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5 Cell and Gene Therapy Decisions to Watch in 2024 – BioSpace

Pictured: A collage representing cell and gene therapy/Taylor Tieden for BioSpace

In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven additional cell and gene therapy products are expected to receive approval by years end, according to a March report from the Alliance for Regenerative Medicine.

All signs point to 2024 surpassing 2023 as a landmark year for cell and gene therapy, David Barrett, CEO of the American Society of Gene & Cell Therapy (ASGCT), told BioSpace.

The first approval this year belonged to Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy, which won the FDAs nod in January for use in transfusion-dependent beta thalassemia. This followed the agency's December 2023 approval of Casgevy as one of the first two cell-based gene therapies to treat patients with sickle cell disease. Italso represented the first FDA approval of a therapy using CRISPR/Cas9 technology. Then, in February, Iovance Biotherapeutics Amtagvi was approved as the first one-time cell therapy for a solid tumor and the first tumor-infiltrating lymphocytes therapy, for advanced melanoma patients who have worsened after being treated with certain other therapies failed. Finally, last month, the FDA greenlit Orchard Therapeutics Lenmeldy, which entered the U.S. market as the first gene therapy for children with metachromatic leukodystrophy, and the words most expensive drug, with a $4.25 million price tag.

Looking forward, the FDA has upcoming PDUFA dates for several more novel CGTs, including a traditional in vivo gene therapy delivered via viral vector, a couple of gene-corrected cell therapies in which a patients cells are modified by gene therapy outside of the body and then reinfused, and a new CAR-T.

Two Q1 reports, from ASGCT and theAlliance for Regenerative Medicine, highlight some of these regulatory actions as potential catalysts for the sector, with approvals poised to propel the CGT space. The ASGCT report includes a list of noteworthy events in Q1 2024, while ARMs report makes the case that 2024 could be a banner year for cell therapy.

Here, BioSpace reviews five products under regulatory review that were highlighted by both organizations.

Indication: Hemophilia B

Therapy type: In vivo gene therapy

Action date: April 27

Later this month, the FDA will rule on Pfizers gene therapy for hemophilia B, Beqvez. This engineered version of the factor IX coagulation gene carried by an adeno-associated virus is administered via a single infusion.

Beqvez has been approved by Health Canada to treat adults with hemophilia B based on positive data from the Phase III BENEGENE-2 study, which showed a significant reduction in bleeding rate and infusion frequency.

The Big Picture

An FDA approval would put Pfizer in competition with CSL Behring, whose gene therapy Hemgenix, which is also administered via a single intravenous infusion, became the first FDA-approved gene therapy for hemophilia B in November 2022. Pricing details for Beqvez are not yet available, but Hemgenix costs $3.5 million per dose. Chris Boshoff, Pfizers chief oncology officer, told BioSpace the company aims to leverage its experience to ensure smooth market entry and efficient delivery to eligible patients.

Indication: Recessive dystrophic epidermolysis bullosa

Therapy type: Gene-corrected cell therapy

Action date: May 25

Next up is Abeona Therapeutics pz-cel, which delivers a functional collagen-producing COL7A1 gene into a patients own skin cells using a retroviral vector, for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB).RDEB, a rare connective tissue disorder, causes severe skin wounds, pain and life-threatening complications stemming from compromised immunity due to a deficiency in the COL7A1 gene, preventing the production of functional type VII collagen.

In November 2023, the FDA granted priority review to pz-cel based on clinical data from the Phase III VIITAL study and long-term results from a Phase I/IIa study, which demonstrated sustained wound healing and pain reduction.

The Big Picture

Ira Leiderman, managing director of healthcare at Cassel Salpeter, underscored the importance of evaluating therapeutic options against the rarity and impact of the disease. A positive decision on Abeonas pz-cel will help address the high unmet need of RDEB patients and may lead to transformative interventions in this challenging rare genetic disorder, Leiderman told BioSpace.

If approved, pz-cel would follow Krystal Biotechs Vyjuvek, the first gene therapy approved for recessive or dominant DEB in May 2023. Abeona said in March it is actively preparing for the potential U.S. launch of pz-cel, including discussions with treatment sites and payer engagement.

Indication: Leukocyte adhesion deficiency-1

Therapy type: Ex-vivo vector gene therapy

Action date: June 30

While Rocket Pharmaceuticals initially anticipated a decision on its gene therapy for leukocyte adhesion deficiency-I (LAD-I) by March, the FDA requested more review time and extended the deadline to June 30.

Severe LAD-I, a rare genetic disorder affecting children, is caused by mutations in the ITGB2 gene that lead to life-threatening infections. Without regular bone marrow transplants, survival beyond childhood is rare. Kresladi contains patient-derived hematopoietic stem cells genetically modified with a lentiviral vector to carry functional copies of the ITGB2 gene, crucial for leukocyte adhesion and infection-fighting.

In November 2023, the FDA accepted Rockets BLA for Kresladi with priority review, following positive efficacy and safety data from a global Phase I/II study, in which all nine LAD-I patients were alive 12 to 24 months post-infusion. Significant reductions in infection rates were observed compared to pre-treatment levels, along with the resolution of LAD-Irelated skin lesions and restoration of wound healing capabilities.

Kresladi also holds the FDAs Regenerative Medicine Advanced Therapy, Rare Pediatric, Fast Track and Orphan Drug designations.

The Big Picture

This marks Rockets inaugural product filing and is a notable advancement for patients, offering an alternative to bone marrow transplant, which carries significant risks and may not be readily accessible. Rocket is enhancing its commercial infrastructure in preparation for a potential product launch, including center initiation, channel strategies, education and payer engagement.

Rocket CEO Gaurav Shah told BioSpace the FDA is reallocating reviewers to focus on rare diseases and complex biologics, necessitating changes and a transition period. Shah noted that the delayed decision, based on the FDAs request for clarity on chemistry, manufacturing and controls information submitted by Rocket, is common among CGTs and does not raise significant concerns beyond ensuring the regulator has sufficient resources for the approval process.

Indication: Advanced synovial sarcoma

Therapy type: T cell receptor therapy

Action date: August 4

Adaptimmune is gearing up for the potential launch of its inaugural product in the sarcoma franchise, afami-cel, intended for treating advanced synovial sarcoma, with a PDUFA date set for August 4. Afami-cel received FDA priority review in January.

Synovial sarcoma, which makes up 5% to 10% of soft tissue sarcomas, typically affects individuals under 30, with a five-year survival rate of 20% for metastatic cases. Recurrence is frequent, necessitating multiple lines of therapy and potential exhaustion of treatment options. Afami-cel is a single-dose engineered T cell receptor therapy targeting MAGE-A4-posititive tumor cells. The therapys clinical data from the SPEARHEAD-1 trial revealed that about 39% of patients experienced clinical responses, with a median response duration of around 12 months. Median overall survival was about 17 months, contrasting with historical data of less than 12 months for those who received two or more prior lines of therapy. Some 70% of responders to afami-cel were alive two years post-treatment.

The FDA granted afami-cel Orphan Drug Designation for the treatment of soft tissue sarcomas and Regenerative Medicine Advanced Therapy designation.

The Big Picture

If approved, afami-cel would become the first approved engineered T cell therapy for this type of cancer. In November 2023, the Investigational New Drug (IND) for another T cell therapy, lete-cel, was transferred from GSK to Adaptimmune for the pivotal IGNYTE-ESO clinical trial, following an interim analysis showing a 40% response rate in synovial sarcoma or myxoid/round cell liposarcoma patients.

Adaptimmune CEO Adrian Rawcliffe said that the clinical results from the pivotal trial position lete-cel as a complement to afami-cel, potentially allowing the companys sarcoma franchise to significantly expand its reach. He noted that leveraging the same commercial infrastructure intended for afami-cel could facilitate the efficient delivery of lete-cel to the market. Afami-cel would become the first engineered T cell therapy for a solid tumor. The franchise, including both afami-cel and lete-cel, is projected to deliver up to $400 million in U.S. peak year sales, Rawcliffe said in March.

Indication: B cell acute lymphoblastic leukemia

Therapy type: CAR-T cell therapy

Action date: November 16

In accepting Autolus Therapeutics BLA for its lead next-generation CAR-T therapy obe-cel for relapsed/refractory adult acute lymphoblastic leukemia (ALL) in January, the FDA set a PDUFA target action date of November 16.

Obe-cel, an investigational CD19 CAR-T cell therapy, is designed to enhance clinical activity and safety compared to existing therapies by incorporating a fast target binding off-rate, minimizing T cell activation. In December 2022, Autolus hailed the Phase II FELIX trial as a success, as interim analysis showcased an overall remission rate of 70% for obe-cel in leukemia patients. CAR-T cell concentration peaked and persisted at 75% in peripheral blood after a median of 166.5 days post-infusion. The trial also demonstrated positive safety findings.

Obe-cel holds the FDAs Orphan Drug and Regenerative Medicine Advanced Therapy status. Earlier this month, the company;s obe-cel marketing application was accepted by the European Medicines Agency.

The Big Picture

The potential approval of a second cell therapy for solid tumors this year suggests breakthroughs in treating these cancers may be near, Stephen Majors, a spokesperson for the ARM, told BioSpace. There is an increasing focus on solid tumors, a previously elusive area for cell and gene therapy, he said.

A recent $250 million deal granted BioNTech access to obe-cel, with the partner to aid in the launch and development program expansion and receive royalties based on net sales. Autolus expects obe-cel peak sales to exceed $300 million.

Autolus could face competition from Gilead Sciences subsidiary Kite, which in 2021 gained FDA approval for its CAR-T therapy Tecartus, the first such treatment for ALL, achieving a 65% complete remission rate.

Ana Mulero is a freelance writer based in Puerto Rico. She can be reached at anacmulero@outlook.com and @anitamulero on X.

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5 Cell and Gene Therapy Decisions to Watch in 2024 - BioSpace

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Nanoscope Therapeutics Enhances Mutation-Independent Retinal Gene Therapy Programs with Appointment of Allen … – PR Newswire

DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies. Dr. Ho is Attending Surgeon and Director of Retina Research at Wills Eye Hospital and Co-Director of the Wills Eye Hospital Retina Service.

"We are excited to have Dr. Allen C. Ho join Nanoscope as Chief Medical Advisor," said Sulagna Bhattacharya, Nanoscope co-founder and CEO. "As a world-renowned retina specialist and leader in innovative treatments for retinal diseases, his insight will be invaluable to Nanoscope. MCO-010 has the potential to be the first gene therapy available for patients with advanced retinitis pigmentosa (RP), and Dr. Ho's contributions will help ensure that potential. We also look forward to adding his perspective to our development of MCO-010 for Stargardt disease, building upon the promising results of our Phase 2 STARLIGHT clinical trial."

"I look forward to working even more closely with the team members and leadership at Nanoscope who have been working on ambient light sensitive optogenetic strategies for retinal diseases over the past decade," said Dr. Ho. "The recent release of their 2-year Phase 2b RESTORE Trial data is an important moment for the retina community and I am pleased to help deliver this transformational treatment to serve those suffering from advanced RP. Further, I am also excited about the Nanoscope pipeline and translational potential of image-guided non-viral laser delivery of MCO-020 in improving vision in patients with geographic atrophy secondary to age-related macular degeneration".

Allen C. Ho, MD, FACS, FASRSAllen C. Ho MD, FACS is Attending Surgeon and Director of Retina Research and Co-Director of the Retina Service of Wills Eye Hospital, Professor of Ophthalmology at Thomas Jefferson University and partner of Mid Atlantic Retina. He maintains special interests in macular diseases, diabetic retinopathy, surgical retinal diseases and clinical trials investigating new treatments for vitreoretinal diseases including gene and cell therapies and new surgical drug delivery devices and techniques. His experience includes collaborative translational and clinical trial clinical research with expertise in study design, methodological testing, data analyses, surgical instrumentation and procedure development, execution and communication of these studies and their study results, and commercialization focusing on patient access to care. Dr. Ho has served on the US FDA Ophthalmic Device Panel, American Academy of Ophthalmology (AAO) Innovation Task Force, AAO Ophthalmic Retina Technology Assessment Committee, AAO Retina Measures Group, AAO IRIS Registry Committee and is a leader of Eyecelerator, the largest ophthalmic innovation meeting in the world.

AboutNanoscope Therapeutics, Inc.Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company's lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed their end-of-study visit for the Phase 2 STARLIGHT trial of MCO-010 therapy for patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy. To learn more, visit http://www.nanostherapeutics.com and follow them on LinkedIn.

Investor Contact: Argot Partners 212-600-1902 [emailprotected]

SOURCE Nanoscope Therapeutics

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Documentary about a family’s journey to Minnesota for gene therapy premieres in Minneapolis – MPR News

NINA MOINI: The Minneapolis Saint Paul International Film Festival is currently under way. And tonight, a documentary will premiere called "Sequencing Hope." The film is directed by Lindsey Seavert and Maribeth Romslo. It follows an Alabama family who came to Minnesota to get their young daughter life-saving gene therapy for a rare disease. Let's listen to a clip from the trailer.

CELIA GRACE HAMLETT: Can you hold my hand?

[CHUCKLES]

They give me the medicine.

SUBJECT 1: There is lots of other gene therapy research on the horizon.

SUBJECT 2: That's got far-reaching consequences to move medicine forward.

SUBJECT 3: In my heart, I feel that the good Lord has something in store for Celia Grace.

SUBJECT 4: We just pray, Lord, for a miracle. We pray for a healing for Celia Grace.

SUBJECT 5: You know, when it comes to your kids, you're going to do whatever it takes to protect them.

SUBJECT 6: If it saves one child, then I feel like we have accomplished something.

NINA MOINI: Celia Grace Hamlett was four-years-old when she came to M Health Fairview Masonic Children's Hospital in 2021 and became the first person in the US to undergo the experimental gene therapy. Her family's in town for the film's premiere tonight and Celia Grace's dad, Gary, joins us now along with their doctor, Doctor Paul Orchard. Thank you both for being here.

GARY HAMLETT: Thank you.

PAUL ORCHARD: Thank you, Nina.

NINA MOINI: Yeah, and Gary, let me start with you if I might. Tell us about your daughter, Celia Grace. She's seven-years-old now and I understand she was diagnosed with this rare and often fatal genetic disorder, MLD, when she was diagnosed at three-years-old. What were her options at that point?

GARY HAMLETT: Well, at that point had one or had two options. One was bone marrow transplant or the other was gene therapy that was only being done in Milan, Italy.

NINA MOINI: Wow. Doctor Orchard, can you tell us all what MLD is?

PAUL ORCHARD: Certainly. Appreciate the opportunity to speak with you today. So metachromatic leukodystrophy is a rare inherited disorder. It's what we call a lysosomal disease. The lysosome is an organelle within cells that help break down materials that the cell is attempting to get rid of.

And there's a number of enzymes that are present in the lysosome that help accomplish that. Arylsulfatase A. is one of those. And in this circumstance, if you are unlucky enough to receive a mutation within the arylsulfatase gene from both mom and from dad, then you're affected with the disease.

But both parents who have one normal copy of the gene are absolutely fine. There's nothing to suggest that they have any sort of problem, but again, if you receive an abnormal copy from both parents then you see the disease. And in this situation, it's primarily a neurologic disorder. It occurs in kids as young as one or so in terms of manifestations of the disease, but it's progressive and lethal if there's no therapy.

NINA MOINI: Wow, that's just so much to take in, Gary. And you know, you mentioned having to maybe think about treatment over in Milan. How did you hear about the treatment for MLD that was right here in Minnesota?

GARY HAMLETT: Our doctor neurologist in Alabama, Doctor Matt, is the one that contacted us and said, what would y'all think if I told y'all y'all's daughter was going to make history books? At that point we said, what do you mean? And she said, well, your daughter may be the first child in the United States to receive gene therapy for MLD and it will be done at the Masonic Children's Hospital in Minneapolis under the care of Doctor Paul Orchard.

NINA MOINI: And then what did you think? I mean, were you going to have to pay for that?

GARY HAMLETT: Yes. At that point we didn't really care what it cost us being able to save our daughter's life. So our community started doing fundraisers to try to raise money to pay for this.

NINA MOINI: Wow, yeah. Doctor Orchard, can you explain how the gene therapy works and is it accessible to most people or is it just too costly?

PAUL ORCHARD: Well, the gene therapy clinical trials occurred in Europe, as Gary was alluding to, and the data was sufficiently positive that it was approved as therapy in the EU, essentially. So it's been licensed therapy there for several years, but none of the clinical trials have been done here in the US.

And because of the promise of this new therapy, we were gearing up to being able to offer this regardless, but there was the opportunity in this situation from Celia Grace's diagnosis to be able to intervene. So it's just become licensed therapy in the last month or so, as March 18th.

But prior to that and for Celia Grace, we had to petition the FDA to allow us to use it because it's still considered experimental therapy, and get all the approvals from all the various regulatory groups to be able to do that. So it took some time, but it opened the doors. And now we've treated a total of five patients with compassionate use therapy.

NINA MOINI: All right. Is it still pretty pricey, though? I understand it's among some of the priciest treatments.

PAUL ORCHARD: Yes, it is very expensive. So for the compassionate use treatment as an experimental therapy, the company actually donated the cell product, but it's millions of dollars now as licensed therapy.

NINA MOINI: Yeah. So still working to make it more accessible. Gary, you said something that really struck me in the trailer for the film. You said that you take care of people for a living. I understand you work in law enforcement, but you couldn't fix this for your daughter. And it seems like this film is really an exploration of your family's journey. Tell me how did that feel to feel sort of helpless in the moment, but then to see her go through this journey and be, I mean, cured?

GARY HAMLETT: We just felt very helpless, not knowing the outcome of it, how sick Grace was. Just thinking that we were going to lose our daughter. Possibly by the age of five-years-old.

NINA MOINI: Yeah.

GARY HAMLETT: And seeing her now as a normal seven-year-old, running, playing, is going to graduate in kindergarten, and it's just an amazing feeling.

NINA MOINI: Yeah, I'm sure. And so how is she doing? Tell us a little bit just about how she's getting around fine, and she's feeling well.

GARY HAMLETT: Oh, she is rambunctious, non-stop playing, running, doing her schoolwork. She is just like a typical seven-year-old little girl.

NINA MOINI: Yeah, and I understand some more patients are going to be undergoing that same treatment as Celia Grace, which is great news, Doctor Orchard.

PAUL ORCHARD: Yes, I hope it's going to be widely available. As you mentioned, the cost is going to be significant and attempting to determine how we're going to do this. The vast majority of these patients that we treat are obviously not from Minnesota.

And so being able to get insurance that's going to work across state lines and going to be sufficient for this is going to be a challenge. But that's one of the things that we're currently working on.

NINA MOINI: OK, and Gary, I'll leave the last question for you here. What do you hope people will take away from watching your family's story in this documentary?

GARY HAMLETT: The struggles of not knowing the outcome of your child. The struggles of possibly knowing that you will only have a few years with your child. And then knowing that there are people out there willing to help and willing to do anything possible to save your daughter or your son. I just can never repay everybody that along this journey for what they have done for my child.

NINA MOINI: Yeah, and it really sounds like it's some of the best parts of humanity and also some of the hardest struggles that anyone will go through. Thank you so much for sharing that journey and for being here, Gary. And to you as well, Doctor Orchard, thank you, and congratulations on this film reaching an audience today.

PAUL ORCHARD: Thank you.

GARY HAMLETT: Thank you so much.

NINA MOINI: Gary Hamlett is the father of Celia Grace Hamlett and Doctor Orchard is a pediatric blood and marrow transplant physician at M. Health Fairview. Both are featured in the documentary, "Sequencing Hope," which is premiering tonight at 7:00 PM. We'll have that information on our website, mprnews.org.

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US approves gene therapy treatment for hemophilia | National | elpasoinc.com – El Paso Inc.

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Gene Therapy Trial for Sickle Cell Disease Publishes Final Results – Technology Networks

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In a landmark study, an international consortium led by researchers at Childrens Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) a blockage that results in lack of oxygen and painful episodes for at least one year, and 100% were able to remain hospitalization-free for the same length of time.

The findings, published today in theNew England Journal of Medicine, provide the complete details of the critical clinical trial that led to the FDA approval of CASGEVY for the treatment of sickle cell disease in December 2023.

Sickle cell disease is a lifelong condition that causes intense pain due to deformed blood cells that can cause blockages in blood vessels. This can also lead to strokes, organ damage, and shortened lives.

Researchers have been studying the use of gene therapy and CRISPR technology to edit portions of DNA in people with inherited or genetic disorders, like sickle cell disease. In the case of sickle cell disease, the CASGEVY process edits DNA within the patients own cells and enables the patient to produce a different form of hemoglobin in their red blood cells. Clinical trials at CHOP and other sites have shown that successful gene editing can prevent cells from developing the distinctive crescent shape apparent in sickle cell disease and have eliminated pain episodes in almost all patients. CASGEVY was the first FDA-approved therapy developed with CRISPR technology.

In this clinical trial, sickle cell patients who were having significant issues with their disease began to see their problems resolve within months and improve their quality of life significantly, said senior study authorStephan A. Grupp, MD, PhD, Section Chief of theCellular Therapy and Transplant Section, Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy, and Medical Director of theCell and Gene Therapy Laboratoryat CHOP. Grupp was also one of the principal investigators in the clinical trials that led to the approval of CASGEVY and the leader of the studys steering committee.

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A total of 44 patients received exa-cel with a median follow up of 19.3 months. In a total of 30 patients with sufficient follow-up data to be evaluated, 29 (96.7%) were free of VOCs for at least 12 consecutive months. This information is an update for the US Prescribing Information for CASGEVY, which includes an evaluation of 31 patients resulting in a response rate of 93.5%. The safety of treatment was comparable to treatment with hematopoietic and progenitor stem cells, and no malignancies were reported as a result of treatment.

This study was supported by Vertex Pharmaceuticals and CRISPR Therapeutics.

For more information about CASGEVYs prescribing information, warning and precautions, and adverse reactions, visithere.

Additionally, the results of a clinical trial on the efficacy of exa-cel for the treatment of -thalassemia were also published today in theNew England Journal of Medicine. The preliminary results of the trial led to the FDA approval of CASGEVY for transfusion-dependent -thalassemia in January 2024. Grupp was also one of the principal investigators for this clinical trial.

Reference:Frangoul Haydar, Locatelli Franco, Sharma Akshay, et al. Exagamglogene autotemcel for severe sickle cell disease. NEJM. 2024. doi: 10.1056/NEJMoa2309676

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Sequencing Hope: New film details Alabama girl’s historic gene therapy in Minnesota – KARE11.com

After her life-saving treatment in 2021, 7-year-old Celia Grace Hamlett's story of hope became part of the Minneapolis St. Paul International Film Festival.

MINNEAPOLIS A documentary that just premiered during the Minneapolis St. Paul International Film Festival marks the latest twist in a medical journey that has spanned thousands of miles and last several years for the family of a young girl from Alabama.

"We've been so excited and waiting on this," said Gary Hamlett as he walked into the premiere of "Sequencing Hope" with his 7-year-old daughter Celia Grace Hamlett.

KARE 11 first connected with Celia Grace and her family nearly three years ago, when she and her family traveled from their home in Alabama to undergoing an experimental gene therapy treatment at M Health Fairview Masonic Children's Hospital in 2021 that offered her a chance at a normal life.

"(MLD) affects the brain, it affects the nerves, it's progressive and it's lethal," said Dr. Paul Orchard, the Pediatric Blood and Marrow Transplant Physician at M Health Fairview who petitioned the FDA to make Celia Grace the first patient to receive gene therapy to treat MLD in the United States. "I think it's really an opportunity to move the field forward and to better treat the kids."

Thefilm documents how Celia Grace has done exactly that. It follows her and her family through the long journey to that therapy, and her life in the years since.

"She is doing so well, you would never be able to tell she was sick," said Kassie Hamlett, Celia Grace's mother. "Everything is the same as it was, so it has not progressed any at all, so just praying it continues to stay that way."

The history made by Celia Grace came too late for Pat Lang. Her daughter, Maddie Lang, was diagnosed with MLD long before gene therapy was an option.

The documentary also followed Pat's care for Maddie before she died from MLD in 2022.

"The fact there is now something that can save them is a beautiful thing," Pat Lang said. "Am I jealous? You bet. But is it a great thing? You bet."

Just before the documentary premiered, another great thing happened. The FDA approved the gene therapy treatment, opening the door to many others.

"It's a very exciting thing to be able to be able to offer this now and it's not just MLD, it's sickle cell disease, and a number of other inherited diseases," Dr. Orchard said. "They are going to have the same type of opportunity, so this is really where the field is going to go."

"I want people to see our documentary and not give up hope," Kassie said. "The road is rough, but the end is very rewarding."

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Sequencing Hope: New film details Alabama girl's historic gene therapy in Minnesota - KARE11.com

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