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Results For "rare-diseases"

109 News Found

Moderna and CytomX partner for mRNA-based conditnionally activated therapeutics
News | January 11, 2023

Moderna and CytomX partner for mRNA-based conditnionally activated therapeutics

Collaboration to generate and develop therapeutics for oncology and non-oncology conditions


Moderna announces advances across mRNA pipeline
News | January 10, 2023

Moderna announces advances across mRNA pipeline

Moderna and Merck announced mRNA-4157/V940, in combination with KEYTRUDA, demonstrated a 44% reduction in the risk of disease recurrence or death in melanoma patients


Applied Therapeutics partners with Advanz Pharma for commercialization of Govorestat in Europe
Biotech | January 05, 2023

Applied Therapeutics partners with Advanz Pharma for commercialization of Govorestat in Europe

Advanz Pharma will leverage its specialty, hospital, and rare disease expertise and infrastructure to commercialize AT-007 in Europe for both Galactosemia and SORD Deficiency


Takeda launches CINRYZE in India for hereditary angioedema patients
News | December 15, 2022

Takeda launches CINRYZE in India for hereditary angioedema patients

CINRYZE is plasma-derived C1-I NH approved for routine prevention (prophylaxis), short-term prevention or pre-procedure prevention, and acute attacks of HAE.


Emmes launches Cell and Gene Therapy Center
News | December 09, 2022

Emmes launches Cell and Gene Therapy Center

The new center will focus on supporting the clinical trials clients who are developing cell and gene therapies


AstraZeneca receives import and market permission from CDSCO for Dapagliflozin
Drug Approval | November 26, 2022

AstraZeneca receives import and market permission from CDSCO for Dapagliflozin

The receipt of this permission paves way for the launch of Dapagliflozin (Forxiga) tablets of 10 mg in India for the specified additional/expanded indication, subject to the receipt of related statutory approvals


Inceptua Early Access and Sentynl Therapeutics launch early access program for Nulibry for pediatric patients
News | October 19, 2022

Inceptua Early Access and Sentynl Therapeutics launch early access program for Nulibry for pediatric patients

MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae


CHMP recommends conditional marketing authorization for Spesolimab for generalized pustular psoriasis flares
News | October 17, 2022

CHMP recommends conditional marketing authorization for Spesolimab for generalized pustular psoriasis flares

CHMP positive opinion is based on evidence from the EFFISAYIL trial, the largest clinical trial in


BioMarin simplifies organizational structure to increase efficiency
News | October 09, 2022

BioMarin simplifies organizational structure to increase efficiency

The reduction in force will result in financial savings of approximately $50 million annually beginning in 2023


South Korea nextgen sequencing tests market to grow at 4% CAGR during 2022-2030: GlobalData
Clinical Trials | September 28, 2022

South Korea nextgen sequencing tests market to grow at 4% CAGR during 2022-2030: GlobalData

There is also a growing interest in whole generation sequencing (WGS)