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Results For "rare-diseases"

109 News Found

Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine
Biotech | May 13, 2022

Neurocrine Biosciences receives Orphan Drug Designation for Valbenazine

The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation


Novo Nordisk and Flagship Pioneering partner to create a portfolio of transformational medicines
Biotech | May 10, 2022

Novo Nordisk and Flagship Pioneering partner to create a portfolio of transformational medicines

Pioneering Medicines and Novo Nordisk Bio Innovation Hub will leverage Flagship Pioneering’s bioplatform companies to create a portfolio of research programmes within cardiometabolic and rare diseases


Strengthening the access to rare disease treatment in India
Healthcare | May 09, 2022

Strengthening the access to rare disease treatment in India

Takeda and United Nations Global Compact Network of India (UNGCNI) join hands


NORD announces over US $ 100,000 in grant funding available for rare disease research
Biotech | May 02, 2022

NORD announces over US $ 100,000 in grant funding available for rare disease research

Three RFPs now open for qualified researchers through NORD's Jayne Holtzer rare disease research grants program


Genome Insight draws US $ 23 million in Series B Funding
Startup | May 01, 2022

Genome Insight draws US $ 23 million in Series B Funding

Funds will be allocated to accelerate rapid production and high-end curation of whole genome sequences for its clinical application


Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine
Biotech | April 28, 2022

Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine

Danish National Genome Center partners with Lifebit to deliver nationwide personalised medicine


Bridge Biotherapeutics enters into an exclusive license agreement with Shaperon
Biotech | April 19, 2022

Bridge Biotherapeutics enters into an exclusive license agreement with Shaperon

By strengthening the company's fibrotic disease development pipeline, Bridge will enhance its strategic focus on the development of comprehensive treatment solutions for idiopathic pulmonary fibrosis (IPF)


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


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