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Results For "rare-disease"

212 News Found

Henlius' Serplulimab granted Orphan-Drug Designation for Small Cell Lung Cancer
Biotech | April 08, 2022

Henlius' Serplulimab granted Orphan-Drug Designation for Small Cell Lung Cancer

The company plans to file its NDA in China and MAA in the EU in 2022, making Hansizhuang potentially to be the world's first PD-1 inhibitor for the first-line treatment of SCLC


ASC Therapeutics gets green light to advance gene therapy for Hemophilia A
Biotech | March 30, 2022

ASC Therapeutics gets green light to advance gene therapy for Hemophilia A

A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies


New spesolimab data show promising results in patients with generalised pustular psoriasis flares
Biotech | March 28, 2022

New spesolimab data show promising results in patients with generalised pustular psoriasis flares

Clinical improvements were consistent across patient subgroups including age, gender, ethnicity, and IL-36 gene mutation status


Clarivate deals report highlights emerging trends in biopharma deal-making
Biotech | March 14, 2022

Clarivate deals report highlights emerging trends in biopharma deal-making

RNA, cell & gene therapies, artificial intelligence, CRISPR and oncology-related deals dominate the landscape


Emmes acquires Casimir, its fourth major acquisition
Biotech | March 11, 2022

Emmes acquires Casimir, its fourth major acquisition

Casimir further differentiates Emmes’ industry-leading rare disease research capabilities


Zydus Lifesciences subsidiary Sentynl acquires BridgeBio’s Nulibry
Biotech | March 09, 2022

Zydus Lifesciences subsidiary Sentynl acquires BridgeBio’s Nulibry

Nulibry is approved by the U.S. Food and Drug Administration (USFDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening paediatric genetic disorder


Suvoda unveils new brand to reflect expanded focus in complex clinical trial management
Biotech | March 08, 2022

Suvoda unveils new brand to reflect expanded focus in complex clinical trial management

Alongside brand refresh, the leading clinical trial technology company announces eConsent and eCOA offerings to better support the patient clinical trial journey


AstraZeneca and Neurimmune enter licencing agreement to develop and commercialise NI006
Biotech | March 01, 2022

AstraZeneca and Neurimmune enter licencing agreement to develop and commercialise NI006

Under the terms of the agreement, the upfront payment from Alexion to Neurimmune is US $ 30m


GliaPharm SA collaborate with Wyss Centre for Alzheimer therapy
Biotech | February 25, 2022

GliaPharm SA collaborate with Wyss Centre for Alzheimer therapy

The collaboration will accelerate the testing of GliaPharm’s lead molecules in preclinical models of Alzheimer’s disease and further the development of neuroimaging biomarkers


Biohaven licenses Taldefgrobep Alfa from Bristol Myers Squibb
Biotech | February 25, 2022

Biohaven licenses Taldefgrobep Alfa from Bristol Myers Squibb

It is a Phase 3 ready anti-myostatin adnectin for Spinal Muscular Atrophy (SMA).