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Results For "rare-diseases"

123 News Found

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


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


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).


ValGenesis selected for validating French pharma firm
Digitisation | February 18, 2022

ValGenesis selected for validating French pharma firm

With ValGenesis VLMS, change control driven validation activities are reduced from weeks to mere hours, and the company gains faster access to detailed metrics that help reduce costs and cycle time


Moderna announces expansion in key Asian markets
News | February 16, 2022

Moderna announces expansion in key Asian markets

New subsidiaries to be established in Malaysia, Taiwan, Singapore, and Hong Kong


Sanofi unveils new corporate brand and logo
News | February 05, 2022

Sanofi unveils new corporate brand and logo

Sanofi unveiled a new bold and unifying corporate brand that supports the modernization and transformation the company launched in December 2019