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The genes in your bodys cells play an important role in your health indeed, a defective gene or genes can make you sick.

Recognizing this, scientists have been working for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure or prevent a disease or medical condition.

Now this research on gene therapy is finally paying off. Since August 2017, the U.S. Food and Drug Administration has approved three gene therapy products, the first of their kind.

Two of them reprogram a patients own cells to attack a deadly cancer, and the most recent approved product targets a disease caused by mutations in a specific gene.

What is the relationship between cells and genes?f

Cells are the basic building blocks of all living things; the human body is composed of trillions of them. Within our cells there are thousands of genes that provide the information for the production of specific proteins and enzymes that make muscles, bones, and blood, which in turn support most of our bodys functions, such as digestion, making energy, and growing.

Sometimes the whole or part of a gene is defective or missing from birth, or a gene can change or mutate during adult life. Any of these variations can disrupt how proteins are made, which can contribute to health problems or diseases.

In gene therapy, scientists can do one of several things depending on the problem that is present. They can replace a gene that causes a medical problem with one that doesnt, add genes to help the body to fight or treat disease, or turn off genes that are causing problems.

In order to insert new genes directly into cells, scientists use a vehicle called a vector which is genetically engineered to deliver the gene.

Viruses, for example, have a natural ability to deliver genetic material into cells, and therefore, can be used as vectors. Before a virus can be used to carry therapeutic genes into human cells, however, it is modified to remove its ability to cause an infectious disease.

Gene therapy can be used to modify cells inside or outside the body. When its done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.

In gene therapy that is used to modify cells outside of the body, blood, bone marrow, or another tissue can be taken from a patient, and specific types of cells can be separated out in the lab. The vector containing the desired gene is introduced into these cells. The cells are left, to multiply in the laboratory, and are then injected back into the patient, where they continue to multiply and eventually produce the desired effect.

Before a company can market a gene therapy product for use in humans, the gene therapy product has to be tested for safety and effectiveness so that FDA scientists can consider whether the risks of the therapy are acceptable in light of the benefits.

Gene therapy holds the promise to transform medicine and create options for patients who are living with difficult, and even incurable, diseases. As scientists continue to make great strides in this therapy, FDA is committed to helping speed up development by prompt review of groundbreaking treatments that have the potential to save lives.

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What Is Gene Therapy? How Does It Work? | FDA

Ever since the dawn of mankind, diseases have plagued humans over the ages. Years of innovations and advancements in science has provided us with a deeper understanding of how diseases work. This has led to lower mortality rates and longer lifespans. But there are some diseases that just cannot be cured using traditional medicine or surgery. Gene therapy is an experimental technique that caters to patients with such diseases.

Gene therapy is a technique which involves the replacement of defective genes with healthy ones in order to treat genetic disorders. It is an artificial method that introduces DNA into the cells of the human body. The first gene therapy was successfully accomplished in the year 1989.

The simple process of gene therapy is shown in the figure below:

In the figure, the cell with the defective gene is injected with a normal gene which helps in the normal functioning of the cell. This technique is employed mainly to fight against the diseases in the human body and also to treat genetic disorders. The damaged proteins are replaced in the cell by the insertion of DNA into that cell. Generally, improper protein production in the cell leads to diseases. These diseases are treated using a gene therapy technique. For example, cancer cells contain faulty cells which are different from the normal cells and have defective proteins. Hence, if these proteins are not replaced, this disease would prove to be fatal.

Basically, there are two types of gene therapy

This type usually occurs in the somatic cells of human body. This is related to a single person and the only person who has the damaged cells will be replaced with healthy cells. In this method, therapeutic genes are transferred into the somatic cells or the stem cells of the human body. This technique is considered as the best and safest method of gene therapy.

It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes which are passed from the parents to their children. The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms. Germline gene therapy is not legal in many places as the risks outweigh the rewards.

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Gene Therapy - Discover How It Works Its Types And ...

Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease. For example, scientists must find better ways to deliver genes and target them to particular cells. They must also ensure that new genes are precisely controlled by the body.

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How does gene therapy work?: MedlinePlus Genetics

Marianne De Backer,Head of Business Development & Licensing in Bayers Pharmaceuticals Division, pictured above. Photo courtesy of Bayer.

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies.

The launch of the C> Platform is supported by a number of collaborations and acquisitions the Germany-based company made over the past year, including the October acquisition of AskBio's AAV-based gene therapy pipeline, as well as its 2019 deal for BlueRock Therapeutics and that companys induced pluripotent stem cells (iPSC) platform.

Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors.While these are three companies considered key to the future of Bayers C> Platform, the company is not done fleshing out this new area. Marianne De Backer, Head of Business Development & Licensing in Bayers Pharmaceuticals Division, said the core pieces of the platform are in place, but noted there are other areas left to explore in the rapidly evolving space.

In an interview with BioSpace, De Backer outlined Bayers thought process for opening its purse strings and diving into the deep end of the cell and gene therapy space.

The roots of Bayers platform began in 2014, when the company stepped into the gene therapy space through a collaboration for hemophilia A with Dimension Therapeutics, which was later acquired by Ultragenyx in 2017. Although that never panned out as hoped, Bayer still saw significant potential in cell and gene therapy. De Backer said last year, the company decided it was time to heavily invest in this area.

We analyzed where we wanted to play to become a leader in the field, De Backer said from Germany. There is an incredible unmet need in some diseases that can only be addressed through cell and gene therapies.

As a result, the company settled on four areas of focus iPSC, allogenic CAR-T, genome mutations and gene editing. In order to make a big impact in these areas, De Backer said it was important to find pioneers in the field and bring them into the fold. The company flexed its M&A muscle and has done precisely that with its three announced partnerships. Each of the companies met strict criteria that includes assets in clinical development, an industry-leading platform and in-house manufacturing capabilities.

We have set the bar high. We wanted to come up with a deal that had pioneers in the field, De Backer said.

She added she was incredibly happy to secure deals with these companies, noting that the core pieces are in place for the C> Platform. However, she said the company wants to strengthen its position in gene editing.

The platform is already bearing fruit. Last week, BlueRock announced the U.S. Food and Drug Administration cleared an Investigational New Drug application for a Phase I study of pluripotent stem cell-derived dopaminergic neurons in advanced Parkinsons disease.

Although Bayer has been and will continue to seek out companies to acquire and forge strategic partnerships with, De Backer said part of the companys strategy is to keep the companies at arms length, rather than fold them into Bayers own operations.

We want them to focus on their science and make sure they keep their entrepreneurial culture, De Backer said, describing the company as a docking station for its subsidiaries and partners.

Collaborative agreements will be a key for the companys strategy in this space. De Backer said the past 10 months of the global pandemic has shown how important a cooperative landscape is to the development of promising new treatments. She touted the speed of vaccine development this past year and said that type of laser-like focus has proven drug development is prime for this kind of disruption.

The silver lining of this pandemic has shown that we can do things differently, De Backer said.

In addition to cell and gene therapy, De Backer said digital technology will also be a key focus for Bayer. Machine learning and artificial intelligence will bolster drug design programs and help spur that kind of innovation in drug discovery De Backer anticipates. An ongoing partnership with Recursion Pharmaceuticals is expected to lead to new development for fibrotic disease treatments.

De Backer said Recursions purpose-built artificial intelligence-guided drug discovery platform is the kind of tool Bayer hopes to harness to benefit patients across multiple indications. Despite all of the cutting-edge technologies now at in its arsenal, De Backer said the patients remain the companys priority. It is the patient who they ultimately serve, she said.

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Bayer Continues Eying Potential Partnerships in Cell and Gene Therapy Space - BioSpace

Special delivery: Stem cells can be modified to produce a therapeutic protein in the brain.

Laguna Design / Science Photo Library

Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.

Researchers presented the work virtually on Monday and Tuesday at the 2021 Society for Neuroscience Global Connectome.

One of the biggest hurdles for targeted gene therapy is getting enough treatment to the right spot. In the first study, researchers overcame this obstacle by developing stem cells that produce a therapeutic protein inside the brain.

The team is using the approach to develop a treatment for Angelman syndrome, which is caused by mutation in or deletion of the maternal copy of the gene UBE3A. Because the paternal copy of the gene is typically silent, loss of the maternal copy results in an absence of UBE3A protein. People with Angelman syndrome usually have intellectual disability and motor impairments, and many are autistic.

The researchers had previously used modified stem cells to produce a protein that can activate the paternal copy of UBE3A. Transplanting the cells into the brains of Angelman syndrome model mice boosts levels of UBE3A protein, they found. However, the treatment required multiple direct injections into the animals brains.

In the new work, they instead tried injecting the cells into a pocket of cerebrospinal fluid at the base of the skull an approach that is less invasive and can be performed multiple times. They compared the results with direct injection into the animals hippocampus. In both cases, the mice had UBE3A expression in the brain for up to three weeks.

Mice that received direct injection of the stem cells had fewer Angelman syndrome traits than controls, as measured by their motor skills.

This suggests that though the new route is effective, it may not provide a high enough dosage, says Peter Deng, a postdoctoral researcher inKyle Finkslab at the University of California, Davis, who presented the work. And because the transplanted cells produce protein for only a limited period of time, the effects are temporary a limitation the team is addressing.

Deng and his colleagues also found that monkeys treated with the stem cells had the therapeutic protein throughout their brain and spinal cord three weeks after injection, which suggests the approach has potential for treating people.

The second approach presented at the conference improves the delivery of a more permanent form of gene therapy that uses adeno-associated viruses (AAVs).

Researchers typically inject these viruses directly into the brain, and the viruses usually only affect cells immediately surrounding the injection site.

Youre required to use a ton of the virus to penetrate the whole brain, says Jerzy Szablowski, assistant professor of neuroengineering at Rice University in Houston, Texas, who presented the work.

One potential workaround is to inject the AAV into the blood and use focused ultrasound to temporarily open up the blood-brain barrier, allowing the AAV to cross into the brain. Sometimes with this approach, however, the virus also inserts itself into other organs.

In their new work, the team developed AAVs that more easily cross the blood-brain barrier and more selectively target neurons than previous versions do. As a result, the new AAVs can be given in lower doses, reducing the amount of tissue affected outside the brain, Szablowski says.

To identify the most efficient AAV, Szablowski and his colleagues designed 2,100 new viruses, injected them all into the bloodstream of mice and applied focused ultrasound to the animals skulls. The mice had been engineered so that AAVs that successfully inserted themselves into a neuron got tagged with a marker. The team performed genomic sequencing on the mouse brains a few weeks later and read out the levels of viruses.

Compared with the previously most effective AAV, the top five newly identified AAVs targeted twice as many cells in the brain (including more neurons), and nearly half as many cells outside the brain, the researchers found.

The approach could be used to more efficiently deliver treatments for conditions such as Angelman syndrome or Parkinsons disease, the team says.

Read more reports from the 2021 Society for Neuroscience Global Connectome.

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New gene therapy methods deliver promise - Spectrum

An innovative public-private partnership took a big step in its plan to open a center next year that aims at boosting advances in cell and gene therapy in the region, signing a 10-year lease for a 40,000-square-foot facility in Watertown.

Project participants refer to the facility, which has not been formally named, as the center for advanced biological innovation and manufacturing (CABIM). The goal is to increase availability of materials like genetically altered cells that are essential to advancing discoveries from the lab to clinics for use in treating patients.

There has been great progress in developing pharmaceuticals small-molecule drugs to treat a wide range of diseases, said Harvard Provost Alan Garber, who has led the effort. But many conditions resist treatment with conventional pharmaceuticals. Cell-based therapies offer biological approaches that are complementary to and sometimes far more effective than chemistry-based treatments.

Scientists say that a bottleneck in manufacturing such biological materials is slowing the development of cutting-edge advances in gene therapy, stem cell science, regenerative medicine, CRISPR/Cas9 gene editing, and cancer immunotherapy. An array of treatments based on those and similar technologies such as those involving RNA, peptides, and oligonucleotides are in development, in clinical trials, and in some cases already in the clinic.

This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

Alan Garber, Harvard provost

The center, whose creation was announced in late 2019, is led by institutions from both academia and industry. It will contain both manufacturing and innovation space to boost the supply of materials for late-stage research and early clinical trials and provide space to develop ideas that have left the lab but are not yet ripe for corporate investment. It will also emphasize training in the operation of advanced equipment used in cell manufacturing as a way to increase the pool of workers with such critical skills in the region.

The promise of cell-based therapies has been proven, Garber said, pointing to recent gene-therapy trials to treat sickle cell anemia, which showed significant improvement. He also cited stem-cell-based work to treat diabetes by implanting insulin-producing beta cells, developed in the Harvard lab of Xander University Professor Douglas Melton.

The development of tools like CRISPR and progress in stem-cell science are among the advances that have given us hope that we will soon be able to treat cancer, immunological diseases, neurological conditions, and many other inherited conditions far better, Garber said. This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

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Center for cell and gene therapy to open next year - Harvard Gazette