 Sep 2024 Aptadir Therapeutics launches to develop new class of RNA inhibitors against cancer and genetic disorders Italian startup Aptadir Therapeutics has launched with $1.6 million to bring a new class of drugs to the clinic and a technology to be reckoned with. September 27, 2024 - 6 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 10 Jul 2024 Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder As treatments for skeletal dysplasia are intended to alleviate painful symptoms, two drugs are battling out in the clinic to prove their effectiveness. July 10, 2024 - 7 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 6 Jun 2024 CRISPR: A promising path to treating blindness Rigorous research, which has been going on for more than a decade now, suggests that CRISPR could treat genetic diseases including blindness. June 6, 2024 - 7 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Best in Biotech 6 Jun 2024 Eight biotech companies advancing the field of siRNA Explore the growing sector of siRNA and discover eight biotech companies spearheading this innovative field. June 6, 2024 - 11 minutesmins - By Jules Adam Share WhatsApp Twitter Linkedin Email
In Depth 23 May 2024 Is prime editing ready for prime time? Prime editing, a more advanced version of CRISPR/Cas9 technology, has been greenlit by the FDA to begin the first-ever clinical trial for this technology. May 23, 2024 - 7 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 10 Apr 2024 Silencing genes to cure diseases: an in-depth look at siRNA Delve into the promissing and dynamic sector of siRNA technology, with new drug approvals and advancements in delivery methods. April 10, 2024 - 8 minutesmins - By Jules Adam Share WhatsApp Twitter Linkedin Email
In Depth 14 Mar 2024 Rett syndrome: advances in gene therapy brings hope Explore the latest advancements in treating Rett syndrome, a rare genetic disorder, including groundbreaking gene therapies and drug trials. March 14, 2024 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 12 Feb 2024 RNA editing set to take off: could DNA’s short-lived cousin overcome the limitations of CRISPR gene editing? Find out all about about RNA editing, as two candidates have just managed to reach the clinic for the first time. February 12, 2024 - 7 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
News and Trends 28 Jul 2023 FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS Pharma Inc.’s NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy. The breakthrough designation is based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan. In June 2023, NS-089/NCNP-02 was granted Rare […] July 28, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 18 Jul 2023 Boost for Charcot-Marie-Tooth patients as Novartis acquires DTx Pharma DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its fatty acid ligand conjugated oligonucleotide (FALCON) platform, has been acquired by Novartis. The FALCON platform enables the delivery and activity of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake. DTx Pharma’s lead […] July 18, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 23 Jun 2023 Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […] June 23, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email