Week's news analysis

Is it time for the return of gene therapy?

By The Editor - 10 December 2020

The R&D on Covid-19 vaccines and treatments is not the only goal of Big Pharma. Interest in gene therapy development remains very high, as evidenced by several recent deals and agreements.

  • In July, Harvard University's Massachusetts Eye and Ear had entered into an exclusive licensing agreement with Biogen to develop a potential treatment for inherited retinal degeneration (IRDs) due to mutations in the PRPF31 gene, which are among the most common causes for autosomal dominant retinitis pigmentosa. Inherited retinal degenerations are a group of blinding eye diseases caused by mutations in over 270 different genes. Mutations in the PRPF31 gene are the second most common cause of dominant IRD and lead to defects in the function of the retinal pigment epithelial (RPE) cells and photoreceptors of the retina.

  • Beginning of December, Janssen penned a deal to acquire the rights to Hemera Biosciences' investigational gene therapy HMR59, which is designed to help preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration (AMD). According to Janssen, patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the body's natural immune response called complement. In geographic atrophy, which affects around 5 million people worldwide, an overactivity of complement destroys cells in the macula, the central part of the retina. HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, with the theory being it can help stop further damage and retain a patient's vision. a Phase I study of HMR59 for patients with geographic atrophy is complete, while a second early-stage trial evaluating it in patients with wet AMD is currently conducting follow-up visits to determine long-term safety. " Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight", James List, M.D., Ph.D., global therapeutic area head, cardiovascular and metabolism, Janssen R&D, explained.

  • Beginning of December too, Bayer created a cell and gene therapy platform to support its growing pipeline of advanced therapy medicinal products. The german company claims five advanced assets and more than fifteen preclinical prospects in cell and gene therapies. Over the last years, it bought BlueRock Therapeutics, which gave it a foothold in cell therapies through the latter's induced pluripotent stem cell (iPSC) technology. It also acquired adeno-associated virus (AAV) gene therapy player Asklepios BioPharmaceutical. The two companies will both be integrated into the new C platform.

  • A few days later, December 7, Bayer announced another deal in the field of gene therapy again, through a pact focusing on tough-to-treat lung cancers with Atara Biotherapeutics. The company also entered the CAR-T research space and more precisely the area of solid tumors. For some time, CAR-T has been focused heavily on blood cancers, where there have been several amazing successes. The deal with Atara focuses on off-the-shelf T-cell immunotherapy ATA3271 for high mesothelin-expressing tumors, with a focus on more rare and tough-to-treat high mesothelin-expressing tumors such as malignant pleural mesothelioma and non-small cell lung cancer.

In 2019, genomic medicine has surged to the forefront of biotech, with almost 30 biotech M&A deals, up from less than 20 in 2018. " The increase was led by a number of high profile gene and cell therapy acquisitions in both the public and private spaces", according to the American gene technologies.

Since gene therapy has become a new approach to treating a large variety of diseases, several approvals have taken place. Ex-vivo cell therapy treatments for blood cancers Kymriah and Yescarta were approved by US regulatory authorities in fall of 2017. Spark's treatment for a rare type of blindness, Luxturna, also took place in 2017.Last but not least, in Mays 2019, the FDA approved gene therapy treatments with a nod towards AveXis' Zolgensma for spinal muscular atrophy.

2018 Gene Therapy Deals
  • 1/22/18 Celgene- Juno Cellular immunotherapy using autologous CAR T;
  • 4/9/18 Novartis-AveXis AAV9 gene therapy for spinal muscular atrophy, Zolgensma;
  • 8/10/18 Astellas-Quethera Recombinant AAV for use in glaucoma and other ophthalmologic;
  • 9/20/18 Amicus-Celenex 10 clinical & pre-clin AAV programs in neurologic lysosomal storage disorders;
  • 7/19/18 PTC Tx-Agilis Bio AAV Aromatic L-Amino Acid Decarboxylase Deficiency.
2019 Gene Therapy Deals
  • 2/25/19 Roche-Spark Tx AAV gene therapies in blindness, hemophilia, lysosomal storages disorders, neurodegenerative diseases;
  • 2/27/19 Sarepta-Myonexus AAVrh.74 Five gene therapy candidates to treat forms of Limb-Girdle Muscular Dystrophy. 3 clinical, 2 preclin assets;
  • 3/4/19 Biogen-Nightstar AAV clinical stage gene therapy. AAV treatments for retinal disorders choroidemia;
  • 9/12/19 Castle Creek-Fibrocell Autologous LV cell & gene therapy for skin & connective tissue disease. Ph3 for recessive dystrophic epidermolysis bullosa;
  • 12/2/19 Astellas-Audentes AAV based genetic medicines. X-linked myotubular myopathy, Pompe and Duchenne muscular dystrophy.
Source: (American Gene Technologies - March 2020)

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