Rare disease Archives

Opinion 20 Sep 2023

The next frontier in genomic technologies for rare diseases

By Neil Ward, VP and General Manager at PacBio EMEA It’s been 40 years since the passage of the Orphan Drug Act, which catalyzed the development of hundreds of therapies…

September 20, 2023 - 7 minutesmins - By External Contributor

Duchenne muscular dystrophy gene therapy

In Depth 7 Sep 2023

Hope for patients with life-threatening muscle disorder after years of clinical trial failures

Duchenne muscular dystrophy is a rare condition that is typically treated with the help of steroids, to manage symptoms, as scientists are yet to close in on a cure. However,…

September 7, 2023 - 8 minutesmins - By Roohi Mariam Peter

News and Trends 14 Jul 2023

Life saved: AI discovers existing drug works for rare disease

A patient's life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, and Every Cure, used artificial intelligence to use an existing drug to treat…

July 14, 2023 - 4 minutesmins - By Jim Cornall

In Depth 10 Jul 2023

Unleashing the potential: Optimizing mRNA for the discovery of transformative medicines

By Dr. Alexander Zehnder, CEO, CureVac The success of mRNA vaccines in COVID-19 propelled the role of this new modality to the forefront of medicine. Not only are new prophylactic…

July 10, 2023 - 8 minutesmins - By External Contributor

News and Trends 6 Jul 2023

£100M LifeArc program aims to help people living with rare diseases

LifeArc, a self-funded, non-profit medical research organization and charity, is launching a new program to invest more than £100 million ($127.2 million) by 2030 to deliver new breakthroughs to improve…

July 6, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 5 Jul 2023

Narcolepsy drug gets Chinese approval

RareStone Group, a rare disease-focused company aiming to establish the first rare disease ecosystem in China, has announced that the Chinese National Medical Products Administration (NMPA) has approved pitolisant (Wakix)…

July 5, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 20 Jun 2023

BIO International Convention 2023: The Highlights

BIO International Convention, one of the largest networking events hosted for the biotech industry, has been influential in helping biopharmas discover new opportunities and building and strengthening partnerships within the…

June 20, 2023 - 14 minutesmins - By Dylan Kissane

In Depth 14 Jun 2023

How are biotechs addressing rare chromosome disorders?

Human beings typically have 23 pairs of chromosomes. But sometimes, in rare cases, an error during cell division can result in cells having extra or missing chromosomes, indicative of chromosomal…

June 14, 2023 - 7 minutesmins - By Roohi Mariam Peter

Osteopetrosis ADO2 radiopaedia case ID 8037

News and Trends 16 May 2023

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

SiSaf Ltd, an RNA delivery and therapeutics company, has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation…

May 16, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 12 May 2023

Cell and gene therapy company NewBiologix exits stealth with $50M

NewBiologix SA has emerged from stealth as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell…

May 12, 2023 - 3 minutesmins - By Jim Cornall

Podcast 12 May 2023

Beyond Biotech podcast 45: International MPS Awareness Day

This week’s podcast is sponsored by Quantoom. International MPS Awareness Day May is the big month when it comes to awareness - there are almost 100 awareness months, weeks, and…

May 12, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 28 Apr 2023

Mezzion Pharma raises $40M for treatment option for heart surgery patients

Mezzion Pharma, a rare disease focused pharmaceutical company, has secured nearly $40 million to fund FUEL-2, its confirmatory phase 3 clinical trial, along with future commercialization and regulatory submissions in…

April 28, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 24 Apr 2023

LifeArc launches £40M funding call to create Translational Rare Disease Centres

LifeArc is inviting applications from academic institutions for its latest £40 million ($49.8 million) initiative to fund four or five new Translational Rare Disease Centres in the U.K., along with…

April 24, 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…

April 21, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 12 Apr 2023

New technique developed for safer gene editing

CRISPR-Cas9 is widely used to edit the genome by studying genes of interest and modifying disease-associated genes. However, this process can be associated with potential side effects, including unwanted mutations…

April 12, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 29 Mar 2023

Pharming immunodeficiency drug given FDA approval

Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult…

March 29, 2023 - 4 minutesmins - By Jim Cornall

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 clotting protein factor IX often due to a spontaneous mutation…

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

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 body’s own defenses turn against itself. Here we’ll take a…

March 3, 2023 - 8 minutesmins - By Alexander Burik

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 people around the world living with a rare disease. More…

February 28, 2023 - 8 minutesmins - By Jim Cornall

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 Behring. The one-time gene therapy etranacogene dezaparvovec sold under the…

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

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