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5 Steps to Take After Receiving a Chronic Disease Diagnosis

Posted: April 19, 2023 at 5:24 pm

Have you recently been diagnosed with a chronic disease? Coming to terms with your diagnosis isn’t easy, but the best way to move forward is to focus on your next steps. What do you need to do now that you know about your condition? From learning about your illness to making impactful lifestyle changes to manage your symptoms, Immortality Medicine is here to share five important steps to take after receiving a chronic disease diagnosis.

Learn About Your Condition

After learning about your diagnosis, one of the first things you should do is research your condition. You want to become an expert on your illness! The more you know about what’s going on in your body, the better equipped you’ll be to make impactful decisions regarding your treatment and management strategies. It’s a good idea to talk to your doctor or nurse about your condition before turning to the internet. Ask them for trusted sources of medical information where you can read more on your own. There’s a lot of misinformation out there, so make sure you know how to spot it!

Review Your Health Insurance

Your health insurance coverage will save you a lot of money as you pursue treatment, tests, and other healthcare visits going forward. Take some time to review your coverage and see if you need to make any changes to your plan. If you don’t have health insurance because you’re self-employed, start looking into your options. Insurers can no longer deny you coverage due to an existing condition, so you shouldn’t have any trouble finding a suitable health insurance plan that will accommodate your diagnosis. Take a look at the Health Insurance Marketplace or the Freelancers Union to compare plans.

Move into an Accommodating Home

Depending on your condition, you may want to move into a new home that’s more accommodating. If, for example, your chronic condition will have an impact on your mobility, moving into an accessible home will help you maintain your independence. You might also want to move to be closer to a hospital or treatment center or live somewhere more supportive of a health-focused lifestyle.

Just remember that you may need some time to save up a down payment for your new home. The more money you can save, the lower your interest rate and monthly mortgage payment. Learn about the down payment process so you can make a realistic savings plan for your big move.

Create an Exercise Plan

Regular exercise will help you maintain mobility and ward off the negative symptoms of your condition. Everyday Health explains that exercise helps lessen the severity of fibromyalgia and improve the overall quality of life for people with chronic pain. Talk to your doctor for safe exercise recommendations. For example, these foam rolling exercises are great for loosening up tight muscles and improving flexibility, which can reduce pain and improve your range of motion.

Make Healthy Dietary Swaps

Like engaging in regular exercise, making healthy dietary swaps is a great way to manage your condition. The American Diabetes Association explains that making healthy lifestyle changes is often very difficult because people try to change too many things at once or choose changes that are too different from their normal routines. Sticking to smaller changes, like swapping a few foods from your diet, is a great place to start without getting overwhelmed. If you need to lower your cholesterol, for example, try swapping butter for olive oil, eating fish instead of red meat, and snacking on homemade popcorn instead of chips.

Receiving a diagnosis of a chronic illness isn’t fun. But with a solid plan of action, you can learn to live with your condition. Review your health insurance options, learn more about your diagnosis, and consider moving into a more accommodating home. Jumping into action after your diagnosis is a great way to move forward and avoid excessive worry.

Immortality Medicine is a science and medical news website for adults interested in the latest breakthroughs in human longevity. If you have any questions, don’t hesitate to reach out!

Recommendation and review posted by Diane Harrison

Polyplus Launches Innovative Plasmid for AAV Vector Production, Strengthening Its Position in Biologic and Cell … – BSA bureau

Posted: March 27, 2024 at 2:47 am

Expanding AAV Vector Production Capabilities with Off-the-Shelf Plasmid Solutions

Polyplus(part of Sartorius, a distinguished upstream solutions provider for biologic and cell and gene therapy production and a part of the Sartorius group, has announced the introduction of pPLUS AAV-RC2, a groundbreaking RepCap plasmid tailored for the production of adeno-associated virus vector 2 (AAV2). This launch is a significant addition to Polypluss portfolio, aligning with the companys strategic direction to become a leading provider of critical raw materials for AAV production.

The pPLUS AAV-RC2 plasmid, now available off-the-shelf, is designed to streamline the manufacturing process for AAV vectors, offering a ready-to-use solution that enhances both efficiency and reliability in vector production. This initiative complements the recent introduction of pPLUS AAV-Helper in September 2023, and is part of a broader strategy to offer a comprehensive range of RepCap plasmids catering to various AAV serotypes throughout 2024.

Roel Gordjin, Chief Commercial Officer of Polyplus, highlighted the companys vision and progress, stating, In 2020, we made a commitment to evolve into a leading delivery supplier with capabilities across the expanding range of modalities in the market. Since then, our portfolio has steadily expanded for transfection reagents, and now we are making our ambition to become a key supplier of critical raw materials for AAV production clear. Gordjin emphasized the companys proactive approach in understanding and meeting client needs, positioning Polyplus as a go-to supplier for AAV manufacturers.

The manufacturing of pPLUS AAV-RC2 is conducted entirely in-house at the Xpress Biologics GMP compliant facility in Belgium, ensuring the highest standards of quality and supply chain security. This move is indicative of Polypluss commitment to supporting the cell and gene therapy sectors with high-quality, dependable solutions that accelerate the transition from research to commercial-scale production.

With this launch, Polyplus not only reinforces its standing in the biotechnology industry but also underscores its dedication to supporting the advancement of gene therapies and biologics, offering innovative solutions that meet the complex needs of AAV vector production.

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Polyplus Launches Innovative Plasmid for AAV Vector Production, Strengthening Its Position in Biologic and Cell ... - BSA bureau

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Orchard Sets $4.25M US Price for Gene Therapy Lenmeldy on Heels of Approval – BioSpace

Posted: March 27, 2024 at 2:47 am

Pictured: Illustration of a gene therapy showing a syringe injecting a treatment into a DNA strand/iStock,Ilya Lukichev

Orchard Therapeutics on Wednesday revealed details regarding the U.S. commercial launch of the newly approved Lenmeldy (atidarsagene autotemcel), giving the one-time gene therapy for metachromatic leukodystrophy a wholesale acquisition cost of $4.25 million.

To justify Lenmeldys pricing, Orchard touted not just the therapeutic benefits of the gene therapy but also the potential long-term impact treatment may have on overall healthcare utilization, according to Wednesdays announcement. Lenmeldy treatment could also help minimize the productivity loss for caregivers as well as open up more life opportunities for patients, the company contends.

The biotech also noted that it arrived at Lenmeldys price tag after completing a comprehensive health technology assessment (HTA) by the Institute for Clinical and Economic Review (ICER), an independent and non-profit organization that conducts cost-effectiveness analyses of treatments. The ICER had set Lenmeldys health benefit price benchmark at up to $3.94 million.

Bennett Smith, general manager of North America at Orchard, in a statement said that the ICER found Lenmeldy to have the highest value-based price for any treatment it has evaluated to date, adding that the gene therapy has also been recognized by various HTA authorities all over the world.

Metachromatic leukodystrophy (MLD) is a rare and genetic metabolic disorder afflicting one in every 100,000 live births. The disease is caused by a mutation in the arylsulfatase A (ARSA) gene, which in turn leads to the toxic build-up of sulfatides in various organs in the body, such as the brain, liver and kidneys.

Over time, this pathologic accumulation causes organ damage, leading to the hallmark symptoms of MLD like cognitive and behavioral problems. Patients with MLD also eventually lose the ability to move, talk and eat.

To address the underlying genetic cause of MLD, Lenmeldy delivers a functioning copy of the ARSA gene using the patients own stem cells. The FDA approved Lenmeldy on Monday, making it the first-ever authorized gene therapy for early-onset MLD in the U.S.

To further illustrate the value of Lenmeldy, Orchard in its news release on Wednesday said that the gene therapy was able to significantly and strongly improve survival in treated children at six years of age. Most patients who were treated with Lenmeldy were also able to walk without assistance and reach normal language and performance IQ scores at age five.

Touting these data, Orchard CEO Bobby Gaspar in a statement called Lenmeldy truly a paradigm-shifting medicine with the potential to stop or slow the progression of this devastating childhood disease with a single treatment.

Lenmeldys hefty price tag continues the trend of rapidly ballooning wholesale acquisition costs for gene therapies. Vertex Pharmaceuticals and CRISPR Therapeutics priced their sickle cell disease treatment Casgevy (exagamglogene autotemcel) at $2.2 million, while bluebird bio gave its competing therapy Lyfgenia (lovotibeglogene autotemcel) a price tag of $3.1 million.

By comparison, Sareptas Duchenne muscular dystrophy gene therapy Elevidys is priced at $3.2 million.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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Orchard Sets $4.25M US Price for Gene Therapy Lenmeldy on Heels of Approval - BioSpace

Recommendation and review posted by G. Smith

Stem Cell Editing Repairs Severe Immunodeficiency – The Scientist

Posted: March 27, 2024 at 2:47 am

The B and T cells of the adaptive immune system recognize unique features on infectious microbes that enter the body. They accomplish this feat using B-cell and T-cell receptors, which take on various shapes to bind to different antigens on foreign invaders. Recombination activating gene 1 (RAG1) is central to this shapeshifting behavior.1 It shuffles the order of DNA sequences in the genes for these receptors, producing multiple versions of the immune receptors that can bind staggering combinations of antigens. However, some people carry mutations in RAG1 that prevent the enzyme from recombining the DNA sequences that code for these receptors. Without properly functioning receptors, B and T cells fail to develop, leading to severe combined immunodeficiency (SCID), a condition in which even the mildest of infections can prove lethal. In a study published in Science Translational Medicine, researchers developed an efficient method to repair RAG1 genes in immune cell progenitors called hematopoietic stem cells (HSC) taken from SCID patients, and revealed that they could restore immune function in mice.2

When you would like to correct the gene, you have to keep in mind that close to the gene, there are a lot of regulatory elements that are relevant for correct gene expression. -Maria Carmina Castiello, San Raffaele Scientific Institute

Maria Carmina Castiello and Anna Villa, two translational immunologists at the San Raffaele Scientific Institute, set out to overcome some of the challenges with editing the RAG1 gene that researchers previously faced. In the past, scientists have taken healthy, functional HSC and inserted them into SCID-model mice, but they often get destroyed by other types of immune cells that recognize the transplants as foreign.3 Normally, doctors use immunosuppressants like chemotherapy before transplantation to deplete immune cells, but this isnt an option for SCID patients. This disease can be associated with severe organ damage, so the critical conditions of the patients do not allow them to receive chemotherapy, Villa explained.

Castiello and her colleagues took a different approach, modifying a SCID patients own stem cells to express a functional RAG1 gene. While other research groups had successfully added RAG1 to patient HSC, they were unable to properly regulate expression of the gene, and therefore couldnt ensure that the stem cells were safe or would effectively replenish B and T cells.

Introducing the gene into the wrong site in the genome may have partly caused this shortcoming. When you would like to correct the gene, you have to keep in mind that close to the gene, there are a lot of regulatory elements that are relevant for correct gene expression, Castiello said.

Rather than adding a functional copy of RAG1, the researchers decided to modify the existing copy, ensuring that the regulatory networks remained intact. In fact, other researchers succeeded when they took a similar approach to edit RAG2.4

Before Castiello and her team could fix the gene, however, they had to choose their editing strategy. Some researchers use base editing, which modifies single letters in the DNA sequence to correct other genetic disorders of these stem cells, like sickle cell disease and -thalassemia.5

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However, RAG-1mutations can occur at several different sites within the gene, so base editing wouldnt cover every type of mutation. Instead, the research team used the clustered regularly interspaced short palindromic repeats (CRISPR-Cas9) system to cut out a large section of the mutant gene, and then provided cells with the correct DNA sequence using a lentiviral delivery system. Since the correct sequence was nearly identical to the original gene, the cell could swap the sequences unassisted using homology-directed repair (HDR), a built-in DNA repair pathway that fixes double-strand DNA breaks using complementary DNA as a template.

Once Castiello and her colleagues swapped the HSCs old, mutated coding sequence for a fresh one, they had to test whether the gene produced a functional RAG1 protein. They inserted a backwards green fluorescent protein(gfp) gene flanked by sequences that RAG1 recognizes. Promisingly, they found that the edited RAG1 inverted gfp comparably to RAG1 in HSC from healthy donors, thereby switching it to an on state, resulting in a functional gfp gene.

They next had to check that these edited cells could restore immune function in the body. They transplanted these edited human cells into SCID-model mice and found that B and T cells spiked to levels similar to those seen in mice that received HSC from healthy donors.

Whats intriguing from the study is that we dont need to correct all the stem cells. If we manage to correct at least 10 percent of the stem cells, this is going to give us a therapeutic benefit, said Saravanabhavan Thangavel, a geneticist at the Institute of Stem Cell Research and Regenerative Medicine who was not involved with the work. However, he also mentioned, We need to track the HDR-edited cells long term. The researchers need to ensure that the modified cells persist in the bodies of people with SCID so that their newly gained immunity doesnt wane over time. If, by chance, the HDR-edited cells faded away, they may not have a therapeutic benefit, Thangavel added.

Down the line, the team aims to refine their protocol. We are trying to increase the editing efficiency that we achieve, Castiello said. She also wants to optimize delivery of the gene into the cells by comparing different methods. In this study they used lentiviruses to deliver the DNA template to the stem cells, but they plan to test other strategies like using lipid nanoparticle conduits that conceal the DNA template and fuse with the cell membrane to release the DNA into the cell.

The team will also have to test the safety of this gene editing strategy and find a way to scale up production of the edited stem cells, Castiello added. Then they should be able to test their edited cells in people with the hope of eventually treating the variety of conditions caused by RAG1defects. We are really committed to translating our strategy to the clinic, she said.

Continue reading below...

Anna Villa and Maria Carmina Castiello are inventors with two patents involved with editing RAG genes.

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Stem Cell Editing Repairs Severe Immunodeficiency - The Scientist

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Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in … – GlobeNewswire

Posted: March 27, 2024 at 2:47 am

OPGx-LCA5 will advance to the next highest dose in mid-2024 based on positive safety and efficacy data

OPGx-LCA5 is well-tolerated and demonstrated clear signs of biological activity

RESEARCH TRIANGLE PARK, N.C., March 26, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose. Opus anticipates initiating the next cohort mid-2024. There are also future plans to expand the study population to include subjects 13 years or older.

In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose, said principal investigator Tomas S. Aleman, M.D., from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.

LCA5 is a form of an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss.

Based on these early clinical data, were excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5, said Ben Yerxa, Ph.D., chief executive officer of Opus. Wed like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.

For more information on the trial, visit clinicaltrials.gov (NCT05616793).

About OPGx-LCA5 OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in nine patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

About Opus Genetics Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit http://www.opusgenetics.com.

Media Contact: Gina Mangiaracina 6 Degrees gmangiaracina@6degreesPR.com

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Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in ... - GlobeNewswire

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New Gene Therapy for Rare MDL to Cost $4.25 Million – Managed Healthcare Executive

Posted: March 27, 2024 at 2:47 am

Orchard Therapeutics has set the wholesale acquisition cost of its newly approved gene therapy, Lenmeldy (atidarsagene autotemcel), at $4.25 million for the one-time treatment. Company officials in a press release said this price reflects its clinical, economic and societal value.

The FDA had announced the approval of Lenmeldy on Monday. It is the first approved gene therapy to treat children with juvenile metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the bodys metabolic system.

We are confident in the potential long-term clinical outcomes of Lenmeldy and will continue to work with public and private payers to structure outcomes-based and other types of innovative reimbursement models that appropriately balance the needs of patients and families for adequate access, healthcare systems for affordability, as well as support future research and development of treatments for ultra-rare diseases like MLD, Frank Thomas, president and chief operating officer of Orchard Therapeutics, said in a press release.

Orchard Therapeutics has a patient support program for patients and their caregivers who will assist with both private and public insurance to access Lenmeldy. Infants with MLD qualify for government assistance so state Medicaid programs are expected to be part of the insurance mix.

Five treatment centers with specialized expertise in transplant and the treatment of neurometabolic diseases are being activated to administer Lenmeldy.

Company officials said they relied on an analysis by the Institute for Clinical and Economic Review (ICER). But the actual price is above the range that ICER analysts had determined would meet the standards for cost-effectiveness. ICERs analysis determined the health benefit price benchmark (HBPB) for Lenmeldy to be between $2.29 million and $3.94 million.

ICERs assessment used a placeholder price of $2.8 million, and ICER noted that the gene therapy provides the most health benefit for presymptomatic disease. They stressed the need for newborn screening of MLD.

Orchard noted in its press release that it is currently conducting 10 prospective newborn screening studies for MLD with about 275,000 newborns screened to date. The data from these studies provide critical evidence to support applications for universal screening of MLD in the United States.

For infants showing signs of early disease, ICER officials said there is concern that the gene therapy may carry the risk of hastening progression of physical and cognitive decline before stabilization occurs. Because of this, ICER officials said they concluded there was a at least a small net health benefit.

MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene, which results in the accumulation of sulfatides in the brain and other areas of the body. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see.

The safety and effectiveness of Lenmeldy was assessed based on data from 37 children who received Lenmeldy in two single-arm, open-label clinical trials and in an expanded access program. Children who received treatment with Lenmeldy were compared with untreated children.

In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children. All children with pre-symptomatic late infantile MLD who were treated with Lenmeldy were alive at 6 years of age, compared with only 58% of children in the natural history group. At 5 years of age, 71% of treated children were able to walk without assistance. Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

The most common side effects of Lenmeldy are fever and low white blood cell count, mouth sores, respiratory infections, rash, medical line infections, viral infections, fever, gastrointestinal infections and enlarged liver.

This story first appeared on Formulary Watch and has been edited.

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New Gene Therapy for Rare MDL to Cost $4.25 Million - Managed Healthcare Executive

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FDA Approves Third Duchenne Muscular Dystrophy Treatment in Nine Months – BioSpace

Posted: March 27, 2024 at 2:47 am

Pictured: A boy sits in a wheelchair on a beach/iStock, ChiccoDodiFC

A young biotech has its first approved product after the FDA greenlit ITF Therapeutics Duvyzat (givinostat) Thursday as the third new treatment for Duchenne muscular dystrophy since last June. The approval of Duvyzat for patients six years and older was granted to ITFs parent company, Italfarmaco.

Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of Duchenne muscular dystrophy (DMD), according to the FDAs announcement.

Duvyzat adds to list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation, Debra Miller, founder and CEO of CureDuchenne, said in a statement emailed to BioSpace.

Leading up to the decision, Italfarmaco reported final results from the Phase III Epidys trial, which met the primary endpoint of a change in the four-stair climb assessment from baseline to 72 weeks.

Duvyzat also showed favorable outcomes in key secondary measures, according to the company, including on the North Star Ambulatory Assessment (NSAA), a 17-item scale that measures motor function skills. On the NSAA, patients treated with the new drug saw less worsening compared to placebo after 18 months. Patients received a standard-of-care steroid regimen throughout the trial. Duvyzat was generally well tolerated, with no treatment-related severe or serious adverse events.

Craig McDonald, chair of the Department of Physical Medicine & Rehabilitation at UC Davis Health and the studys lead U.S. investigator, told BioSpace he has been quite impressed with the data on Duvyzat. McDonald, who has treated nine patients with the drug, said he has a number of patients who are still ambulatory at 16 or 17 years old. DMD patients on steroid treatment typically lose the ability to walk at around 10 to 13 years of age, McDonald noted.

Duvyzat is a small molecule that acts by inhibiting histone deacetylases (HDACs), a group of enzymes that modulate gene and protein expression in the muscle. Deregulation of HDACs is a major consequence of the lack of dystrophin associated with DMD, Matt Trudeau, head of ITF, told BioSpace in an interview ahead of the approval.

While ITF only incorporated in January 2024, Duvyzat has been in development at Italfarmaco for the past six to eight years, Trudeau said. He added that ITF has the right team in place to get the drug to patients quickly. Were bringing in a team that knows how to turn systems on quickly and move with certain purpose and urgency and passion for the patients that we hope to serve.

The new company has leveraged the rare disease talent epicenter of Boston and Cambridge, Mass., to build its team, which comprises former employees of Biogen, bluebird bio and Genzyme, companies that have proven success in the rare disease space, Trudeau noted.

While Trudeau said ITFs express purpose is to create the systems, processes and communication tools to bring Duvyzat to DMD patients, the opportunity exists to establish a rare disease business in the U.S. For the moment, though, he said ITF is laser-focused on DMD.

Theyre not the only ones paying attention to the degenerative disease. In June 2023, the FDA greenlit Sareptas Elevidys as the first gene therapy for DMD. Then, in October, Santhera Pharmaceuticals got the nod for Agamree, a novel corticosteroid that will be marketed in the U.S. by Catalyst Pharmaceuticals.

The DMD pipeline is also flush with new candidates, including Pfizers mini-dystrophin gene therapy fordadistrogene movaparvovec, for which Phase III data are anticipated in the second half of 2024, and REGENXBIOs one-time gene therapy. REGENXBIO reported positive efficacy data earlier this month from the first patient to receive a higher dose level of the treatment in a Phase I/II trial.

I think weve seen some exciting therapeutics come along under investigation in recent years, McDonald said.

Heather McKenzie is a senior editor atBioSpace. You can reach her atheather.mckenzie@biospace.com. Also follow her onLinkedIn.

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FDA Approves Third Duchenne Muscular Dystrophy Treatment in Nine Months - BioSpace

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