A clinical study of Bedford-based Nanoscope Therapeutics one-size-fits-all gene therapy for a rare genetic retina disorder meaningfully improved sight for 11 blind patients, according to the company.

The proprietary therapy delivers multi-characteristic opsin an optically activated protein similar to a molecular solar cell to retinal cells, and can be used to treat an array of diseases.

Nanoscope said it now believes its trial results are long-lasting, possibly improving vision for the patients entire lives. The optimism is based on observing the 11 patients progress for 52 weeks following the trial.

Classic gene therapy can only treat one disease at a time and there are hundreds of genes responsible for causing blindness. Optogenetic gene therapy can target hundreds of different diseases, including retinitis pigmentosa, age-related macular degeneration and Stargardt disease, all with a single product.

The therapy requires no retinal implants, invasive surgeries or goggles. Instead, the gene therapy is administered in a doctors office by a single injection through the eye.

Samarendra Mohanty, co-founder and president of Nanoscope Therapeutics, explained that genes are delivered to the retina, not photoreceptors, because in this instance photoreceptors like rods and cones are already destroyed.

The gene therapy transforms retinal cells into new photoreceptors.

Its like installing a solar panel on your house, on top of your roof ... you get [a] current generated for using in a home, Mohanty said. [Its the] same way the eye needs the current to send to [the] brain.

The year-long study began in 2018. Patients who had no vision at all were selected to participate in 2019. Initial results from the study were reported by the company last year, revealing that all patients experienced improved visual acuity at 16 weeks. All also saw a greater than 90% improvement in accurately describing shapes.

They used to come with their family member or chaperone, co-founder and CEO Sulagna Bhattacharya said about patients in the clinical trial. By 16 weeks, people start[ed] coming all by themselves. Of course, not driving the car, but taking public transportation, she said.

Patients were able to take over daily chores and activities that they could not do alone prior to the gene therapy treatment, the company said.

Nanoscope Therapeutics can now begin studying its gene therapy through a randomized test, which has potential to become pivotal, Mohanty said. The companys goal is to make the treatment available globally at an affordable price.

Current gene therapies can cost $3 million or more, Bhattacharya said. We want to make sure that regular people can afford it because its one drug applicable for so many patients, she said.

The drugs wide range of applications means it can be mass-produced, increasing affordability, Bhattacharya said.

Nanoscope Therapeutics is a spin-off from Nanoscope Technologies, a Bedford company founded in 2009 that has gotten backing from the National Institutes of Health. Nanoscope Therapeutics received a $2 million grant from the National Eye Institute in June 2020, and landed an undisclosed funding round a month later.

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Bedford biotechs gene therapy improves sight for 11 patients with a rare genetic retina disorder - The Dallas Morning News

When Iveric Bio established its gene therapy advisory committee recently, the move signaled the opening of a second front in its mission to treat retinal diseases for which there are no treatments.

Retinal medicine is an extremely nuanced field, so we needed the best of the best scientists and clinicians out there, said Pravin U. Dugel, M.D., president of Iveric Bio. We established the committee once the basic science matured. Now were at the precipice of putting retinal gene therapy into humans.

The mission of the Gene Therapy Inherited Retina Disease Scientific Advisory Committee is to translate the science to the clinic.

A lot of companies miss the translation aspect. Just because the science is valid in the lab and in pre-clinical studies, doesnt necessarily mean it will be adapted to the clinic, Dugel told BioSpace.

The committees insights also will help us prioritize our pipeline and design clinical trials to be more impactful, he added. For example, determining clinical impact isnt as simple as checking vision. The visual acuity, which is a measure only of the central retina, may not improve in first-in-human studies and may not be the appropriate biomarker for some diseases.

Instead, better trial endpoints may be multi-luminance mobility testing, microperimetry, quality of life questionnaires, etc., depending on the target disease. Choosing the appropriate biomarkers, such as reading speed, contrast sensitivity, or other elements would offer meaningful improvements to people who are going blind. Also, the factors that are meaningful to an 85-year-old patient may be different than those of a 14-year-old, he pointed out.

Iveric Bio was founded by retinal specialists. Before joining Iveric Bio 18 months ago and recently becoming president of the company, Dugel himself was a practicing retinal specialist and remains an internationally known physician.

He is a clinical professor at the USC Roski Eye Institute, Keck School of Medicine, University of Southern California; has served as a visiting professor in some 16 nations; and has been the principal investigator in more than 100 multicenter trials. He received the Senior Honor Award from the American Academy of Ophthalmology and has served as a board member for the largest retina society in the United States, the American Society of Retina Specialists (ASRS), and the largest retina society in Europe, EURETINA.

In selecting the gene therapy advisory committee, therefore, we werent starting from scratch. The retinal world is small. There are only 2,500 retinal specialists in the U.S., and everybody knows everybody. They each have a certain niche, he said.

Dugel has worked with many of these committee members before joining the company and has known many throughout his professional life. This collegiality is expected to enhance Iveric Bios gene therapy program.

Members of the new Gene Therapy Inherited Retina Disease Scientific Advisory Committee are:

The gene therapy program on which they are advising currently has five different products, all in the pre-clinical and research phases. The furthest along are IC-100 for RHO-adRP and IC-200 for best1-related retinal diseases. For IC-200, a Phase I/II trial is planned for the second half of this year.

The remaining programs are miniCEP290 for Lebers congential amaurosis type 10; miniABDA4 for autosomal recessive Stargardt disease; and miniUSH2A for Ushers syndrome Type 2A. They are dubbed mini-gene programs because, in these programs, the naturally occurring gene is larger than the capacity of the standard adeno-associated viral (AAV) vector used for gene therapy. Iveric Bio, therefore, is working to develop smaller gene constructs to enable AAV gene delivery.

The company also has two therapeutic product candidates in development. One, Zimura (avacincaptad pegol), is in development for the treatment of geographic atrophy secondary to macular degeneration.

Zimura currently is being studied in a Phase III clinical program, and if results at 12 months are positive, we plan to submit Zimura to the FDA for final approval in this indication, Dugel said.

Zimura also is in a Phase IIb clinical trial to treat autosomal recessive Stargardt disease.

The other product candidate, IC 500, is an HtrA1 inhibitor. It is designed to stop the progression of macular degeneration and is in preclinical development.

When I entered this field, about 30 years ago, treatments for retinal diseases were almost entirely surgical. We couldnt do anything for macular degeneration or most inherited diseases. We could repair retinal detachments, but were unable to treat most of the medical problems we encountered, Dugel recalled. Since then, theres been astonishing progress in this field.

Many of the advances have been in wet macular degeneration.

Were not doing that. We are focusing on diseases for which there is no treatment, Dugel told BioSpace. Retinal conditions with unmet needs are not necessarily rare, either. The biggest cause of blindness is dry macular degeneration, yet theres no treatment for that.

Hes hoping Zimura will solve that.

These two silos, therapeutics and gene therapy, will help Iveric Bio expand its footprint in the retinal arena. The ultimate goal is to uphold Iveric Bios mission statement: to develop transformative therapies for retinal diseases.

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Iveric Bio Expands into Gene Therapy for Untreated Retinal Diseases - BioSpace

Collaboration to Support AavantiBios Diverse Gene Therapy Pipeline; Development and Supply of GMP Cell Lines and Viral Banks

Agreement to Include Manufacture and Supply of Material for GLP Tox Studies and Clinical Trial for Friedreichs Ataxia Patients

CAMBRIDGE, Mass. & SAN DIEGO, Calif.--(BUSINESS WIRE)-- AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, and National Resilience, Inc. (Resilience), a company building the worlds most advanced biopharmaceutical manufacturing ecosystem, today announced a strategic collaboration to support the development and manufacturing of AavantiBios pipeline of therapies, including its lead program in Friedreichs Ataxia (FA).

Resilience will provide process development and GMP manufacturing services including cell lines and viral banks for AavantiBios adeno-associated viral (AAV) vector-based therapeutic FA candidate for use in both pre-clinical studies, and Phase I/II clinical trials in the U.S. and Europe. Resilience will assist in the development and optimization of the manufacturing processes for GLP Tox and first-in human material at its 183,000-square-foot, state-of-the-art facility in Alachua, Florida.

"This partnership supports our immediate and long-term objectives in developing and ultimately commercializing our diverse pipeline of gene therapies, beginning with our lead FA program, said Bo Cumbo, President and Chief Executive Officer of AavantiBio. With an emphasis on Chemistry, Manufacturing and Controls (CMC) for gene therapies, we are committed to ensuring the quality of manufacturing processes along with analytical development. We look forward to a lasting collaboration with Resilience as we execute on our mission of bringing new therapies to patient populations."

We are excited to work with an innovative development-stage company like AavantiBio who is utilizing a unique AAV approach to address the underlying causes of rare genetic diseases, said Rahul Singhvi, Sc.D, Chief Executive Officer of Resilience. Our highly-experienced team will work collaboratively with AavantiBio on process development and analytical testing to enable a seamless transition from development to manufacturing.

FA is a rare inherited neuromuscular disease that causes progressive nervous system damage and movement problems. The multisymptomatic disease usually begins in childhood and leads to degeneration in the spinal cord, peripheral nerves and cerebellum (the part of the brain that controls synchronization and balance) and causes impaired muscle coordination (ataxia) that worsens over time. The neurological degeneration caused by the disease results in unsteady movements, impaired sensory function, and even the loss of speech. Affected individuals can also develop heart problems, diabetes, or curvature of the spine. Though rare, FA affects 1 in every 40,000-50,000 people and is the most common form of hereditary ataxia in the United States.

About AavantiBio, Inc.

AavantiBio is a gene therapy company backed by a premier syndicate of life sciences investors including Perceptive Advisors, Bain Capital Life Sciences, and RA Capital Management, who led the companys recent $107 million Series A financing. Headquartered in Cambridge, Massachusetts, AavantiBio is advancing a diversified gene therapy pipeline in areas of high unmet medical need, including a lead program in Friedreichs Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. The company benefits from strategic partnerships with the University of Floridas renowned Powell Gene Therapy Center and the MDA Care Center at UF Health where AavantiBios co-founders and renowned gene therapy researchers Barry Byrne, M.D., Ph.D. and Manuela Corti, P.T., Ph.D. maintain their research and clinical practices. Learn more at http://www.aavantibio.com.

About Resilience

Resilience (National Resilience, Inc.) is a first-of-its-kind manufacturing and technology company dedicated to broadening access to complex medicines and protecting biopharmaceutical supply chains against disruption. Founded in 2020, the company is building a sustainable network of high-tech, end-to-end manufacturing solutions to ensure the medicines of today and tomorrow can be made quickly, safely, and at scale. Resilience offers the highest quality and regulatory capabilities, and flexible and adaptive facilities to serve partners of all sizes. By continuously advancing the science of biopharmaceutical manufacturing and development, Resilience frees partners to focus on the discoveries that improve patients lives. For more information, visit http://www.Resilience.com.

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AavantiBio and Resilience Announce Strategic Collaboration for Gene Therapy Development and Manufacturing - BioSpace

Beginning this week, the U.S. Food and Drug Administration (FDA) ended its compliance and enforcement discretion policy with regard to certain human cell, tissue, and cellular and tissue-based products (HCT/Ps). HCT/P manufacturers without an FDA-approved marketing application may find themselves subject to FDA scrutiny under the Federal Food, Drug, and Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act). Firms will have to think hard about whether to submit investigational new drug applications (INDs) or marketing applications, wind down production, or risk running afoul of applicable laws and regulations governing unapproved biological products. They should also be mindful of how data collected to date can help to inform next steps.

HCT/Ps are articles containing or constituting human cells or tissues that are intended for implantation, transplantation, infusion, or transfer into a human recipient.1 They are regulated under sections 351 and 361 of the PHS Act and/or the FD&C Act and implementing regulations.2 Generally, HCT/Ps will have undergone substantial manufacturing, and clinical data will be necessary in order to establish the safety and effectiveness of these products prior to marketing.3 However, there are two exceptions to this general rule. First, FDA will exempt HCT/Ps from the premarket review and approval processes and regulate them exclusively under section 361 if they:

Second, FDA has explained that it views autologous cells or tissues that are removed from an individual and implanted into the same individual without intervening processing steps beyond rinsing, cleansing, sizing, or shaping to raise no additional risks of contamination and communicable disease transmission beyond that typically associated with surgery.5 As such, premarket review and other regulatory requirements will not apply to an HCT/P that is removed and reimplanted in its original form in the same individual during the same surgical procedure.6

In the fall of 2017, FDA announced a temporary compliance and enforcement discretion policy, under which FDA applied a risk-based approach to enforcement actions for HCT/P manufacturers whose products were subject to the premarket review process but did not yet meet regulatory requirements.7

The enforcement discretion did not mean that FDA was turning a completely blind eye to HCT/P manufacturers that were marketing unapproved products. Indeed, the Agency stated its intent to take enforcement action against products that had safety concerns reported or that FDA believed had the potential to raise safety concerns. Thus, notwithstanding the enforcement discretion policy, FDA focused its enforcement actions on HCT/Ps that posed a higher risk, as measured by the products route of administration and the diseases and conditions for which they were being used,8 and issued at least 14 warning letters and 24 untitled letters to HCT/P manufacturers during the enforcement discretion period.9 And, in a potential preview of things to come, FDA also sent approximately 400 it has come to our attention letters to various health care providers, clinics, and manufacturers of cellular products, alerting them that they may be marketing unapproved HCT/Ps and subject to FDAs compliance and enforcement policy.10

FDA initially intended the compliance and enforcement policy to last three years (through November 2020) to afford HCT/P manufacturers time to comply with the premarket approval requirements.11 In July 2020, FDA extended the compliance and enforcement policy through May 31, 2021.12 Leaders at the Agency, including Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER), and Melissa Mendoza, Deputy Director for CBERs Office of Compliance and Biologics Quality, confirmed that the policy would not be extended beyond May 31, 2021.13 The compliance and enforcement discretion policy is now over.

Beginning on June 1, 2021, manufacturers of cell and gene therapy HCT/Ps that do not hold an approved marketing application or fall within one of the two exempt categories described above, are operating at risk of violating the PHS Act and/or FD&C Act. Ultimately, such manufacturers need to engage with FDA, and in particular with CBER, to determine the path to an approved marketing application. In most cases, this means submitting a biologics license application (BLA) under section 351(a) of the PHS Act. This type of BLA, known as a stand-alone BLA, will need to include (among other things) all of the data and information necessary to establish the ongoing quality, safety, purity, and potency of the HCT/P for its intended uses.14 It will be particularly important for manufacturers of HCT/Ps to consider what data they may have collected through real world use of their products to date, including during the period of enforcement discretion, and how that data may be able to be used to support approval of their BLAs. The rapidly evolving chemistry, manufacturing, and controls (CMC) standards and guidance for cell and gene therapy products should be consulted for important context.15 An acceptable manufacturing facility inspection is typically required before a BLA can issue,16 and many inspections have been significantly delayed during the current COVID-19 public health emergency.17

The first step toward compliance generally will be to open an IND to conduct clinical trials in support of such a BLA, and to engage in discussion with FDA regarding appropriate submission of a marketing application. In addition to standard BLA-focused meetings under the Prescription Drug User Fee Act (PDUFA), including initial pre-IND meetings,18 CBERs Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting process allows for informal preliminary advice before the pre-IND phase.19

HCT/Ps also may qualify for designation as regenerative medicine advanced therapies (RMAT). This designation, which must be granted by FDA, can be requested either concurrently with an IND submission or by amending an existing IND. Cell therapies, therapeutic tissue engineering products, human cell and tissue products, certain human gene therapies and xenogeneic cell products, and combination products that include such therapies or productsother than HCT/Ps that fall within an exception and are regulated solely under section 361 of the PHS Actwill be eligible for RMAT designation if they are intended for serious or life-threatening disease or conditions, and preliminary clinical evidence indicates the potential to address unmet medical needs.20 RMAT-designated products will receive the benefits of fast track and breakthrough therapy designation, including early interactions with FDA that can be used to discuss potential surrogate or intermediate endpoints to support accelerated approval.21 This can also mean fewer COVID-19-related inspection delays.22 RMATs may also receive priority review if they are intended to treat serious conditions and, if approved, would provide a significant improvement in the safety or effectiveness of the treatment of the condition.23 RMATs also may be appropriate for approval through the accelerated approval pathway, and there may be additional benefits in terms of fulfilling associated post-approval requirements.24

Importantly, though, even active discussions with FDA will not preclude enforcement action, including issuance of warning and untitled letters, and potentially injunctions,25 civil monetary penalties,26 and seizure.27 FDA has made clear that, for products that do not fall within one of the exempt categories, the risk of enforcement can be averted only by obtaining approval of a marketing application.28 In fact, last month, CBER Director Marks drew a red line: Such products should not be marketed without FDA approval.29

Manufacturers of cell and gene therapy HCT/Ps without FDA approval can take discrete steps to mitigate the risk of regulatory enforcement. Specifically, manufacturers should carefully review the applicability of exemptions from premarket review requirements to their HCT/Ps and consider if their products or distribution processes can be restructured to align with the exemptions. Where appropriate, manufacturers should prioritize engaging with FDA and initiating concrete steps toward preparing marketing applications. They should also ensure that, in the meantime, their HCT/Ps are used only under an IND. Alternately, manufacturers may consider winding down operations, particularly if the likelihood of obtaining an approval or qualifying for an exemption proves infeasible. If manufacturers choose to continue existing operations without taking corrective action, they operate at risk of FDA enforcement.

1 21 C.F.R. 1271.3(d).

2 FDA, Guidance for Industry, Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use at 34 (July 2020), https://www.fda.gov/media/109176/download [hereinafter HCT/P Guidance]; see also 21 U.S.C. 355(a); 42 U.S.C. 262(a); 21 C.F.R. Part 1271.

3 See 42 U.S.C. 262(a) and 264; 21 C.F.R. 601.2(a).

4 See 21 C.F.R. 1271.10(a); HCT/P Guidance, at 3.

5 FDA, Guidance for Industry, Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception at 3 (Nov. 2017), https://www.fda.gov/media/89920/download.

6 See 21 C.F.R. 1271.15(b).

7 See HCT/P Guidance, at 22 n.32.

8 FDA, Press Release, FDA Announces Comprehensive Regenerative Medicine Policy Framework (Nov. 15, 2017), https://www.fda.gov/news-events/press-announcements/fda-announces-comprehensive-regenerative-medicine-policy-framework.

9 Melissa Mendoza, Deputy Director, Office of Compliance and Biologics Quality, Center for Biologics Evaluation and Research, Address at the Food and Drug Law Institute: Key Issues in Regenerative Medicine Regulation and Approval (May 18, 2021).

10 Id.; see also Peter Marks, Advancing the Development of Safe and Effective Regenerative Medicine Products, U.S. Food & Drug Admin. (Apr. 21, 2021), https://www.fda.gov/news-events/fda-voices/advancing-development-safe-and-effective-regenerative-medicine-products.

11 See 82 Fed. Reg. 54290, 5429192 (Nov. 17, 2017); HCT/P Guidance at 22 n.32.

12 See HCT/P Guidance at 12.

13 See Peter Marks, Director, Center for Biologics Evaluation and Research, Address at the Food and Drug Law Institute: Center for Biologics Evaluation and Research (CBER) Session (May 20, 2021); see also Mendoza, supra note 9, Marks, supra note 10.

14 See 42 U.S.C. 262(a); see also 21 C.F.R. 601.2(a); HCT/P Guidance at 34.

15 See 85 Fed. Reg. 5447, 544748 (Jan. 30, 2020); FDA, Guidance for Industry, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (Jan. 2020); see also FDA, Cellular and Gene Therapy Guidances, https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances (last updated Jan. 5, 2021) (providing directory to other FDA cellular and gene therapy guidance documents).

16 42 U.S.C. 262(c); 21 C.F.R. 601.20(d).

17 See FDA, Resiliency Roadmap for FDA Inspectional Oversight at 2 (May 2021), https://www.fda.gov/media/148197/download.

18 See FDA, Draft Guidance for Industry, Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products at 3 (Dec. 2017), https://www.fda.gov/media/109951/download.

19 See FDA, INTERACT Meetings, https://www.fda.gov/vaccines-blood-biologics/industry-biologics/interact-meetings (last updated July 9, 2020).

20 21 U.S.C. 356(g).

21 Id.; FDA, Guidance for Industry, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions at 6 (Feb. 2019), https://www.fda.gov/media/120267/download [hereinafter RMAT Expedited Programs Guidance].

22 See Resiliency Roadmap for FDA Inspectional Oversight, supra note 17, at 2 (RMAT-designated products are considered mission-critical for inspection prioritization).

23 See RMAT Expedited Programs Guidance at 9.

24 21 U.S.C. 356(g)(6)(7); RMAT Expedited Programs Guidance at 911.

25 See 21 U.S.C. 332(a) and 331(a)(b).

26 See id. 333(a).

27 See id. 334(a).

28 See Mendoza, supra note 9 (At this point, thats not good enough to avoid potential compliance or enforcement action. So please, please do not count on any enforcement discretion going forward.)

29 See Marks, supra note 13.

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The End of Enforcement Discretion for Cell & Gene Therapies: Thinking Through Next Steps - JD Supra

On Jun 3, 2021

A geneticist with Morristown-based Atlantic Health Systems physician network, Atlantic Medical Group, recently treated the first patient in the United States in a clinical trial with a genetic therapy developed by BioMarin, designed to help patients who are not able to metabolize phenylalanine, an amino acid found in nearly all proteins.

Darius Adams, MD, medical director of the Jacobs Levy Genomic Medicine and Research Program at Atlantic Health System and division chief of pediatric genetics, provided the therapy as part of a clinical trial that includes Atlantic Health System and currently two other hospitals worldwide.

The investigational therapy was given intravenously to an adult diagnosed with phenylketonuria(PKU), a genetic disorder that affects people from birth. The patient will be monitored for five years to determine the long-term safety and efficacy of the treatment.

We are excited to be on the forefront of studying this new therapy that could radically improve the lives of so many people, said Dr. Adams. The impacts of phenylketonuria stretch further than simply dietary concerns, and this therapy offers great promise to these individuals to possibly having a better quality of life, throughout their lifetime.

Dr. Adamss work is supported by his colleagues, Christina Flora, research coordinator, and Lindsay Schumacher, dietician.

Newborn screeningbegan with the development of a blood test for phenylketonuria in the 1960s. The test was given to babies soon after birth, in order to allow physicians and dietitians to begin dietary therapy as needed as soon as possible.

While a certain amount of phenylalanine is needed by the body, an overabundance of it can cause a range of problems, from behavioral and intellectual disorders, to severe brain damage.

To date, patients who have PKU have had limited treatments available to them, primarily in the form of specific dietary restrictions that begin in a persons infancy or daily injections when older. Due to an abundance of carbohydrates replacing protein in these altered diets, obesity can often be a side effect. Maintaining these restrictions can become challenging later in life, such as during college years and depending upon a persons profession in adulthood.

The disorder is caused by a genetic mutation in a persons DNA. The gene therapy carries a gene sequence to replace it, targeting the liver cells and releasing the genetic material. The body then reads the gene sequence and makes a copy of the enzyme that can break down the phenylalanine.

We are in an exciting period of genomic medicine, exploring the ability to help correct genetic diseases, said Dr. Adams. We are hopeful that the success of this gene therapy will be the doorway to solving many more common health disorders.

Dr. Adamss colleagues throughout at Atlantic Health System are enthusiastically supportive of his participation in the trial.

At Goryeb Childrens Hospital and Childrens Health throughout Atlantic Health System, we strive to make patients lives healthier from the very earliest stages of childhood, said Walter Rosenfeld, MD, chair of pediatrics. While this initial trial involved an adult, its success offers great hope to parents and patients of all ages as an important new tool that could have a huge impact on their lives.

The Jacobs Levy Genomic Medicine and Research Program at Atlantic Health System provides individualized, full-spectrum genomic care, designed to ensure that patients receive the medical and health services that will most benefit them.

This includes diagnostic testing based on symptoms, family history and result consultation with a licensed genetic counselor to thoroughly explain positive results and discuss further medical care options. For chronic patients, care includes follow up visits, blood level monitoring and medication management.

The genomic program treats and follows numerous patients diagnosed with PKU, with doctors and a dietitian helping them to closely monitor and track their level of phenylalanine, maintain their dietary modifications and prescribe injectable treatment.

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Patient at Atlantic Health Receives First US Dose for New Gene Therapy - njbmagazine.com