Gene therapy - Articles - Page 25

Immune

City of Hope receives CIRM funding to support cell and gene therapy clinical trials

Aug. 10, 2023

Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia (AML) and severe aplastic anemia.

Regeneron brings hearing loss gene therapy programs in-house in $213M Decibel buy

Aug. 9, 2023

By Jennifer Boggs

Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.

Immuno-oncology

INT-2104, newly engineered lentiviral vector platform to treat B-cell malignancies

Aug. 3, 2023

Researchers from Interius Biotherapeutics Inc. presented the development and preclinical evaluation of a novel gene therapy candidate, INT-2104, as potential candidate for the treatment of B-cell malignancies.

Drug Design, Drug Delivery & Technologies

Alexion, Astrazeneca Rare Disease agrees to acquire Pfizer’s preclinical rare disease gene therapy portfolio

July 31, 2023

Alexion, Astrazeneca Rare Disease, part of Astrazeneca plc, has entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc.

Newco news

EG 427 advances toward clinic with HSV-based gene therapy for overactive bladder

July 13, 2023

By Nuala Moran

EG 427 SAS has added a further a further €5 million (US$5.6 million) to its series A, closing the round at €18 million and setting the stage for the first clinical trial of a gene therapy in the treatment of a chronic disorder.

Urology

EG 427 announces final series A closing to fund gene therapy to treat neurogenic bladder overactivity

July 13, 2023

EG 427 has announced the final closing of a series A financing, bringing the total raised in the...

Newco news

Look sharp: Tenpoint Therapeutics closes $70M series A round to pursue vision

July 12, 2023

By Cormac Sheridan

Tenpoint Therapeutics Ltd. raised $70 million in a series A funding round to pursue ambitious plans to reverse vision loss using both ex vivo cell engineering and in vivo cell reprogramming approaches.

Neurology/Psychiatric

Regenxbio announces new exon skipping program for Duchenne muscular dystrophy

July 12, 2023

Regenxbio Inc. has announced it is developing a...

Illustration of ear next to DNA double helix

Ear, Nose & Throat

Sensorion submits CTA in UK for OTOF-GF for otoferlin gene-mediated hearing loss

July 10, 2023

Sensorion SA has submitted a clinical trial application (CTA) for OTOF-GT to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA). Sensorion plans to submit the CTA in Europe in the coming weeks.

Illustration of the inside of an eye with macular degeneration

Ocular

Skyline cleared to advance SKG-0106 gene therapy for wet AMD

July 3, 2023

Skyline Therapeutics (Shanghai) Co. Ltd. has received FDA clearance for its IND application for a phase I/IIa trial of SKG-0106, a one-time intravitreally delivered recombinant adeno-associated virus (AAV) gene therapy for the treatment of neovascular age-related macular degeneration (wet AMD). A global phase I trial will be initiated soon.

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City of Hope receives CIRM funding to support cell and gene therapy clinical trials

Regeneron brings hearing loss gene therapy programs in-house in $213M Decibel buy

INT-2104, newly engineered lentiviral vector platform to treat B-cell malignancies

Alexion, Astrazeneca Rare Disease agrees to acquire Pfizer’s preclinical rare disease gene therapy portfolio

EG 427 advances toward clinic with HSV-based gene therapy for overactive bladder

EG 427 announces final series A closing to fund gene therapy to treat neurogenic bladder overactivity

Look sharp: Tenpoint Therapeutics closes $70M series A round to pursue vision

Regenxbio announces new exon skipping program for Duchenne muscular dystrophy

Sensorion submits CTA in UK for OTOF-GF for otoferlin gene-mediated hearing loss

Skyline cleared to advance SKG-0106 gene therapy for wet AMD

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Today's news in brief

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