genetic disorders Archives

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

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

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

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

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

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

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

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

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

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

News and Trends 9 May 2023

Fabry disease treatment gets EU marketing authorization

Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc. have announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) for the treatment of adult patients with Fabry disease. “People living with Fabry disease often perceive their disease as burdensome and still experience unmet medical needs,” said […]

May 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 21 Apr 2023

Orphalan announces U.S. launch of drug to treat Wilson disease

Orphalan SA, an international orphan drug development and commercialization company, has announced the commercial launch in the U.S. for Cuvrior. Cuvrior is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine. Cuvrior was approved by the United States […]

April 21, 2023 - 3 minutesmins - By Jim Cornall

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