Interview 3 Mar 2023
Interview: how has the first gene therapy for hemophilia B transformed research?
Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective…
Rare disease
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In Depth 3 Mar 2023
Let’s Take a Closer Look at 7 Autoimmune Diseases Biotechs are Fighting to Treat
While we mostly think of disease as being caused by bacteria or viruses, an illness can often occur when the…
News and Trends 28 Feb 2023
What’s happening on Rare Disease Day?
Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million…
News and Trends 28 Feb 2023
European Commission approves first gene therapy for hemophilia B
The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL…
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News and Trends 28 Feb 2023
GC Biopharma acquiring hemophilia rare disease pipeline
GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to…
Opinion 28 Feb 2023
Life changing treatments for rare disease – a beacon of hope
By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease.…
Best in Biotech 28 Feb 2023
5 advancements in rare disease research over the past year
There are more than 7,000 rare diseases that affect hundreds of millions of people worldwide. As Rare Disease Day is…
Best in Biotech 28 Feb 2023
10 biotech companies making a difference in rare diseases
February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients…
News and Trends 24 Feb 2023
Beyond Biotech podcast 35: Rare Disease Day
As February 28 is Rare Disease Day, we have three guests on the podcast today all relevant to rare diseases.…
News and Trends 23 Feb 2023
Moderna and Life Edit Therapeutics look to speed up gene editing therapies
Moderna, Inc., a biotechnology company working on messenger RNA (mRNA) therapeutics and vaccines, and Life Edit Therapeutics Inc., an ElevateBio…
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News and Trends 21 Feb 2023
First patient enrolled in Pharming APSD trial
Pharming Group N.V. says the first patient has been enrolled in its phase III clinical trial evaluating the investigational drug…
News and Trends 21 Feb 2023
FDA approves alpha-mannosidosis drug
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the…
News and Trends 17 Feb 2023
Positive results for INOVIO recurrent respiratory papillomatosis trial
INOVIO Pharmaceuticals has announced positive preliminary results from the second cohort of its phase 1/2 clinical trial evaluating INO-3107 for…
News and Trends 10 Feb 2023
Janssen reports positive topline phase 2 HDFN results
The Janssen Pharmaceutical Companies of Johnson & Johnson has announced positive topline results from the proof-of-concept phase 2 open-label UNITY…
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2 Feb 2023Improving rare disease diagnosis and treatment with next generation sequencing technology
Rare diseases refer to over 7,000 different known health conditions that together affect up to over 350 million individuals worldwide.…
News and Trends 19 Jan 2023
Hundreds of genetic discoveries from FinnGen study
New results from the FinnGen research consortium include previously unknown genetic risk factors for many debilitating diseases. These findings have…
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In Depth 11 Jan 2023
Drug repurposing emerges as viable option for rare disease treatment
With few options available for the treatment of rare diseases, the practice of drug repurposing has gained traction as an…
Opinion 12 Dec 2022
Artificial intelligence: a new era in rare genetic disease diagnosis
Despite the onset of the genomics era, rare disease diagnosis remains a challenge. Nostos Genomics’ co-founder, Rocío Acuña Hidalgo, and…
News and Trends 7 Dec 2022
PacBio and Boston Children’s Hospital to study genome to benefit patients
PacBio, a developer of sequencing solutions, says its HiFi sequencing technology will be used in a pilot project for the…
News and Trends 2 Dec 2022
Khondrion’s sonlicromanol produces encouraging results for next stage of study
Biopharma company, Khondrion, has marked announced improvements from patients with MELAS spectrum disorders taking its lead asset, sonlicromanol. Updates were…