Category Archives: Gene therapy

ALBANY, N.Y., May 13, 2022 /PRNewswire/ --The value of the global particle therapy market stood at US$ 560.3 Mn in 2021. The global market is likely to rise at a CAGR of 8.6% during the forecast period, from 2022 to 2031. The global particle therapy market is anticipated to cross the value of US$ 1.2 Bn by 2031. Minimal risk of treatment-induced illnesses,risein cancer incidences, and increase in number of particle therapy centers throughout the world are projected to be the growth factors for the particle therapy market. Proton therapy's main objective is to enhance radiation therapy by employing proton beams in the treatmentof targeted tumors and radiosurgery.

A surge in the number of trials has demonstrated the positive effects of particle therapy in certain clinical settings, and breakthroughs in image guidance,beam delivery,as well astreatment planning are predicted to facilitatepositive particle therapy market projections during the forecast period. In addition, favorable clinical findings have resulted in a growth in the number of treatment centers globally, with some in the construction orplanningstages.

Companies in the global particle therapy marketare turning difficult cooperation initiatives and ideas into solutions fosteringsocial innovation to improve patient outcomes. Top research and care institutions are collaborating to promote radiotherapy advancements. Technological advancements in cyclotron devices are driving the market.

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Global Particle Therapy Market: Growth Drivers

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Modernization of healthcare in terms of both infrastructure and services have pushed the healthcare industry to new heights, Stay Updated withLatest Healthcare Industry Research Reportsby Transparency Market Research:

Cancer Gene Therapy Market: Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market.

Gene Therapy Market: Increase in competition among the leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs to gain the first mover advantage is expected to boost the growth of the gene therapy market during the forecast period.

CAR T-cell Therapy Market: The CAR T-cell therapy is emerging as a breakthrough innovation in cancer treatment. Clinical trials conducted globally are showing promising results in end stage patients and especially, for patients suffering from Acute Lymphocytic Leukemia. Such findings are translating into revenue opportunities for companies in the CAR T-cell therapy market.

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Particle Therapy Market to Surpass Value of US$ 1.2 Bn by 2031, Observes TMR Study - PR Newswire

Genes can be delivered into a group of cells in a patient's body in two ways. The first, calledin vivo (in VEE-voh), is to inject the vector directly into the patient, aiming to target the affected cells.

The second, calledex vivo (ex VEE-voh), is to deliver the gene to cells that have been removed from the body and are growing in culture. After the gene is delivered, integration and activation are confirmed, and the cells are put back into the patient.

Ex vivo approaches are less likely to trigger an immune response, because no viruses are put into patients. They also allow researchers to make sure the cells are functioning properly before they're put in the patient. Several gene therapy successes useex vivo gene delivery as an alternative to bone marrow transplants.

Bone marrow containsstem cells that give rise to many types of blood cells. Bone marrow transplants are used to treat many genetic disorders, especially those that involve malfunctioning blood cells. Ideally, a "matched" donor, often a relative, donates bone marrow to the patient. The match decreases the chances that the patient's immune system will reject the donor cells. However, it's not always possible to find a match. In these cases, the patient's own bone marrow cells can be removed and the faulty gene corrected with gene therapy. The corrected cells can then be returned to the patient.

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Gene Delivery: Tools of the Trade - University of Utah

References

1. National Institutes of Health. Genetics Home Reference. Help me understand genetics. Accessed May 3, 2021. https://medlineplus.gov/download/genetics/understanding/primer.pdf2. FDA Commissioner. What is gene therapy? How does it work? US Food and Drug Administration. Accessed July 1, 2021. https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work3. Biology Online Dictionary. Monogenic disease. Accessed April 19, 2021. https://www.biologyonline.com/dictionary/monogenic-disease4. Griffiths AJF, Miller JH, Suzuki DT, et al. An Introduction to Genetic Analysis, 7th edition. W.H. Freeman; 2000.5. STAT Reports. The STAT guide to viral vectors, the linchpin of gene therapy. STAT News; 2019.6. Collins M, Thrasher A. Gene therapy: progress and predictions. Proc Biol Sci. 2015;282(1821):770-788.7. Guha TK, Wai A, Hausner G. Programmable genome editing tools and their regulation for efficient genome engineering. Comput Struct Biotechnol J. 2017;15:146-160.38. Koshravi MA, Abbasalipour M, Concordet J-P, et al. Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: a promising approach for gene therapy of beta thalassemia disease. Eur J Pharmacol. 2019;854:398-405.9. Ceasar SA, Rajan V, Prykhozhij SV, Berman J, Ignacimuthu S. Insert, remove or replace: A highly advanced genome editing system using CRISPR/Cas9. Biochimica et Biophysica Acta. 201610. Choi J. Huntingtons Outreach Project for Education, at Stanford: Gene Silencing. Accessed July 1, 2021. https://hopes.stanford.edu/gene-silencing/11. Balasubramanian S, Habegger L, Frankish A, MacArthur DG, et al. Gene inactivation and its implications for annotation in the era of personal genomics. Genes Dev. 2011;25(1):1-10.12. Grant SG, Campbell CE, Duff C, et al. Gene inactivation as a mechanism for the expression of recessive phenotypes. Am J Hum Genet. 1989;45:619-634.13. Ellebrecht CT, Bhoj VG, Nace A, et al. Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science. 2016;353(6295):179-184.14. Chandler RJ, Venditti CP. Gene therapy for metabolic diseases. Transl Sci Rare Dis. 016;1(1):73-89.15. Keeler AM, ElMallah MK, Flotte TR. Gene therapy 2017: progress and future directions. Clin Transl Sci. 2017:10(4):242-248.16. Rees H, Liu D. Base editing: prescision chemistry on the genome and transcriptome of living cells. Nat Rev Genet. 2018;19(12):770-788.17. American Society of Gene & Cell therapy (ASGCT). Gene and cell therapy FAQs. Accessed July 1, 2021. https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs18. NIH National Cancer Institute. NCI dictionary of cancer terms. CAR T-cell therapy. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/car-t-cell-therapy19. Almasbak H, Aarvak T, Vemuri MC. CAR T cell therapy: a game changer in cancer treatment. J Immunol Res. 2016;2016:5474602.20. National Cancer Institute. NCI dictionary of cancer terms: CRISPR-Cas9. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/crispr-cas921. Encyclopedia Britannica. Gene editing. Accessed July 1, 2021. https://www.britanica.com/science/gene-editing

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How does Gene Therapy Work | Types of Gene Therapy

The Gilbert Family Foundation, a private fund established by Dan and Jennifer Gilbert, announced Thursday it is funding more than$18 million in grants toward a cure for Type 1 neurofibromatosis.

The money will fund a three-year campaign as part of the organization's Gene Therapy Initiative. Four grants totaling $4.4 million will be invested into further developing research that initially began in 2018, while the additional $13.8 million will be invested into 12 new research projects.

Commonly referred to as NF, neurofibromatosis is acondition that causes tumors to form on nerves anywhere in the body. Type 1 usually appears in childhood, while types 2 and 3 appear in early adulthood.

The Gilberts have been passionate advocates in the fight to end NF since their oldest son, Nick, was born with NF1. Diagnosed at 15-months, Nick has suffered through multiple brain surgeries, loss of vision and rounds of chemotherapy. In 2018, he underwent an eight-hour operation and spent more than a month in recovery.The disease can be inherited, but none of the Gilbert's other four children have been diagnosed.

Jennifer Gilbert, who co-foundedthe Gilbert Family Foundation in 2015 to increase NF research, said they are committed to finding a cure for the disease. Since then, the family has committed $72.5 million to curing NF1.

"NF1 affects 1 in 3,000 people throughout the world, including our son Nick. The progress from our last research cycle and the promise of this one continues to give us hope that anyone enduring NF1 will see a cure in our lifetime," Jennifer Gilbert said in a statement.

The foundation launched its Gene Therapy Initiative in December 2018 to fund research focused on developing therapies that address the underlying cause of NF1.Historically, NF1 drug development addressed only the symptoms caused by the disease, demonstrating the need for organizations like the Gilbert Family Foundation, Childrens Tumor Foundation and others who are working toward a cure.

The Gene Therapy Initiative is researching to develop various types of therapies, including gene replacement, gene editing, RNA editing, exon skippingand nonsense mutation suppression, officials said.GTI is also invested in developing methods to improve the accuracy and efficiency of delivering NF1 gene therapies to their intended parts of the body.

Type 1 NF can cause bone deformities, learning disabilities and high blood pressure. Type 2 can cause hearing loss, vision loss, and difficulty with balance. Type 3 can cause chronic pain throughout the body.

Their lead Ph.D. researchers includeCharles Gersbach of Duke University, Robert Kestertonofthe University of Alabama at Birmingham, Casey Maguire ofMassachusetts General Hospital and Miguel Sena-Esteves ofthe University of Massachusetts.

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Gilbert Foundation invests $18M toward Gene Therapy Initiative - Detroit News

Helixmith said it has signed a three-party memorandum of understanding with Cartexell and GI Cell for joint research on cell gene therapy products.

The trilateral MOU will encourage cooperation between the three companies to identify candidate materials for cell gene therapy and cooperate in manufacturing and commercializing clinical trial drugs.

Accordingly, the three companies will utilize Helixmith's Cell & Gene Therapy Center's technology and one-stop service for cell gene therapy development and production, Cartexell's CAR-T (Chimeric Antigen Receptor) cell therapy technology, and GI Cell's immune cell culture and freezing system.

"The company expects this MOU to show differentiated competitiveness in the cell gene therapy business," Helixmith CEO Yoo Seung-shin said. "In the future, we will leap forward as a global gene therapy company representing Korea in the global market through various agreements with excellent companies."

Cartexell CEO Seo Jae-hee also said, "Through the convergence of Heliximith and Cartexell's advanced CAR gene engineering and delivery technology with GI Cell's differentiated immune cell culture technology, we expect to secure a CAR-NK cell therapy pipeline early."

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Helixmith, Cartexell, GI Cell to jointly research cell gene therapy - KBR

TESSA technology is a novel process for transfection-free, scalable manufacture of adeno-associated viruses(AAV) and represents a major advancement in AAV manufacturing by improving AAVyields and particle quality. TESSA technology produces significantly more AAV than plasmid-based manufacturing, generating enough material to address the growing demand in healthcare needs for patients suffering from cancer and other life-threatening diseases.

As part of this collaboration, WuXi ATU will supply the materials, equipment, funding, and training required to further develop and commercialiseTESSA technology in Singapore.BTI will contribute research expertise, facilities and access to its network of partnersto support WuXi ATU in its research and development in cell and gene therapy in Singapore.

"We are delighted to launch this collaboration with Singapore's renowned Bioprocessing Technology Institute," said David Chang, CEO of WuXi Advanced Therapies. "We hope that working together on the development and commercialisationof TESSA technology will be the first step in a long-term partnership."

Dr Koh Boon Tong, Executive Director of A*STAR's BioprocessingTechnology Institute added, "With BTI'sextensive experience in bioprocessingscience and engineering, we areexcited to collaborate with WuXi ATU in the area of advanced cell and gene therapy and further R&D together. This is a significant step towards Singapore's goal to be an innovative biotherapeutics bioprocessing R&D hub."

About WuXi Advanced Therapies (WuXi ATU)

As the advanced therapies business unit of WuXi AppTec, WuXi Advanced Therapies is a Contract Testing, Development and Manufacturing Organization (CTDMO) that offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies. Our services and solutions accelerate time to market and support customer programs around the world. For more information, please visit https://www.advancedtherapies.com

About WuXi AppTec

As a global company with operations across Asia, Europe, and North America, WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable global pharmaceutical and healthcare industry to advance discoveries and deliver groundbreaking treatments to patients. Through its unique business models, WuXi AppTec's integrated, end-to-end services include chemistry drug CRDMO (Contract Research, Development and Manufacturing Organization), biology discovery, preclinical testing and clinical research services, cell and gene therapies CTDMO (Contract Testing, Development and Manufacturing Organization), helping customers improve the productivity of advancing healthcare products through cost-effective and efficient solutions. WuXi AppTec received AA ESG rating from MSCI in 2021 and its open-access platform is enabling more than 5,800 collaborators from over 30 countries to improve the health of those in need and to realize the vision that "every drug can be made and every disease can be treated."Please visit: http://www.wuxiapptec.com

About A*STAR's Bioprocessing Technology Institute

Bioprocessing Technology Institute (BTI) is a research institute under A*STAR. Established in 1990, BTI is positioned as Singapore's pillar of research and development for the biomanufacturing sector. BTI's core capabilities span across the bioprocessing value chain, largely comprising Product Innovation, Cell Line Development, Media Development, Downstream Processing, Process Development and Scale-up, and Analytical Science & Technologies. Through strategic partnerships and application-driven research, BTI seeks to create value and impact in product markets including biologics, cell and gene therapy, exosomes, vaccines, engineered tissues, process analytical technologies and cell culture systems. For more information on BTI, visit http://www.a-star.edu.sg/bti.

About the Agency for Science, Technology and Research (A*STAR)

The Agency for Science, Technology and Research (A*STAR) is Singapore's lead public sector R&D agency. Through open innovation, we collaborate with our partners in both the public and private sectors to benefit the economy and society. As a Science and Technology Organisation, A*STAR bridges the gap between academia and industry. Our research creates economic growth and jobs for Singapore, and enhances lives by improving societal outcomes in healthcare, urban living, and sustainability. A*STAR plays a key role in nurturing scientific talent and leaders for the wider research community and industry. A*STAR's R&D activities span biomedical sciences to physical sciences and engineering, with research entities primarily located in Biopolis and Fusionopolis. For ongoing news, visit http://www.a-star.edu.sg.

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Davy WuExecutive Director, Brand & Corporate CommunicationsWuXi AppTecTel: +86-21 2066 3111Email: [emailprotected]

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WuXi Advanced Therapies and A*STAR announce partnership to advance cell and gene therapy in the Asia-Pacific region - PR Newswire

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