Gene therapy – media

Janssen buys rights to Hemera’s eye disease gene therapy candidate

3rd December 2020 Johnson & Johnson’s pharmaceuticals division Janssen has purchased rights to acquire an investigational gene therapy asset for geographic atrophy from Hemera Biosciences. Geographic atrophy is an advance and severe form of age-related macular degeneration (AMD) which affects five million people globally. Hemera’s investigational gene therapy, HMR59, is administered as a one-time intravitreal injection to aid vision preservation in patients with geographic atrophy. HMR59 is designed to increase the ability of retina cells to produce a soluble form of CD59, a protein that protects the retina from damage. Patients with wet AMD often have low levels of this protein, which is an essential component of the body’s natural immune response. “Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces,” said James List, global therapeutic area head, cardiovascular & metabolism, Janssen Research & ...
- Source: drugdu
- 310
- December 9, 2020
Gene therapy
Bayer to acquire Asklepios Bio in foray into gene therapy worth up to $4 billion

By Ludwig Burger FRANKFURT (Reuters) – Bayer <BAYGn.DE> agreed to acquire unlisted U.S. biotech firm Asklepios BioPharmaceutical Inc for as much as $4 billion in a bet on gene therapy with the help of modified viruses. Germany’s Bayer will pay $2 billion upfront and up to an additional $2 billion in milestone payments contingent on development achievements, it said on Monday. The North Carolina-based takeover target, also known as AskBio, is trying to use the harmless adeno-associated virus as a delivery device to bring genetic repair kits against a range of diseases into the body.Drugs and farming pesticides maker Bayer needs to upgrade its drug development pipeline amid a weaker outlook for agricultural sales and as it seeks to finalise an $11 billion settlement over claims its Roundup weedkiller causes cancer. Among AskBio’s most advanced projects are early tests on volunteers of prospective treatments against Pompe disease – a ...
- Source: drugdu
- 294
- November 2, 2020
Gene therapy
Hemophilia A Gene Therapy by Spark Therapeutics Needs Phase 3 Trial

People with the bleeding disorder hemophilia A require lifelong treatment, which would include regular infusions of a clotting promoter factor VIII. Treatments could be expensive and time-consuming.
- Source: FierceBiotech
- 838
- August 9, 2018
Clinical Trials Gene therapy hemophilia A
Sarepta Therapeutics and Brammer Bio Partnership to Develop Gene Therapy

Biopharma Sarepta Therapeutics and Brammer Bio enter into a long-term strategic partnership to design and develop new manufacturing facilities at CDMO’s plant in Massachusetts. The new partnership is for the development of the production of its microdystrophin, Duchenne Muscular Dystrophy (DMD) therapy and future gene therapies.
- Source: FiercePharma
- 608
- June 18, 2018
Company Insight DMD Gene therapy
Scientists see positive results from 1st ever gene editing therapy

A second patient has been treated in a historic gene editing study in California, and no major side effects or safety issues have emerged from the first man’s treatment nearly three months ago, doctors revealed Tuesday.
- Source: NYPost
- 505
- February 7, 2018
gene editing Gene therapy New Approvals
Spark Therapeutics Enters into a Licensing and Supply Agreement for Investigational Voretigene Neparvovec Outside the U.S.

Spark Therapeutics (ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into a licensing agreement with Novartis Pharmaceuticals to develop and commercialize investigational voretigene neparvovec outside the U.S.
- Source: finance.yahoo
- 542
- January 26, 2018
Gene therapy Market Views voretigene neparvovec
Is the $850,000 price tag for a blindness-curing gene therapy a sign of future medical costs?

Gene therapies are an undeniably exciting new frontier for medicine. From Kymriah's genetically modified, cancer-hunting immune cells, to Luxturna's vision-restoring healthy gene replacement, these therapies are fundamentally unlike 20th-century medicines which generally require ongoing drug treatment. Gene therapies can be one-off doses that in effect, cure a specific disease.
- Source: New Atlas
- 452
- January 11, 2018
FDA Gene therapy Market Views
Sangamo partners with Pfizer to develop gene therapy for ALS

Sangamo Therapeutics Inc and Pfizer Inc said on Wednesday they would work together to develop a gene therapy to treat ALS
- Source: Reuters
- 506
- January 4, 2018
ALS Company Insight FDA Gene therapy Pfizer

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