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A New Hope for Sickle Cell Patients: Groundbreaking Gene Therapy Under FDA Review

Unlike current treatments that include drugs, blood transfusions, and bone marrow transplants, exa-cel aims to provide a one-time, DNA-altering solution. By modifying ...
Nov 03

Immuneel Therapeutics, a biotech startup, raises $15 million in a Series A round

The biotech sector in the country has been seeing a lot of activity, and the government is also promoting startups in the industry. Immuneel Therapeutics ...
Jun 23

Novartis acquires a gene-therapy company Gyroscope Therapeutics

Swiss healthcare giant Novartis has agreed to pay $1.5 billion to acquire British biotech company Gyroscope Therapeutics. ...
Dec 24

Grace Science receives U.S. FDA orphan drug designation for GS-100, an AAV9 Gene Therapy for NGLY1 deficiency

Latest FDA drug approval update...
Jun 24

Amryt Pharma received orphan drug designation by the US FDA for AP103

AP103 is based on Amryt's gene-therapy platform technology and offers a potential treatment for patients with DEB...
Dec 24

Thermo Fisher scientific expands clinical supply chain services in Europe

Today’s clinical supply chain and logistics market in Europe is expected to double by 2025 and more than triple by 2030...
Dec 21

Eli Lilly announces agreement to acquire Prevail Therapeutics

The acquisition will establish a new modality for drug discovery and development at Lilly ...
Dec 15

PFIZER AND SANGAMO ANNOUNCE UPDATED PHASE 1/2 RESULTS FOR HEMOPHILIA A GENE THERAPY

We continue to be encouraged by the findings from this Phase 1/2 study, which now include durable factor VIII expression through one year of ...
Dec 07

Shape Therapeutics Unveils AAVidâ„¢ Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery Challenge in Gene Therapy

Shape Therapeutics, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate ...
Dec 04

Orchard Therapeutics announces US FDA clearance of IND application for OTL-200 for metachromatic leukodystrophy

The company believes that OTL-200 offers tremendous potential to transform the lives of many young patients with MLD...
Nov 21

AskBio and Selecta Biosciences receive orphan drug designation for MMA-101 to treat methylmalonic acidemia

AskBio and Selecta expect to initiate a Phase 1 clinical trial of MMA-101 and ImmTOR for patients with MMA in the first half of ...
Nov 21

Passage Bio’s PBGM01 receives Orphan Drug Designation from EMA for treatment of GM1 Gangliosidosis

...
Oct 27

PFIZER RECEIVES FDA FAST TRACK DESIGNATION FOR DUCHENNE MUSCULAR DYSTROPHY

Pfizer Inc. today announced that its investigational gene therapy candidate being developed to treat Duchenne muscular dystrophy ...
Oct 01

City of Hope collaborates with Chimeric to develop cell therapy

City of Hope enters licensing agreement with Chimeric Therapeutics Limited to develop its pioneering chlorotoxin CAR T Cell therapy ...
Sep 22

AveXis renamed Novartis Gene Therapies

Previously acquired AveXis renamed as Novartis Gene Therapies...
Sep 04

Catalent signs manufacturing agreement with AstraZeneca for COVID-19 Vaccine AZD1222

Catalent and Astrazeneca partners for the Covid vaccine manufacturing...
Aug 26

Merck Boosts Commercial Viral Vector and Gene Therapy Manufacturing Capacity

New, €100 million facility will more than double existing production capacity. Supports large-scale commercial manufacturing of viral and gene therapy products...
Apr 22

USA starts Gene Therapy Clinical Trial That Aims to Reverse 20 Years of Aging in Humans

This is the world's first IRB-approved clinical trial aimed at reversing aging by at least 20 years; it is also the world's ...
Dec 30

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