Tumor suppressor genes, or antioncogenes, tell healthy cells:

If tumor suppressor genes get turned off due to mutations, cells are at risk of becoming cancerous and growing uncontrollably.

Most cancer cells have more than 60 mutations. A challenge for researchers is finding which mutations cause certain types of cancer. Mutations in tumor suppressor genes are one of the two main types of mutations that lead to cancerous changes in cells. Mutations to oncogenes, which tell cells when its time to grow, are the second main type.

Many types of tumor suppressor genes have been discovered, and researchers will very likely discover more of these genes in the future. Read on to learn more about the role tumor suppressor genes play in cancer development and treatment.

Every cell in your body contains long strands of tightly coiled DNA called chromosomes that carry your genetic information. You have 23 pairs of chromosomes in all your cells except for your sex cells. These cells only contain one pair of 23 chromosomes.

Your chromosomes contain approximately 20,000 genes that hold instructions for your cells. Certain genes are turned on and off in each cell depending on the cells function. A change or damage to a gen is called a gene mutation.

Two main types of gene mutations are known to lead to the development of cancer:

The University of Texas Tumor Suppressor Gene Database lists 73 tumor suppressor genes that may play a role in cancer development. Its very likely that more genes will be discovered in the future.

Tumor suppressor genes are broadly divided into five categories:

Tumor suppressor gene mutations have been identified in many types of cancer, including:

Mutations in tumor suppressor genes can lead to tumor genesis, or the uncontrolled growth of cells. You have two copies of most genes in your body, one from each of your parents. Research has found that one copy of most tumor suppressor genes is enough to control cell division (a form of replication). This is called the two-hit hypothesis.

Many tumor suppressor genes have been studied, and its likely that many more havent been discovered yet. Some of the more well-known genes include:

Gene mutations can be inherited or acquired.

Most tumor suppressor gene mutations are acquired not inherited. But most genes linked to inherited cancers are tumor suppressor genes. Most oncogene mutations are also acquired.

Researchers are continuing to improve their understanding of why some genes mutate. Mutations in the TP53 gene have been linked to ultraviolet light exposure and tobacco smoke.

Targeted gene therapy is a promising area of cancer research that may allow doctors to deliver more personalized treatment.

Chemotherapy has long been a mainstream cancer treatment, but it often causes debilitating side effects due to damage to healthy cells. In recent years, researchers have been exploring how to use targeted therapies to treat cancer.

Targeted therapies use drugs to target cancer cells while leaving healthy cells mostly undamaged. Targeted gene therapy modifies specific genes in cancer cells.

Currently, nearly all targeted gene therapies target oncogenes and not tumor suppressor genes. Its easier for researchers to develop methods to turn off oncogenes than it is for them to turn on tumor suppressor genes.

Translating cancer research into new treatments is a long process. However, theres been steady progress in developing drugs to target the TP53 gene and some other tumor suppressor genes.

Tumor suppressor genes tell healthy cells when to destroy themselves, slow their growth, or repair DNA. Mutations to these genes can cause cells to become cancerous and multiply out of control.

More than 70 types of tumor suppressor genes may play a role in the development of cancer. Researchers are examining ways to target tumor suppressor genes to treat cancer.

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Tumor Suppressor Genes: Role in Cancer and Cancer Therapy - Healthline

Abeona Therapeutics Inc.

NEW YORK and CLEVELAND, April 29, 2022 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in cell and gene therapy, today announced that preclinical data on AAV204, a novel adeno-associated virus (AAV) capsid from Abeonas in-licensed AIM capsid library, will be presented at the Association for Research and Vision in Ophthalmology (ARVO) Annual Meeting, to be held on May 1-4, 2022 in Denver, CO and virtually on May 11-12, 2022.

The poster presentation will highlight the ability of AAV204 to produce more robust transduction in the macula area of the eye following para-retinal administration in non-human primates, an ocular administration method that does not require a subretinal detachment. The presentation details are as follows:

Posterboard Number: 3170-F0444Title: AAV204, a Novel AAV Capsid, Demonstrates Superior Macular Transduction Following Para-Retinal Administration in Non-human PrimatesPresenter: Brian Kevany Ph.D., Chief Technical Officer and Head of Research at AbeonaPoster Session Date/Times: Tuesday, May 3, 2022, from 5:30-7:30 PM MDT

About the AIM capsid library AIM capsids are novel AAV serotypes that target delivery of genetic payloads to the central nervous system (including the retina), lungs, eye, muscle, liver and other tissues with potentially improved tropism profiles key to enable treatment of a variety of devastating diseases. AIM vectors have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeonas lead clinical program is EB-101, its investigational autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development. The Companys development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeonas novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeonas fully integrated cell and gene therapy cGMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and potential commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

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Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition; continued interest in our rare disease portfolio; our ability to potentially commercialize our EB-101 product candidate; obtaining a strategic partnership to take over development activities for ABO-102; our ability to enroll patients in clinical trials; the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies; the impact of competition; the ability to secure licenses for any technology that may be necessary to potentially commercialize our product candidates; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; reducing our operating expenses and extending our cash runway; our ability to execute our operating plan and achieve important anticipated milestones; and other risks disclosed in the Companys most recent Annual Report on Form 10-K and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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Abeona Therapeutics Announces Presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting - Yahoo Finance

13-year-old Killian McCarty has a rare form of leukodystrophy. He's a constant inspiration to his mom, who is raising awareness to find a treatment and cure.

MERIDIAN, Idaho When Killian McCarty was born thirteen years ago, his parents were elated. He was their first child, and all signs pointed to a healthy, happy baby boy.

"When Killiam was born, it was a typical pregnancy, everything was great and happy," said Tara McCarty, Killian's mom. "But, as time went on, we started noticing some missed milestones."

Killian was diagnosed with cerebral palsy at the age of two, and for years, that's what his mom and dad thought he had.

However, years later, when he continued to regress and his condition worsened, they found out their son was misdiagnosed. It turns out, Killian never had CP.

"The doctor said 'it just doesn't make sense. You have a perfect birth story, you have no events in childhood, you have an abnormal MRI and regression of symptoms, I don't think you are looking at cerebral palsy,'" Tara McCarty said. "Killian is 10 at this time, and I'm thinking what have we been doing for the last 8 years?"

The McCarty's were given a new diagnosis. It was not the news they wanted to hear.

"We discovered it's one of the most rare leukodystrophies, there's only about 200 kids with this in the world," Tara McCarty said.

According to NORD, the National Organization for Rare Disorders, Leukodystrophiesare a group of rare, progressive, metabolic, and genetic diseases that affect the brain, spinal cord, and often the peripheral nerves.

Killian has Tubb4a leukodystrophy, one of about 50 types of leukodystrophies that cause a loss of myelin, the white fatty insulation that coats the nerves in the brain and spinal cord, which allow messages to be sent from the brain to the muscles. It can also cause atrophy of the basal ganglia and/or cerebellum, leading to movement impairments and difficulty talking and swallowing. It is a progressive disease, so sadly kids afflicted will continue to lose function as they get older.

"Leukodystrophy is a degenerative condition, and this is where I get emotional," said Tara McCarty holding back tears. "Things are going to continue to decline, and it is so rare there is no treatment or cure for it. You basically take things as they come. You have to come to terms with that grief."

Killian is working hard to combat the effects of this rare disease. He now uses a wheelchair full-time.

"He does five therapies a week, and we are typically downtown at St Luke's two or three times a month to see a specialist. He's had surgeries, x-rays. He now has a feeding tube, because swallowing is so difficult, and he was losing a lot of weight. Fine motor skills are becoming really hard for him," Tara McCarty said. "It's just hard, it's hard as a parent, and it's hard for him to understand that things may not get better. Hopefully, we can maintain where he is today.

Through all of the therapies and a recent debilitating surgery for scoliosis, Tara says Killian is the one that keeps everyone around him positive.

"He's just this pretty cool kid, he's cracking jokes with the doctors, he has the nurses laughing," Tara said. "Certainly, we have our days where we want to throw in the towel, and pull covers over our head, but it's not an option. It never is."

Despite all of his hurdles, Killian is one of the most active kids you'll ever meet. His mom says if there's a sport out there, he wants to try it!

"I love football, baseball, basketball, hockey... just pretty much any sport," Killian told us. "When I play sports, it's like I'm not in a wheelchair."

If Killian wants to try it, Tara finds a way to make it happen. She has had help from the Challenged Athletes Foundation (CAF) and other adaptive sports organizations. He's tried skiing, hockey, sailing, fishing, skateboarding, hiking, you name it!

"If he's got a passion for it, we need to find ways to figure out how to make it work. We don't do anything small here, we go big or go home, " said Tara.

Killian says he's so grateful for his family, he has a younger sister and brother. His dad is so proud of him, and he says his mom is his biggest fan.

"I love that they are always around me when times get tough," said Killian. "Mom pushes me around, everywhere we go."

Like so many kids, Killian started to watch a lot of YouTube during the pandemic. He loves to watch spicy food challenges! He asked his mom if they could create a hot sauce together. So, Tara went to work!

"So, we did a lot of research on how to make hot sauce at home, and we learned how to ferment vegetables and peppers to make our own hot sauce," Tara told us.

"It's called Killian's Wheely Hot Sauce, and it's wheely hot!" said Killian with a big smile.

Killian loves to help his mom with all the steps that go along with making the sauce, it's one of the many projects they work on together. The family often gives the hot sauce to friends as gifts.

Tara is dedicated to finding a treatment and a cure for leukodystrophy. She has partnered with other moms around the world to raise awareness and money for research. While there is currently no cure for this type of leukodystrophy, significant advancements in research have been made. With awareness and funding, clinical studies and FDA approval could be just a few years away. Treatment in the form of gene therapy could halt the progression and improve the quality of life for kids suffering from Tubb4a leukodystrophy now.

"They have really great research going on, but because of the rareness of this, it's hard to get the funding to get to clinical trials and get to treatment. It's there, medical researchers are so close," said Tara. "I think the big thing for us is advocacy, to find something that can help our kids. Our goal is within Killian's lifetime to come up with a treatment and a cure."

Tara says her son is her hero, and her inspiration to keep pushing forward.

He doesn't give up on anything, he's going to try. If you tell him maybe we better not, or I don't think that you can, you better look out because he's going to prove you wrong.

Killian is well aware of what's ahead of him, but he never stops chasing his dreams. He has a special message for people who are reading this right now.

"Just go do stuff! Live life to the fullest."

For more information on Killian's type of leukodystrophy, and to donate to funding research visit http://www.h-abc.org.

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7's HERO: Meridian boy with rare degenerative disease inspires with his love of sports - KTVB.com

OPINION:

In 2018, former President Donald Trump signed into law a bill that gave terminally ill patients who had exhausted all other therapies and could not enroll in a clinical trial the right to try experimental drugs not yet approved by the U.S. Food and Drug Administration. The bills passage gave thousands of terminally ill Americans the help, hope and fighting chance they could be cured.

Last week, Arizona Gov. Doug Ducey expanded on Mr. Trumps signature bill allowing terminally ill Arizonians the ability to seek individualized treatments something excluded from the original bill. Its solid legislation that should be replicated on the national stage.

The Arizona bill was prompted by the Riley family, whose 1-year-old daughter Olivia was diagnosed with an extremely rare and fatal brain disease that robbed her of the ability to walk and talk within 90 days of her symptom onset. Olivia, now three, is currently in hospice care and is unlikely to survive her childhood.

Sadly, Olivias sister Keira was born with the same rare disease. A newborn at the time of her diagnosis, Keira had not yet experienced any symptoms. Unlike Olivia, Keira had treatment options personalized gene therapy, which could modify her DNA so her body could function normally. The treatment option was not available to her in the U.S. because it hasnt been FDA approved so the Rileys had to raise hundreds of thousands of dollars to move to Italy, where Keira could seek treatment.

Today, Keira is a healthy 2-year-old toddler.

Shes doing things that we never got to see Livvy do, her mother Kendra said, trying to promote her story. Shes running, shes climbing, shes having full conversations with us. Shes enrolled in preschool for the fall. These are really great, everyday things for normal parents, but for us, theyre huge, huge milestones that we didnt get to see with Livvy. So its really, really heartwarming to see that happen for Keira. And its all because of this treatment.

The Rileys, along with the Goldwater Institute, were the key advocates behind Arizonas state law, the Right to Try for Individualized Treatments Reform, and are urging other states in the nation to pass similar bills.

Although Arizonas legislation was passed on a bipartisan basis, Democratic critics of Mr. Trumps 2018 Right to Try legislation said the act gives false hope to patients and has the potential to worsen their condition.

This is absolute nonsense. Every American who is facing death from a rare disease deserves the opportunity to pursue whatever cutting-edge technology is available and shouldnt have to travel overseas to do so.

Every family who has a loved one struggling with terminal illness deserves the right to try even if the therapy fails. Its unconscionable that bureaucrats in Washington or in the state legislature would deny anyone potentially life-saving treatments.

Arizona became the first state in the nation to pass whats being dubbed Right to Try 2.0. Other states should follow suit.

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EDITORIAL: Arizona's 'Right to Try 2.0' - Washington Times

According to the recent GlobalData report Looking Ahead to 2022 The Future of Pharma, sales of biologics are forecast to significantly overtake those of innovative small molecules over the next five years, with biologic sales forecast to be $120bn greater than small molecule sales by 2027. This emphasises the continuing trend seen within the pharmaceutical industry of the continuing rise and dominance of biologics, and the whole sector pivot towards them was highlighted within the GlobalData webinar Trends in New Drug Approvals and Manufacturing.

As shown in Figure 1, nearly all of the subtypes of biologics GlobalData tracks are expected to grow significantly in sales revenue and will account for 55% of all innovative drug sales by 2027. Biologics sale are currently largely driven by monoclonal antibodies such as Ono Pharmaceuticals Opdivo, Regeneron Pharmaceuticals Dupixent and Mercks Keytruda, which are forecast to account for 46% of all biologic sales in 2027. Keytruda alone, which is marketed for oncology indications like non-small cell lung cancer, head and neck cancer and squamous cell carcinoma, is forecast to account for 4% of all biologics sales in 2027.

While monoclonal antibodies are projected to dominate biologic sales in 2027, gene therapies and gene-modified cell therapies are expected to display the greatest growth, with a forecast increase of more than 1,000% for both molecule types between now and 2027. Unlike monoclonal antibodies, however, gene therapy sales are being driven by currently unapproved pipeline therapies such as Rocket Pharmaceuticals RPA-501, which is currently only in Phase I and is indicated for glycogen storage disorders, a rare genetic disorder.

Despite the significant growth and predicted dominance of biologics in the future, small molecule sales are also forecast to continue to grow, with small molecule sales forecast to grow by 49% in 2027. This growth is driven by drugs for the central nervous system therapy area such as Bristol-Myers Squibbs Zeposia, which is indicated for multiple sclerosis and is forecast to become a blockbuster drug by 2027, with sales of more than $2bn.

Biologics have solidified their lead over small molecules and are now primary engines of value creation for large-cap pharma companies. As this trend continues, the increasing dominance of biologics within the sales of large-cap and mega-cap bio/pharma companies will be seen. This will have consequences not only for the sponsors, but also, as discussed in the webinar Trends in New Drug Approvals and Manufacturing, lead to an increase in contract manufacturing outsourcing for cell and gene therapies.

Process Innovation in Vacuum Drying and Vacuum Pumps

Contract Manufacturing and Packaging Services

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Biologic sales forecast to pass innovative small molecule sales in next five years - Pharmaceutical Technology