Category Archives: Gene Medicine

PARP inhibitors interfere with cancers ability to repair damage to its DNA. They are becoming increasingly useful in treating ovarian cancer.

Valencia Halls oncologist wanted to give her the best possible chance of staying in remission, so in June 2017, she prescribed Zejula (niraparib), a once-daily oral treatment thats part of an emerging class of medicines known as poly (ADP-ribose) polymerase (PARP) inhibitors. Just a few months before Valencia Halls disease returned, Zejula had become the first PARP inhibitor approved by the Food and Drug Administration (FDA) to treat women with recurrent ovarian cancer who do not have a genetic abnormality but previously responded well to platinum-based chemotherapy. Valencia Hall was a perfect candidate for the drug, which is referred to as a maintenance treatment because its prescribed with the goal of preventing ovarian cancer from returning.

She has been free of the disease since starting Zejula and has experienced no side effects, aside from a temporary drop in platelet counts that her oncologist corrected by dialing down Valencia Halls daily dose. Zejula has allowed me to live my life as I see fit, says Valencia Hall, 51, a freelance graphic artist in Phoenix. Its an oral medication, so I dont have to go in for infusions. The opportunity to have this as a maintenance therapy is exciting its hope.

Zejula is one of three PARP inhibitors that are taking an increasingly prominent role in ovarian cancer treatment and improving patients prognosis. With dozens of clinical trials underway, including some that seek to combine PARP inhibitors with other cancer treatments, that role could expand even more.

Drugs in this class work by inhibiting the PARP enzyme, which normally helps damaged DNA repair itself. Preventing this repair causes cancer cells to die, especially those that already have DNA repair defects due to a mutated BRCA 1 or 2 gene or other abnormalities. So far, clinical trials have shown that these drugs can lengthen the time until the disease progresses; their impact on the length of life is still being investigated. For women using a PARP inhibitor for maintenance, the drugs can help increase the time between courses of chemotherapy for recurrent disease.

In the past, we would take patients who had a high risk of recurrence and just watch them until cancer came back because we didnt have maintenance therapies that were effective, tolerable or convenient, says Dr. Bradley Monk, a professor and director of the division of gynecologic oncology at Creighton University School of Medicine at St. Josephs Hospital and Medical Center in Phoenix, Arizona, and also medical director of gynecologic oncology research for the U.S. Oncology Research Network. PARP inhibitors have been significant because theyre expanding the treatment opportunity for many patients, he adds.

Each year, more than 22,000 women in the U.S., about half of whom are over age 63, receive a diagnosis of ovarian cancer, according to the American Cancer Society. A small proportion of women have inherited mutations in BRCA1, BRCA2 or other cancer-related genes that raise their risk of ovarian cancer and can take steps to fend off the disease, including surgery to remove their ovaries. But most cases occur out of the blue, and because the symptoms can be vague and easily overlooked, like bloating or stomach pain, many women do not receive a diagnosis until the disease has advanced to the point where it can be hard to treat. The disease will recur in about 85% of women who initially respond to chemotherapy.

For women with ovarian cancer, PARP inhibitors have been an option since 2014, when the FDA approved Lynparza (olaparib) as a maintenance therapy for patients with BRCA mutations who had received three or more chemotherapy treatments. Rubraca (rucaparib) followed in 2016 and Zejula in 2017.

PARP INHIBITORS REACH MORE PATIENTS

Over the past two years, the FDA approved more uses of PARP inhibitors so that many more patients can benefit from these medicines and access the drugs earlier in treatment. In April 2018, Rubraca was approved as a maintenance therapy to treat recurrent ovarian cancer in women who responded at least partially to platinum-based chemotherapy, whether or not they had a genetic mutation. That December, Lynparza was approved as a first-line maintenance treatment for BRCA-mutated ovarian cancer, meaning patients can be given the drug after successfully completing just one round of platinum-based chemotherapy.

That was based on a clinical trial showing the drug reduced the rate of disease progression or death by 70%.

Most recently, in October 2019, the FDA approved Zejula for use in patients with advanced ovarian cancer associated with a cellular abnormality called homologous recombination deficiency. BRCA1 and BRCA2 are two of these types, but in ovarian cancer, about 17 other such genetic abnormalities can drive the disease. Between 41% and 50% of ovarian tumors are thought to have homologous recombination deficiency, which can be detected with a tumor test, Myriad myChoice CDx, that the FDA also approved last October.

In a clinical trial leading to the approval, 24% of participants with homologous recombination deficiency-positive ovarian cancer responded well to Zejula, experiencing some tumor shrinkage. That may not sound like a high response rate, but it surpasses the overall response rate to PARP inhibitors among all patients with ovarian cancer either with or without mutations, says lead clinical trial investigator Dr. Kathleen Moore, associate professor of gynecologic oncology at Stephenson Cancer Center at the University of Oklahoma.

These are heavily pretreated patients in which the response rate has typically been 12%, so this was double what we normally see, Moore says. The clinical benefit here was quite high. She adds that patients with BRCA mutations did particularly well: Nearly 40% of those who previously responded well to platinum-based chemotherapy responded to Zejula. The most common side effects include gastric upset, mouth sores, rash, headache and dizziness. The drug can also cause anemia and abnormal blood counts, which, in rare cases, can lead to myelodysplastic syndrome, a bone marrow problem, or the blood cancer acute myeloid leukemia.

As PARP inhibitors continue to help a widening population of patients with ovarian cancer, a push is underway to use them earlier in the treatment process. Several trials presented at the 2019 European Society for Medical Oncology conference demonstrated the potential value of that strategy.

One study found an 84% survival rate among patients who took Zejula for two years following chemotherapy, regardless of their homologous recombination deficiency status, compared with 77% who got a placebo. The median progression-free survival period (time from treatment to disease progression) for patients taking the drug was 14 months compared with eight months for those on placebo.

Another study evaluated an investigational PARP inhibitor, veliparib, combined with chemotherapy as a first-line treatment followed by veliparib alone for maintenance. Median progression-free survival among patients on that regimen was 23.5 months versus 17.3 months for those taking a placebo.

The third trial presented at the conference involved Avastin (bevacizumab), a drug that cuts off the blood supply to tumors. The FDA approved Avastin in 2018 to treat advanced ovarian cancer in conjunction with chemotherapy.

During the more recent trial, patients with newly diagnosed advanced ovarian cancer took Avastin plus chemotherapy, followed by Avastin with Lynparza for maintenance. Those who took Lynparza along with Avastin had a median progression-free survival of 22 months compared with 17 months for those who received Avastin and a placebo.

In all three studies, progression-free survival rates were the highest among homologous recombination deficiency-positive patients, but the fact that PARP inhibitors extended survival even among those without those mutations was encouraging to oncologists who treat ovarian cancer.

These were all positive studies that undoubtedly show that PARP inhibitor maintenance following chemotherapy will extend beyond BRCA-associated ovarian cancer, Moore says. Its highly likely that a much larger proportion of women with ovarian cancer who are diagnosed in 2020 will receive a PARP inhibitor (than in previous years). Women will be able to live longer with their cancer because were developing effective therapies that push out progression-free survival.

COMBINATIONS GAIN STEAM

A clinical trial investigating a novel PARP combination proved a lifesaver for Diane Sarver, who first received a diagnosis of ovarian cancer in 2010. Chemotherapy put her cancer in remission three times, but when she relapsed again in 2015, she decided to search for a novel treatment strategy and traveled from her home in Lake Oswego, Oregon, to The University of Texas MD Anderson Cancer Center in Houston.

Sarver was entered into an early-phase trial combining Lynparza with the investigational drug AZD2014, which interferes with a cellular pathway (called phosphatidylino-sitol-3 [PI3] kinase) that drives resistance to PARP inhibitors. Within seven weeks of starting the combination therapy which consists of two oral drugs taken twice a day , Sarvers ovarian cancer came under control. As part of the ongoing trial, she continues to take the two drugs, which, she says, have caused no side effects and shes still disease-free.

Whats notable about Sarvers case is that she didnt inherit any genetic mutations that would predict such a long-lasting response to PARP inhibitors. Although her tumor tested positive for a rare BRCA mutation, it was considered nonactionable scientists did not yet know whether or not the mutation produced an aberrant protein that would make it likely to respond to the drug combination being studied. The theory behind the trial is that blocking PI3 kinase may transform tumors that would not normally respond to PARP inhibition into responders.

Im amazed that Ive had such a good outcome, says Sarver, 69, a retired clinical technologist and mother of two grown children. Her only limitation is that she has to fast two hours before and after taking the drugs, she says, but the lack of side effects has given her the freedom to travel, speak to medical students about her experiences and spend time with her family.

Ive seen both children through their college graduations, Sarver says. I feel completely functional and am grateful to have never experienced any fatigue or other physical restriction.

MD Anderson is now planning several midstage clinical trials combining PARP and PI3 kinase inhibition, including one that pairs Lynparza with Piqray (alpelisib), a drug currently used to treat some patients with breast cancer. In an early trial of the combination that was reported in April 2019, 36% of patients had a partial response of some tumor shrinkage and half achieved stable disease, meaning their cancer didnt get worse. That was impressive, considering the bulk of the patients had become resistant to platinum chemotherapy, says Dr. Shannon Westin, a clinical investigator in the department of gynecologic oncology and reproductive medicine at MD Anderson. And there was a similar response rate regardless of mutation status, which was very exciting.

RESEARCHERS PURSUE MORE USES

Pairing PARP inhibitors with drugs that boost the immune systems ability to fight cancer is another idea being investigated in the treatment of ovarian cancer, because tumors with unstable DNA repair abilities might also be easier for the immune system to recognize. For example, Keytruda (pembrolizumab) inhibits programmed cell death protein 1 (PD-1), an immune checkpoint responsible for keeping the immune system under control.

The drug essentially takes the brakes off the immune system so it can better recognize and attack cancer. In an early trial combining Keytruda with Zejula, 65% of patients with ovarian cancer saw their disease come under control, either with total or partial tumor shrinkage or with stable disease.

Several other ongoing studies are combining PARP inhibition with immunotherapy, including a trial of Zejula with Tecentriq (atezolizumab), an inhibitor of a protein called programmed death-ligand 1 (PD-L1), and Cotellic (cobimetinib), which inhibits the cancer-associated mitogen-activated (MEK) protein. Another study combines Zejula with TSR-042, an investigational PD-1 blocker.

Could PARP inhibitors be useful for some women even earlier in the treatment process? MD Anderson recently started a small trial designed to investigate the potential of using the agents in place of chemotherapy in women with newly diagnosed cancer who have BRCA mutations. During the trial, patients will receive Lynparza for up to three months before moving on to surgery and chemotherapy. A similar trial is ongoing using the PARP inhibitor Talzenna (talazoparib) for BRCA-mutated breast cancer.

There could be many advantages of starting treatment with a PARP inhibitor rather than chemotherapy, Westin says. Patients can take the drugs at home instead of going to a facility for chemotherapy infusions. And PARP inhibitors dont cause many of the uncomfortable side effects common with chemotherapy, like neuropathy (numbness and tingling in the extremities) and hair loss. Some patients could potentially avoid that toxicity, Westin says.

The question is: Do we even need chemotherapy? Westin adds. Can we utilize a more targeted therapy to get better results? This is the next step.

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An Expanding Role For PARP Inhibitors Shows Promise In Treating Ovarian Cancer - Curetoday.com

This report focuses on the global Gene Editing status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Editing development in United States, Europe and China.

Gemstones are elements of minerals which when polished and cut are used for making jewelry and other ornaments, as well as for decoration purposes.

Key drivers attributing to the expansion include early applications of DNA editing to therapeutics. Use of the technology for the disease eradication through direct correction of disturbances in normal physiology, engineering the immune response, and alteration of pathogen targets in the host is anticipated to drive the market with substantial opportunities.

Based on end user, the global gene editing technologies market has been segmented into biotechnology industry, horticulture industry, animal breeding and academic & research institutes. Academic and research institutes are expected to contribute maximum share in the global gene editing technologies market over the f recast period as majority of the therapeutic applications of gene editing are not yet commercialized.

United States is expected to lead the global market due to increasing number academic and research institutes. Market in APAC region is expected to witness significant growth rate over the forecast period owing to expansion activities by key market players in the region.

In 2017, the global Gene Editing market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2018-2025.

The key players covered in this study

Thermo Fisher Scientific

Merck

Horizo??n Discovery

Sangamo BioSciences

Integrated DNA Technologies

Lonza

New England Biolabs

OriGene Technologies

Transposagen Biopharmaceuticals

Editas Medicine

CRISPR Therapeutics

RGen Solutions

Sigma-Aldrich

GeneCopoeia

Genscript Biotech

OriGene Technologies

Agilent Technologies

Market analysis by product type

Crispr

Talen

Zfn

Market analysis by market

Biotechnology & Pharmaceutical Companies

Academic & Government Research Institutes

Contract Research Organizations

Market analysis by Region

United States

Europe

China

Japan

Southeast Asia

India

Central & South America

The study objectives of this report are:

To analyze global Gene Editing status, future forecast, growth opportunity, key market and key players.

To present the Gene Editing development in United States, Europe and China.

To strategically profile the key players and comprehensively analyze their development plan and strategies.

To define, describe and forecast the market by product type, market and key regions.

In this study, the years considered to estimate the market size of Gene Editing are as follows:

History Year: 2017-2018

Base Year: 2017

Estimated Year: 2018

Forecast Year 2018 to 2025

For the data information by region, company, type and application, 2017 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Table of Contents

Chapter One: Report Overview

1.1 Study Scope

1.2 Key Market Segments

1.3 Players Covered

1.4 Market Analysis by Type

1.4.1 Global Gene Editing Market Size Growth Rate by Type (2018-2025)

1.4.2 Crispr

1.4.3 Talen

1.4.4 Zfn

1.5 Market by Application

1.5.1 Global Gene Editing Market Share by Application (2017-2025)

1.5.2 Biotechnology & Pharmaceutical Companies

1.5.3 Academic & Government Research Institutes

1.5.4 Contract Research Organizations

1.6 Study Objectives

1.7 Years Considered

Chapter Two: Executive Summary

2.1 Gene Editing Market Size

2.2 Gene Editing Growth Trends by Regions

2.2.1 Gene Editing Market Size by Regions (2017-2025)

2.2.2 Gene Editing Market Share by Regions (2017-2025)

2.3 Industry Trends

2.3.1 Market Top Trends

2.3.2 Market Use Cases

Chapter Three: Key Players

3.1 Gene Editing Revenue by Manufacturers (2017-2018)

3.2 Gene Editing Key Players Head office and Area Served

3.3 Key Players Gene Editing Product/Solution/Service

3.4 Date of Enter into Gene Editing Market

3.5 Key Players Gene Editing Funding/Investment Analysis

3.6 Global Key Players Gene Editing Valuation & Market Capitalization

3.7 Mergers & Acquisitions, Expansion Plans

Chapter Four: Breakdown Data by Type and Application

4.1 Global Gene Editing Market Size by Type (2017-2025)

4.2 Global Gene Editing Market Size by Application (2017-2025)

Chapter Five: United States

5.1 United States Gene Editing Market Size (2017-2025)

5.2 Gene Editing Key Players in United States

5.3 United States Gene Editing Market Size by Type

5.4 United States Gene Editing Market Size by Application

Chapter Six: Europe

6.1 Europe Gene Editing Market Size (2017-2025)

6.2 Gene Editing Key Players in Europe

6.3 Europe Gene Editing Market Size by Type

6.4 Europe Gene Editing Market Size by Application

Chapter Seven: China

7.1 China Gene Editing Market Size (2017-2025)

7.2 Gene Editing Key Players in China

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Global Gene Editing Market 2020: Drivers, Restraints, Opportunities, Threats, Trends, Applications, Growth Analysis and Forecast To 2025 - Nyse Nasdaq...

WASHINGTON, D.C., Feb. 13, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, today released the agenda for its second annual Cell & Gene Meeting on the Mediterranean. The event will be held from April 15-17, 2020 in Barcelona, Spain.

The event, modeled after ARMs highly successful Cell & Gene Meeting on the Mesa, is expected to attract more than 500 attendees, including senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech representatives, institutional investors, academic research institutions, patient foundations, disease philanthropies, and members of the life science media community.

The agenda includes:

Plenary Session:

Keynote Address:

Panels:

Throughout the two-day event, participants can also attend presentations by more than 50 publicly traded and emerging private companies, highlighting clinical and commercial progress in cell therapy, gene and gene-modified cell therapy, tissue engineering, biomaterials and more. In addition to their presentations, representatives from these organizations will also be available for one-on-one partnering opportunities throughout the conference.

2020 presenting companies include: Adaptimmune, AGTC, Ambys Medicines, AskBio, Aspect Biosystems, Atara Biotherapeutics, Autolus Therapeutics, Avectas, AVROBIO, Axovant Gene Therapies, bluebird bio, Bone Therapeutics, Cabaletta Bio, Caribou Biosciences, Celavie Biosciences, Cellatoz Therapeutics, Cellect Biotherapeutics, CEVEC, Cryoport, Cynata Therapeutics, Flexion Therapeutics, Fraunhofer IZI, Genethon, GenSight Biologics, Healios, Iovance Biotherapeutics, Kiadis Pharma, Kytopen, LogicBio Therapeutics, MeiraGTx, Minerva Biotechnologies, MolMed, Novadip Biosciences, Orchard Therapeutics, Oxford Biomedica, PDC*line Pharma, Polyplus-transfection, Precision BioSciences, Promethera Biosciences, PTC Therapeutics, Recombinetics, REGENXBIO, ReNeuron, Rexgenero, Sangamo, SmartPharm Therapeutics, Standards Coordinating Body for Regenerative Medicine, Theradaptive, ThermoGenesis, Tmunity Therapeutics, Ultragenyx Pharmaceutical, VERIGRAFT, and Vineti.

For full details on the agenda and further information about the event, please visit http://www.meetingonthemed.com.

Registration is complimentary for credentialed members of the media. For members of the media interested in attending, please contact Kaitlyn Donaldson Dupont at kdonaldson@alliancerm.org or Consilium Strategic Communications at ARM@consilium-comms.com.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. Founded in 2009, ARM works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its 350+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centers, investors, and patient groups that comprise the broader regenerative medicine community and is the prominent international advocacy organization in this field.

ARM has 70+ members across 15 countries in Europe. ARM aims to work closely with European stakeholders, leveraging its membership to create a supportive commercial and regulatory environment to create better conditions for the development and commercialization of ATMPs in Europe; develop strong stakeholder support around proposed solutions to improve patient access to ATMPs; promote clear, predictable and efficient regulatory framework across Europe; and promote international convergence of key regulations and guidance. For more information, visit alliancerm.org.

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The Alliance for Regenerative Medicine Releases Agenda for 2020 Cell & Gene Meeting on the Mediterranean - BioSpace

Kiyohiro Yamazaki,1 Yuta Yoshino,1 Kentaro Kawabe,1 Tomomasa Ibuki,1 Shinichiro Ochi,1 Yoko Mori,1 Yuki Ozaki,1 Shusuke Numata,2 Jun-ichi Iga,1 Tetsuro Ohmori,2 Shu-ichi Ueno1

1Department of Neuropsychiatry, Molecules and Function, Ehime University Graduate School of Medicine, Toon, Ehime 791-0295, Japan; 2Department of Psychiatry, Course of Integrated Brain Sciences, Medical Informatics, Institute of Health Biosciences, the University of Tokushima Graduate School, Tokushima 770-8503, Japan

Correspondence: Jun-ichi IgaDepartment of Neuropsychiatry, Molecules and Function, Ehime University Graduate School of Medicine, Shitsukawa, Toon, Ehime 791-0295, JapanTel +81-89-960-5315Fax +81-89-960-5317Email iga.junichi.it@ehime-u.ac.jp

Introduction: Although ATP-binding cassette sub-family A member 7 gene (ABCA7) is known to be associated with Alzheimers disease, the relationship between ABCA7 and schizophrenia has been unknown.Methods: Schizophrenia patients (n = 50; 24 males, 62.1 0.50 years old) and age- and sex-matched healthy controls (n = 50) were recruited for the mRNA analysis. Additionally, a case-control study for the rs3764650 genotypes was performed with 1308 samples (control subjects; n = 527, schizophrenia patients; n = 781). All participants were Japanese, unrelated to each other, and living in the same area.Results: The distributions of the rs3764650 genotypes in schizophrenia patients were not different from that of controls. However, the ABCA7 mRNA expression levels in schizophrenia patients were significantly higher than those in controls by a logistic regression analysis. Additionally, the ABCA7 mRNA expression levels in schizophrenia patients were correlated with the rs3764650 genotypes in a dose-dependent manner.Discussion: The ABCA7 mRNA expression levels in peripheral blood with the rs3764650 genotypes may be related to pathological mechanisms in schizophrenia and may be a biological marker for schizophrenia.

Keywords: schizophrenia, ATP-binding cassette sub-family A member 7 gene, single nucleotide polymorphism, rs3764650, mRNA expression

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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ABCA7 Gene Expression and Genetic Association Study in Schizophrenia | NDT - Dove Medical Press

- Salvador Rico, M.D., Ph.D., named Chief Medical Officer

- Martin Moorhead, Ph.D., promoted to Chief Technology Officer

SOUTH SAN FRANCISCO, Calif., Feb. 11, 2020 /PRNewswire/ --Encoded Therapeutics, Inc.(Encoded), a precision gene therapy company,today announced the appointment of Salvador Rico, M.D., Ph.D., as chief medical officer and the promotion of Martin Moorhead, Ph.D., to chief technology officer. Dr. Rico joins Encoded from Audentes Therapeutics, where he led clinical development of the company's pipeline of gene therapies for neuromuscular disorders. In his three years at Encoded, Dr. Moorhead has guided the development of the company's technology platform for creating innovative AAV-based gene therapies. He previously led the development of clonoSEQ, the FDA-approved next-generation sequencing assay for detecting minimal residual disease in lymphoid malignancies, at Adaptive Biotechnologies.

Encoded Therapeutics, Inc. Logo (PRNewsfoto/Encoded Therapeutics, Inc.)

"Sal is an accomplished physician-scientist with deep experience advancing novel therapeutics through clinical development, and Martin is a strong leader who brings a genomics mindset to all aspects of gene therapy development," said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D."With these appointments, we now have some of the most qualified gene therapy experts in the industry with a proven track record of delivering for patients in need. Their collective experience includes bringing multiple AAV-based gene therapies through clinical development, FDA filings, and approval. I am more confident than ever that our novel gene therapies can make a major impact on patients suffering from debilitating diseases, starting with Dravet Syndrome."

At Encoded, Dr. Rico will lead medical strategy and clinical development of ETX101, which is being developed for patients with SCN1A+ Dravet Syndrome. Dr. Moorhead will lead the technical team that enables Encoded's innovative research platform.

"I am delighted to join an organization that is so committed to transforming patients' lives with the development of next-generation gene therapies," said Dr. Rico. "I look forward to working closely with both the team at Encoded, and with the Dravet Syndrome community, to advance ETX101 through clinical development and ultimately, deliver it to patients in need."

"In building a technology platform that combines the power of genomics and computation with AAV-based gene therapy, Encoded is forging the path for the next generation of precision genetic medicines," said Dr. Moorhead. "I am very proud of what we have accomplished to date and am thrilled at the opportunity to help advance multiple programs for diseases where no treatment options currently exist."

New Leadership Team Appointments

About Encoded

Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positiveDravet Syndrome. For more information, please visitwww.Encoded.com.

Media Contacts

Sarah SuttonGlover Park Groupssutton@gpg.com 202-337-0808

Danielle CanteyGlover Park Groupdcantey@gpg.com 202-337-0808

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SOURCE Encoded Therapeutics, Inc.

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Encoded Therapeutics Expands Gene Therapy Leadership with Key Appointment and Promotion - Yahoo Finance

Biotech innovations dominate the life science news cycle think CRISPR for gene editing or CAR-T for cancer immunotherapy. Medical technologies, encompassing the broad swath of devices and interventional technologies, diagnostics, imaging, and digital medicine, tend to make their marks out of the limelight, yet have an enormous impact on public health and offer significant investment opportunities.

Take, for example, the trajectory of interventional cardiology. Open-heart surgery was long considered the only way to restore blood flow limited by a blockage in a coronary artery. The invention of artery-opening angioplasty not only changed that but, along with significant improvements in surgery overall, contributed to a steep decline in deaths due to cardiac disease. Abetting this decline were advances in engineering minimally invasive techniques, better diagnostic stratification of patients for surgery or interventional approaches, and a rise in improvements in anesthetic monitoring and critical care support. The result is good news for people with acute and chronic cardiac conditions.

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Medtech is a different beast than biotech in the life sciences pack - STAT