The future of cutting-edge gene and cell therapies – case studies and analysis of the market concerning innovative gene and cell therapies in the United States, with the focus on technologies and types of therapy.

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We try to broaden the knowledge necessary to understand the needs of our clients. An example is the analysis of a report concerning the market and novelties of gene and cell therapies in the United States. The purpose of this article is not to describe specific diseases, but for the purposes of legal services, we focus in such analyses on technologies and types of state-of-the-art therapies.

We invite you to read the text containing specific examples, names of therapies, companies, etc. This text is based on the report prepared by the STAT organization, which is a premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. They focus on policy developments in the US, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. The entire report can be reached here:

The future of cutting-edge gene and cell therapies is changing novel approaches and changing the trajectory of treating cancer, lethal genetic diseases and other devastating diseases. But these innovations also come with their own set of challenges, including access issues. The high prices of such therapies, which have entered the market in recent years, and the expected high development costs of others, raise questions about how healthcare systems, payers and patients will pay for treatment.

FDA approves the first personalized cell therapy for patients with: Multiple myeloma.

1) Abecma is approved by the FDA for the treatment of patients with multiple myeloma. The FDA has now approved five patient-specific cell therapies, namely CAR-T. The other four drugs – two from Gilead Sciences and one each from Novartis and Bristol – have been approved to treat various forms of lymphoma – another type of blood cancer. CAR-T targets the BCMA protein. The other four CAR-T treatments are performed the same way, but the T cells are modified to target a protein called CD19.

2) Bristol now joins Gilead as the only drug manufacturer to have two CAR-T therapies on the market. Its first CAR-T, Breyanzi, was approved in February 2021. Breyanzi was invented by Juno Therapeutics, which was also acquired by Celgene, now part of Bristol.

Scientists are turning to machine technology to tackle one of the greatest challenges in gene therapy

1) According to Grimm, machine learning can be used to optimize for any of the factors that make up a successful gene therapy vector. With enough data on the use of synthetic viral vectors in humans, machine learning could also be possible to predict what a patient’s response will be.

2) Two gene therapies have been approved by the FDA: Luxturna, approved in 2017 for the treatment of retinal dystrophy; and Zolgensma, approved in 2019 for spinal muscular atrophy.

Siddhartha Mukherjee establishes his second biotechnology company focused on applying cell therapy to solid tumors

1) Myeloid is changing cell cancer therapies like CAR-T that are already effective for blood cancer and uses a similar idea to treat conditions like glioblastoma, which forms solid tumors. It will use what are known as myeloid cells to check a person’s immune system.

2) Therapeutics have developed new RNA strands that code for receptors that can bind to cancer cells to stimulate bone marrow cells to function.

3) Carisma Therapeutics expects to start a clinical trial for peripheral T-cell lymphoma within the first six months of this year.

Sarept’s gene therapy for Duchenne muscular dystrophy stumbles with a failed study

Sarepta Therapeutics has announced mixed results of its first randomized clinical trial of its gene therapy for Duchenne muscular dystrophy. Currently approved drugs, including two developed and marketed by Sarepta, are capable of producing small amounts of dystrophin, making them only partially effective in slowing the progression of the disease in a group of patients with specific genetic mutations.

A gene therapy pioneer is developing a new approach to reducing the side effects of treatment

Scientist at the University of Pennsylvania and pioneer of gene therapy, James Wilson, developed a new tactic to make these therapies safer for the nervous system. Wilson’s team began looking for ways to help AAV spread more evenly throughout the central nervous system, rather than clustering into DRG cells. In doing so, they discovered that there were two types of microRNAs – called miRNA182 and miRNA183 – that were mainly expressed in DRGs but not in most other forms of neuronal cells. Wilson’s team added coding fragments to a therapeutic gene or transgene that targeted miRNA183 with something like a kill switch.

Startup Spotlight: Coded eyes as gene therapy for Dravet syndrome

The first Encoded gene therapy will target Dravet syndrome – a rare condition that can cause seizures, cognitive deficits, and mobility problems. People with Dravet are also more likely than usual to experience sudden, unexpected death – epilepsy (SUDEP). In the case of Encoded, a virus known as adeno-associated virus or AAV will contain the genetic sequence of a transcription factor that should ultimately increase the amount of normal sodium channels produced in a given cell. Encoded viruses will also contain other sequences that limit these changes to only brain cells that are affected by Dravet syndrome.

New Rare Diseases Gene Therapy Startup Recruits Former Sarepta Director As CEO

1) Three-year gene therapy startup from Florida – Alexander “Bo” Cumbo became the new CEO of the startup. Cumbo was responsible for the marketing of both Sarepty approved drugs, including Exondys 51, the first approved drug for Duchenne muscular dystrophy. He was also involved in Sarepta’s own efforts to develop gene therapies.

2) The gene therapy developed by AavantiBio will use a redesigned virus to deliver healthy copies of the FXN gene to patients. Friedreich’s ataxia is more difficult than some other rare diseases because the gene therapy vector must deliver the replacement gene to the cells of the nervous system and heart muscle cells.

3) AavantiBio continues to work with animals to improve its gene therapy and get the correct dosages before it can apply to the Food and Drug Administration for permission to start clinical trials in humans.

4) Pfizer, Takeda and Voyager Therapeutics are also working on competing gene therapy approaches to Friedreich’s ataxia. All of these programs are still in the animal testing phase.