Holy volume!! Nice to see life again! Long overdue folks ... GLTA

Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IH
January 05 2023 - 07:00AM
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Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OTL-203, a hematopoietic stem cell (HSC) gene therapy being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The company expects to initiate a global registrational trial evaluating the efficacy and safety of OTL-203 compared to standard of care in the second half of 2023.
“Based on data from the proof-of-concept trial, treatment with a single administration of OTL-203 has the potential to address a range of multisystemic manifestations of MPS-IH,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. “Our recent interactions with the FDA have been productive and we look forward to advancing this registrational study designed to generate the data necessary to enable global regulatory submissions and potentially provide an urgently needed new treatment option for the MPS-IH community.”

The study is a multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with MPS-IH compared to standard of care with allogeneic hematopoietic stem cell transplant (HSCT). A total of 40 patients with a confirmed diagnosis of MPS-IH who meet the study inclusion criteria will be randomized 1:1 to receive either OTL-203 or allogeneic HSCT. The study is powered to demonstrate superiority of OTL-203 over HSCT.

The primary endpoint, which will be measured at two years post-treatment, comprises a composite of clinically meaningful outcomes, including death, the need for rescue treatment, treatment failure, immunological complications, as well as severe cognitive and growth impairment. Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability. The company expects to open up to six sites in the United States and Europe.

“The complications associated with MPS-IH involve multiple organ systems and have a lasting impact on patients’ quality of life despite treatment with allogeneic HSCT,” said Paul Orchard, M.D., a study investigator and professor in the Division of Pediatric Blood and Marrow Transplantation and Cellular Therapy Program at the University of Minnesota Medical School. “Existing therapeutic options are associated with significant morbidity and mortality. There is experience acquired over decades that treatment with allogeneic HSCT does not adequately impact manifestations of the disorder such as growth and other skeletal issues. Furthermore, patients can still experience an irreversible decline in neurocognitive function. We look forward to facilitating this study to characterize the potential clinical impact of OTL-203 for MPS-IH patients.”

In an earlier, single center proof-of-concept study, eight patients diagnosed with MPS-IH were treated with investigational OTL-203 between July 2018 and December 2019. Previously published results showed all patients had stable cognitive development post-treatment. In addition, at last follow-up (ranging from 12-24 months), all participants had progressed along expected growth percentiles of healthy children and exhibited longitudinal growth that was considered within the normal range adjusted for age and gender. Treatment with OTL-203 was generally well-tolerated with no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes reported to date.

“MPS-IH is a devastating disease that places a significant burden on affected children and their families,” said Terri Klein, president and chief executive officer of the National MPS Society. “New treatment options are desperately needed to better address some of the more severe symptoms of the disease. We are encouraged by today's announcement and the hope that a one-time gene therapy could offer this community if approved.”

About MPS-I
Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (GAGs). The accumulation of GAGs across multiple organ systems results in multiple symptomatic manifestations of the disease including severe neurocognitive impairment, skeletal deformities, cardiovascular and pulmonary complications, impaired motor function, loss of hearing and corneal clouding. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I. Approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome (MPS-IH), and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have limitations, such as inadequate impact on some of the more severe manifestations of disease, as well as significant morbidity and mortality. At present, Newborn Screening (NBS) for MPS-I has been established in multiple geographies, including the United States and Europe.

About OTL-203
OTL-203 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IH. It uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. OTL-203 is being developed in partnership with the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. OTL-203 has received rare pediatric disease and priority medicines (PRIME) designations from the FDA and European Medicines Agency, respectively.

About Orchard Therapeutics
At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of

Just watch .. AF up?

ORTX

Again, only a matter of time before ORTX rises to the top. Why, you ask? ORTX is doing this for the RIGHT reasons. The good of humanity is always worth more than any dollar signs. GLTA

It's only a matter of time before the United States Food and Drug Administration will adapt all that is gene therapy. Listen to the scientist!!! It's an absolute pleasure to be invested in such a forward looking company!!! GLTA!

ORTX should be going in one direction. UP ,UP, UP, and away!!!

How does a company who just got approved for reimbursement of the world's most expensive drug go down in value???? 3.9 million US dollars for one treatment of Libmeldy. Orchard Thereputics is backed by NICE and NHS for MLD Patients. Things are looking up for this company's future.

ORTX looking prime for a move . Weekly pincher and 30 day bullish divergence on the daily

Everywhere I go - you are there Clay.

ORTX looks like a good one. Was just about
to do some research but you've assessed this
security, so I shall listen to you before
adjudication.

Regards

Briboy i

Learn more about how Orchard is leveraging the power of hematopoietic stem cells to potentially treat disorders of the central nervous system: https://t.co/NBOkAxqTPK pic.twitter.com/NUtxC9rClR

— Orchard Therapeutics (@orchard_tx) January 6, 2021

* * $ORTX Video Chart 01-06-2021 * *

Link to Video - click here to watch the technical chart video

Hey no cheating, stock must rise with dignity and bravery on its own!!!!!

So you need a buyout tomorrow. We'll see what we can do lol!

Need $35 to make stock picking multibaggers a Nobel Prize category with a specific candidate in mind, or just a 4 part mini series with all the profits going to poor kids with a special "feed",lol.

Would love a close near $7 or higher!

ORTX 6.69 :have minor amount, as Ted Williams never let me bat for him, so why should I stand in his way?

$ORTX Looking for $10 minimum

$7.50 Feb Calls for 1.25 are a steal...

https://www.orchard-tx.com/about/our-story/

$ORTX I like this volume here. Added a bunch of calls...

"ex vivo autologous gene therapy"

In some stock and Calls

A one-time treatment. The potential for lifelong impact.
Orchard is building on decades of research and advancements in the field of gene therapy to develop potential cures for rare and often-fatal diseases. Our ex vivo autologous gene therapy approach is designed to use a person’s own blood stem cells and insert into those cells a working copy of the missing or faulty gene. By giving these gene-corrected cells to the patient, we aim to permanently correct genetic disorders with a single treatment.

Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)...

This is not Libmeldy
8K

Orchard Therapeutics was notified earlier this week that a patient treated under a compassionate use program in 2016 with Strimvelis®, a gammaretroviral vector-based gene therapy approved by the European Medicines Agency (EMA) for the treatment of ADA-SCID, has been diagnosed with lymphoid T-cell leukemia. Preliminary findings suggest this diagnosis may be attributable to an insertional event related to treatment with Strimvelis. The patient is undergoing treatment for the leukemia at a specialty center, and we are conducting a full investigation to determine potential causality. Our thoughts are with the patient and family during this time.

Leukemia arising from the insertion of gammaretroviral vectors into the genome, a process known as insertional oncogenesis (or mutagenesis), is a known risk factor for gammaretroviral vector-based gene therapy and is described in the Strimvelis product information as a potential risk of treatment.

Strimvelis is the only gammaretroviral vector-based gene therapy in Orchard’s portfolio. Each of Orchard’s other pipeline therapies employ a self-inactivating (SIN) lentiviral vector-based approach that has been specifically designed to avoid insertional oncogenesis after administration. No evidence of insertional oncogenesis related to lentiviral vector-based hematopoietic stem cell (HSC) gene therapy has been reported in any indication.

Patient safety continues to be our highest priority, and Orchard has notified EMA and relevant local European regulatory authorities of this adverse event. Sixteen patients have been treated with Strimvelis since its approval in 2016, and no additional patients will be treated with the therapy before the investigation is complete. The company will determine the future of Strimvelis following discussions with relevant stakeholders and will provide further updates as appropriate.

Orchard Therapeutics Gets CHMP Approval for Libmeldy
6:29 am ET October 16, 2020 (Dow Jones) Print

By Chris Wack

Orchard Therapeutics Plc said Friday the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy, an investigational gene therapy for the treatment of metachromatic leukodystrophy.

The company said the CHMP's positive opinion will now be reviewed by the European Commission, which has the authority to grant marketing authorization for Libmeldy in the European Union.

A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression.

Libmeldy is designed as a one-time gene therapy. The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh formulation.

Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

“We are encouraged by EMA’s decision to grant PRIME designation to OTL-203, which was based on an initial clinical assessment of data supporting the potential benefit of our HSC gene therapy for patients with MPS-IH beyond the current standard of care,” said Anne Dupraz-Poiseau, PhD., chief regulatory officer of Orchard. “In 2021, we look forward to building upon the promising early data in the ongoing proof-of-concept study and plan to initiate a registrational trial to advance a potential new treatment for patients.”

The PRIME program is designed to enhance regulatory support in the EU for the development of promising investigational medicines that, based on early clinical data, may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier: enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the marketing authorization application. For more information please visit the EMA website at www.ema.europa.eu.

Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.
Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard

$230M cash & burning $38M/Q
European approval of Libmeldy around October
Licensing agreement w/ GlaxoSmithKline $GSK
Analyst PT $17-$31
70% institutional ownership

Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I
September 01 2020 - 07:00AM

Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

“We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up,” said Maria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.”

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

Rapid hematologic reconstitution, with neutrophil and platelet engraftment within 21 days following treatment.
Biological efficacy established by sustained supranormal alpha-L-iduronidase (IDUA) enzyme expression in peripheral blood for all patients within 3 months post-gene therapy and up to 18 months in the first treated patient.
Metabolic correction as measured by reduction in glycosaminoglycan (GAG) levels in urine achieved in all patients by 6 months post-gene therapy, with sustained correction out to 18 months in the first treated patient.

Further results for the first two treated patients demonstrated:
Rapid metabolic correction of GAG levels in the cerebral spinal fluid (CSF), reflecting restoration of IDUA enzyme expression in the central nervous system.
Improved motor function and acquisition of cognitive and language skills.
Stabilization or improvement in white matter abnormalities, cervical canal stenosis, kyphosis and vertebra deformity, as measured by brain and spine MRI scores.
Continued growth progression above the 50th percentile of normal.
Improved range of motion (an indicator of joint stiffness).

“We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. “With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.”

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.