Rare disease Archives

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 mRNA vaccines for various respiratory diseases advancing through clinical development, but mRNA medicines are now being developed in many wide-ranging applications, including cancer and rare […]

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 the lives of people living with a rare disease. The Rare Disease Translational Challenge will bring together researchers in rare disease with LifeArc to get […]

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) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. In China, pitolisant is the first approved innovative drug for […]

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 biotech and pharma community. This year, the event took place from June 5 to 8, in the bustling biotech hotspot of Boston, Massachusetts, and saw […]

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 disorders. Often, prenatal tests are carried out to detect chromosomal abnormalities. Amniocentesis is a diagnostic procedure where the amniotic fluid that protects a fetus during […]

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 by the U.S. FDA. Also, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted rare pediatric disease 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 therapies. The company’s DNA-based platform is being developed to address key manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to […]

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 days being marked. Some of these are well known conditions. Others, however, are less prominent. International MPS (mucopolysaccharidosis) Awareness Day takes place on May 15. […]

May 12, 2023

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 other countries including Europe. Mezzion Pharma is looking to bring to market JURVIGO (udenafil), a highly selective, unique and potent phosphodiesterase type 5 (PDE-5) inhibitor, […]

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 a separate co-ordinating hub. LifeArc, which is a self-funded, non-profit medical research charity, specializes in early-stage translation – the advancement of scientific discoveries towards patient […]

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 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

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 and toxicity. Researchers at Kyushu University and Nagoya University School of Medicine in Japan have developed an optimized genome-editing method that vastly reduces mutations, opening […]

April 12, 2023 - 4 minutesmins - By Jim Cornall

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T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

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Rare disease

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

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

Narcolepsy drug gets Chinese approval

BIO International Convention 2023: The Highlights

How are biotechs addressing rare chromosome disorders?

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

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

Beyond Biotech podcast 45: International MPS Awareness Day

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

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

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

New technique developed for safer gene editing

Subscribe to our newsletter to get the latest biotech news!

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

T cell engagers: A promising, fast-growing field for cancer and autoimmune disease treatments

The biggest biotech funding rounds in February 2025

Grab your free report on 3D organoids and explore their future potential

World Cancer Day: trends in cancer therapeutics to look forward to in 2025

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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