Tags : Gene Therapy

Kite’s Tecartus (brexucabtagene autoleucel) Receives the US FDA’s Approval as

Shots: The accelerated approval follows FDA’s PR and BT designation and is based on ZUMA-2 study assessing Tecartus (formerly KTE-X19) in 74 patients with r/r MCL prior treated with  anthracycline/ bendamustine-containing CT, an anti-CD20 Ab therapy and a BTK inhibitor (ibrutinib or acalabrutinib) Results: 87% patients responded to Tecartus (single infusion), including 62 % patients […]Read More

Roche Signs a License Agreement with Sarepta for its Gene

Shots: Sarepta to receive $1.15B up front including $750M in cash $400M in Sarepta stock, priced at $158.59/share of common stock, up to $1.7B regulatory and sales milestones plus royalties on sales. Sarepta will be responsible for SRP-9001’s clinical development and manufacturing with global clinical cost equally shared Roche to get exclusive rights to commercialize […]Read More

Ferring and Blackstone Collaborate to Establish Joint Venture Focusing on

Shots: Ferring launches a new company FerGene focusing the global development and commercialization of Nadofaragene firadenovec in the US for the patients with Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer Blackstone to invest $400M and its expertise to accelerate the future development and commercialization of Nadofaragene firadenovec while Ferring will invest up to $170M […]Read More

Janssen(J&J) Signs an Exclusive Worldwide License Agreement with MeiraGTx for

Shots: Janssen to get exclusive WW license rights to develop & commercialize certain clinical candidates in MeiraGTx’s inherited retinal disease pipeline. Janssen also has an option to license the portfolio of products for gene targeting in inherited retinal disease, will be developed in collaboration with MeiraGTx The focus of the collaboration is to develop adeno-associated […]Read More

Axovant Sciences Licenses Two Gene Therapy Programs from University of

Shots: Axovant acquires WW development and commercialization rights for AXO-AAV-GM1 and AXO-AAV-GM2 Program and will make payments to University of Massachusetts The focus of the agreement is to develop functional copies of HEXA, HEXB & GLB1 genes encoding the certain enzymes to treat Gm1 Gangliosidosis, Tay-Sachs and Sandhoff Diseases AXO-AAV-GM1 program is initiated for GLB1 gene using AAV vector for […]Read More

Mustang Bio Licenses Gene Therapy from St. Jude Children Hospital

Shots: St. Jude Children’s Research Hospital out-licenses exclusive worldwide rights for Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency also known as “bubble boy” This therapy is presently being assessed in a phase I/II multicenter study in infants (Age ≥2)  In Interim Analysis, 6/8 patients achieved reconstituted immune system within 3-4 mos of treatment, in […]Read More

Novartis to Acquire AveXis with its Gene Therapy Clinical Candidates

Shots: Novartis acquires AveXis, in all stock transaction with its AVXS-101 candidate for replacing the defective SMN1 gene. AveXis to receive $218/share making total deal value $8.7B The focus of the agreement is to utilize AveXis’ AAV9 gene therapy platform to develop therapies for multiple therapeutic areas AVXS-101 is a gene replacement therapy candidate targeted […]Read More