Category Archives: Genetic Therapy

- Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editingfor PKU -

- Manufacturing Enhancements Led to Improved Productivity, Quality and Scalability of Commercial Process, Confirmed in 2,000L Bioreactor -

- Data Highlight Unique Characteristics of AAVHSC Genetic Medicines Platform -

BEDFORD, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of data at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. Among Homologys seven presentations are data from its in vivo nuclease-free gene editing program for phenylketonuria (PKU) and in vivo gene therapy program for metachromatic leukodystrophy (MLD), both of which are in IND-enabling studies. Presentations also focus on the Companys commercial manufacturing platform, as well as data on the differentiating characteristics of Homologys family of AAVHSC vectors, particularly when compared to other AAVs, which highlight the potential of the Companys dual gene therapy and editing platform.

Homology has made substantial progress in understanding the unique properties of our AAVHSC-based technology and this enables us to move our dual genetic medicines platform forward to develop potential treatments, or cures, for patients, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. We are pleased to share data here that describe the molecular methods we have developed to characterize in vivo, nuclease-free gene editing efficiency and precision at the DNA level. Additional data from our in vivo MLD gene therapy program demonstrates the impact on key biomarkers in two species, as well as the durability of effect in the murine model of disease with data out to 52 weeks. Underpinning all our programs is our internal GMP process and manufacturing capabilities, where we have now confirmed our commercial HEK293 suspension platform at the 2,000-liter scale, bringing our total internal capacity to 3,500 liters. Additionally, we are presenting data showing improved AAVHSC packaging as compared to the non-Clade F vector AAV5.

Highlights from Homologys 2020 ASGCT Presentations

The presentation, Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells using AAVHSC15, a New AAV Derived from Hematopoietic Stem Cells (AAVHSC), describes quantitative molecular methods to measure efficiency and precision of nuclease-free, homologous recombination-based gene editing. The studies, which used a single I.V. administration of a gene editing construct to insert the human PAH gene, which is mutated in people with phenylketonuria (PKU), in a humanized liver murine model, show:

Two posters related to Homologys internal commercial manufacturing platform will be presented.In Molecular Design and Characterization of Packaging Plasmid Sequences for Improved Production of Novel Clade F AAVHSCs, the data demonstrate:

In Development and Scalability of Transfection-Based Production and Purification of Novel Clade F Adeno-Associated Viruses Isolated from Human Hematopoietic Stem Cells (AAVHSCs), Homology describes high-quality productivity and scalability of its mammalian, suspension-based manufacturing, including:

Related to Homologys HMI-202 investigational gene therapy for MLD, the presentation, Gene Therapy for Metachromatic Leukodystrophy (MLD) That Crosses the Blood-Nerve and Blood-Brain Barriers in Mice and Non-Human Primates, details that a single I.V. administration:

In collaboration with Childrens Hospital of Philadelphia (CHOP), Homology also presents, In Vivo Transduction of Murine Hematopoietic Stem Cells after Intravenous Injection of AAVHSC15 and AAVHSC17, which shows:

As Homology has advanced its AAVHSC technology, it is presenting mechanistic data on the platform, including the following two presentations.In Role of Terminal Galactose in Cellular Uptake, Intracellular Trafficking, and Tissue Tropism Using Adeno-Associated Viruses Isolated from Human Stem Cells (AAVHSCs), the data show:

In AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors, the studies demonstrate that AAVHSCs:

For more information about the presentations, visit Homologys website at http://www.homologymedicines.com/publications.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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Homology Medicines Announces Presentations on its In Vivo Gene Therapy and Gene Editing Programs and Commercial Manufacturing Platform at the American...

Japan's national health insurance will cover a gene therapy drug for a rare childhood genetic disorder that costs 167 million yen ($1.56 million) per treatment, making it the most expensive medication funded by the public system, government officials said Wednesday.

An advisory panel to the health minister approved provision of Swiss pharmaceutical giant Novartis AG's drug Zolgensma for spinal amyotrophy patients under the age of 2 starting as early as May 20, the officials said.

The drug, which costs over 200 million yen in the United States, is known as the world's most expensive medication.

Supplied photo shows samples of Swiss pharmaceutical giant Novartis AG's gene therapy drug "Zolgensma" for spinal amyotrophy patients. (Photo courtesy of Novartis)

It is a one-time therapy for the genetic disorder, which causes motor neuron loss and muscle wasting, and its coverage by insurance will offer hope for patients of the disease and their families.

With Japan's social security expenses ballooning amid the rapid aging of its population, some experts have expressed concerns over the burden on the insurance system of inclusion of the treatment. They also note that new drugs have tended to be highly expensive in recent years.

But a senior health ministry official said that given the small number of patients of the rare illness, "the fiscal impact is going to be limited."

The previous most expensive drug in Japan was Kymriah, approved last May to treat leukemia and other hematologic cancers, at 33.49 million yen.

Novartis Pharma K.K., a Tokyo-based unit of the Swiss pharmaceutical company, produces and sells both Kymriah and Zolgensma.

Spinal amyotrophy affects one or two infants out of every 100,000 and can lead to severe respiratory problems and early death. Without use of an artificial respirator, it is said that most die within 18 months.

Zolgensma will be given as a one-time infusion into the vein, which can introduce normal genes into human cells to recover motor function.

Novartis expects that the drug will be administered to about 25 patients per year in Japan, estimating annual sales of 4.2 billion yen.

Under the Japanese insurance system, out-of-pocket medical expenses for those under 2 are set at 20 percent of the total. But the real payment is minimal as the central and municipal governments cover almost all the expense under subsidy programs.

Related coverage:

Japan health insurance to cover new leukemia therapy worth 33 mil. yen

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Japan's health insurance to cover $1.5 million gene therapy drug - Kyodo News Plus

PharmaSphere: Emerging Biotechnologies-Gene Therapy market gives us the acute prediction regarding sales and trends:The current report focuses on the impact of Covid-19 on PharmaSphere: Emerging Biotechnologies-Gene Therapy market industry. This report covers all the important areas like how the key players are enhancing their activities for their survival in the worldwide PharmaSphere: Emerging Biotechnologies-Gene Therapy market business. Graphs and flowcharts are being used for analyzing the information to be acceptable.

Key player focused on this market are: Advantagene, Amarna Therapeutics, AnGes MG, Inc., Applied Genetic Technologies Corporation, AskBio, Avalanche, Bluebird bio, Celladon Corporation, Dimension Therapeutics, American Gene Technologies International Inc,

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Providing acute information.

Professional study for the period 2020-2023a.

Details of upstream raw materials, downstream demand and production value.

Market growth factors.

Market segmentation:

Basis of segmentation: Types of products, application and region.

By Type: Type 1, Type 2

Significant PharmaSphere: Emerging Biotechnologies-Gene Therapy application along with their consumption details: Application 1, Application 2

The time period to estimate the market size:

The history year 2015-2019

The base year 2019

Estimated 2020

Forecast 2020-2023a

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Effecting factors, opportunities, challenges, threats of the global PharmaSphere: Emerging Biotechnologies-Gene Therapy market. Competing products.

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Table of Contents:

Chapter 1 About the PharmaSphere: Emerging Biotechnologies-Gene Therapy Industry

1.1 Industry Definition and Types

1.2 Main Market Activities

1.3 Similar Industries

1.4 Industry at a Glance

Chapter 2 World Market Competition Landscape

2.1 PharmaSphere: Emerging Biotechnologies-Gene Therapy Markets by Regions

Market Revenue (M USD) and Growth Rate 2015-2023a

Sales and Growth Rate 2015-2023a

Major Players Revenue (M USD) in 2020

2.2 World PharmaSphere: Emerging Biotechnologies-Gene Therapy Market by Types

2.3 World PharmaSphere: Emerging Biotechnologies-Gene Therapy Market by Applications

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2.4.1 World PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Revenue and Growth Rate 2015-2020

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3.1 Major Production Market share by Players

3.2 Major Revenue (M USD) Market share by Players

3.3 Major Production Market share by Regions in 2020, Through 2023a

3.4 Major Revenue (M USD) Market share By Regions in 2020, Through 2023a

Chapter 4 Supply Chain Analysis

4.1 Industry Supply chain Analysis

4.2 Raw material Market Analysis

4.2.1 Raw material Prices Analysis 2015-2020

4.2.2 Raw material Supply Market Analysis

4.2 Manufacturing Equipment Suppliers Analysis

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PharmaSphere: Emerging Biotechnologies-Gene Therapy Market Current Trends, SWOT Analysis, Strategies, Industry Challenges, Business Overview and...

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA Congress, which will take place virtually from June 11-14, 2020.

An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA, including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial.

The accepted abstract is now available on the EHA conference website: https://ehaweb.org/congress/eha25/key-information-2/.

Abstract Title: Initial Safety and Efficacy Results With a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Hematopoietic Stem and Progenitor Cells in Transfusion-Dependent -Thalassemia and Sickle Cell DiseaseSession Title: Immunotherapy - ClinicalAbstract Code: S280

About the Phase 1/2 Study in Transfusion-Dependent Beta ThalassemiaThe ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About the Phase 1/2 Study in Sickle Cell DiseaseThe ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

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CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is expected to be presented at the European Hematology Associations upcoming congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com/ or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, information regarding the data that is expected to be presented at the European Hematology Association (EHA)s upcoming Congress. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with theSecurities and Exchange Commissionand available through the company's website atwww.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European...

PHENOMENAL WOMEN SCIENCE & RESEARCH

The San Diego Union-Tribune and the Womens Museum of California are celebrating a century of female achievement in San Diego to mark the 100th year of womens suffrage in America.

The second installment of this series pays tribute to pioneering female scientists and researchers who pushed boundaries in exploring our world and beyond and helped cultivate new generations of curious thinkers.

Spotlighted is Sally Ride, the first U.S. woman in space, who encouraged girls interest in science with her namesake educational program, Sally Ride Science, based at UC San Diego. Here are 11 other women in science and research you should know.

Margaret Burbidge

(U-T file)

A lot of people told Margaret Burbidge she was invading a mans world in the late 1930s when she took her first steps toward becoming an astronomer. She was undeterred even though many key telescopes were off-limits to women. Burbidge pushed through the sexism and became one of the most influential astronomers of her era, largely due to her insights about the chemical composition of stars. Her work helped scientists figure out how stars are made and earned her the nickname Lady Stardust. She also helped to develop the Hubble Space Telescope. And in 1962, she became a founding faculty member at UC San Diego, where she continued research that would later earn her the National Medal of Science. Burbidge died on April 5 at the age of 100.

Karen Nelson

(Howard Lipin/The San Diego Union-Tribune)

Karen Nelson was an early bloomer. At age 7, she joined fellow students in studying how nutrients and sunlight affect the growth of plants. The experiment stoked her interest in science. She went on to become a Cornell-trained physiologist who specializes in the study of the human microbiome the genetic material found in all of the micro-organisms that live in and on our bodies. Nelson led the first group of scientists in publishing the first major paper on the human microbiome. The paper spotlighted an obscure area of research that is now regarded as indispensable to understanding and treating everything from diabetes to multiple sclerosis to depression. Nelson today serves as president of the J. Craig Venter Institute, the renowned research center in La Jolla.

The UC San Diego graduate floated out of an airlock and into history in October as a member of the first all-female team of spacewalkers. The 42 year-old astronaut achieved the fete from the International Space Station, during a six-month mission in which she also conducted research thats meant to help astronauts stay safe and healthy on trips to the moon and Mars. Meir also became a popular host of space-to-Earth broadcast interviews, including an especially poignant one with TV host Stephen Colbert. And she appeared on camera to give earthlings who were sheltering at home from the coronavirus lots of advice about how to live in isolation. Her future could be even brighter she is among the astronauts NASA will consider as crew members for missions to the moon.

Ellen Ochoa

(Cindy Lubke Romero/The San Diego Union-Tribune)

Theres a word that often appears immediately after Ellen Ochoas name: first. In 1991, she became the worlds first Hispanic female astronaut. Two years later, she became the first Hispanic woman to travel in space, streaking into orbit aboard the shuttle Discovery. In 1999, she was a member of the first shuttle crew to dock with the International Space Station. In 2013 Ochoa, who grew up in La Mesa and graduated from San Diego State University, became the first Hispanic to be appointed director of NASAs Johnson Space Center. In 2017, she was inducted into the U.S. Astronaut Hall of Fame. A year later, she retired from NASA, capping a career that spanned nearly three decades.

Maria Goeppert-Mayer

(Evening Tribune)

When it was founded in 1960, UC San Diego quickly hired a handful of renowned professors to signal other faculty that La Jolla was the place to be. The first recruits included Maria Goeppert-Mayer, a German-born theoretical physicist whose discoveries about the nucleus of atoms would help revolutionize everything from weaponry to power generation. Her contribution earned her a share of the 1963 Nobel Prize in physics. She was the first woman in the U.S. to win that prize. The San Diego Union-Tribune responded with a now-infamous headline: S.D. Mother Wins Nobel Physics Prize. Fifty-five years would pass before another woman won the Nobel in physics.

Olivia Graeve

(Courtesy of UC San Diego)

When astronauts return to the moon, they may be flying in a spacecraft made safer by Olivia Graeve. The UC San Diego engineer designs new materials that are meant to withstand extreme environments. She developed and tested an extraordinarily strong type of steel, providing a possible material for everything from spacecraft to body armor. The work occurs at the Cali-Baja Center for Resilient Materials and Systems, which Graeve founded after she became UCSDs first Latina engineering professor. In the summer, the Tijuana native also brings students from Mexico and the U.S. to campus to conduct research, helping cultivate new generations of engineers.

Flossie Wong-Staal

(Koji Sasahara/AP)

Its impossible to count how many lives shes saved, but the number is enormous. Flossie Wong-Staal helped identify the cause of AIDS in 1983 while working at the National Cancer Institute. A short time later, she became the first scientist to clone HIV, then finished mapping the virus genetically. In 1990, Wong-Staal joined the UC San Diego faculty, doing landmark research that has helped fight HIV/AIDS. She also helped turn UCSDs Center for AIDS Research into a research power, and greatly advanced the field of gene therapy. The Chinese-American virologist retired from UCSD in 2002 but has remained active in science and was inducted into the National Womens Hall of Fame in 2019.

Ayana Elizabeth Johnson

(Evan Agostini/Invision/AP)

Not long ago, the wondrous Caribbean island of Barbuda did little to protect its coral reefs and manage its fish populations. Today it does a great deal through programs that Ayana Elizabeth Johnson helped to shape after she earned a doctorate at UC San Diegos Scripps Institution of Oceanography. It was a first step in her rapid rise as an influential voice in sustainable fishing and ocean conservation. Johnson went on to found and lead Ocean Collectiv, a La Jolla conservation consultancy. She also founded the Urban Ocean Lab, a think tank that helps coastal cities. And she played a key role in organizing the 2017 March for Science, which drew more than 1 million participants worldwide.

Balboa Park is so lush its hard to believe it was once a bland patch of land. Many people infused it with life. But none were more important than Kate Sessions, a botanist and horticulturalist who leased part of the park as a growing field in the late 1800s. Sessions planted a variety of trees, ranging from oak to cypress to eucalyptus. She also brought in jacaranda, and helped found the San Diego Floral Association. Her work earned Sessions the nickname Mother of Balboa Park. The honor was about more than beauty. Sessions also studied plants and chronicled how they grew and changed, bringing her worldwide attention in the scientific community. In 1939, the year before she died, the American Genetic Association honored Sessions with the Frank N. Meyer medal, one of the most coveted honors in plant genetics.

Shirley Meng

(David Baillot/UCSD)

Everyone knows that batteries die. But were you aware that they first become sick? Thats the word that Shirley Meng uses to describe what happens when batteries stop holding a charge. It is a poorly understood process. But Meng has been making important discoveries about the phenomenon at UC San Diego, where she is director of the Sustainable Power and Energy Center. Meng, a nano-engineer, specializes in creating new tools and techniques for visualizing, in real-time, whats happening as a battery fails. Her work is meant to improve everything from smartphone service to the range of self-driving cars. Meng who is known as the battery doctor also founded Super 8 Technologies, a company that is developing battery technology that could be used by the military and in space exploration.

Carol Padden

(Sandy Huffaker)

Some linguists used to pointedly question whether American Sign Language, or ASL, is a genuine language. Padden helped establish that ASL is not only legitimate, but that it is a very precise, complex and expressive way of communicating. Padden, who is deaf, has done similar work on Al-Sayyid Bedouin Sign Language. Shes made her contributions as a linguistics researcher and communications professor at UC San Diego. She also is dean of the Division of Social Sciences, UCSDs largest program. In 2010, Padden was honored for her work by being named a MacArthur Genius Fellow. She continues to operate a research lab, something rarely done by high-ranking university administrators.

Sources: UC San Diego, San Diego State University, NASA, J. Craig Venter Institute, Wikipedia, MacArthur Foundation, New York Times, Los Angeles Times, San Diego Union-Tribune

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Phenomenal San Diego women in science and research - The San Diego Union-Tribune

XconomyBoston

Gene therapies deliver healthy genetic material to patients cells to replace a mutated, disease-causing variant. Dyno Therapeutics aims to create better delivery vehicles for those genes by using machine-learning tools to engineer new types of harmless viruses that are more effective and simpler to manufacture.

The Cambridge, MA-based biotech emerged from stealth Monday having signed deals with two drug makers who want to apply its technology to their gene therapy effortsagreements its founders say are lucrative enough to fund the company for years to come. Dyno spun out of George Churchs lab at Harvard Medical School in late 2018 with $9 million in seed funding from Polaris Partners and CRV.

Now Dyno has signed collaboration agreements with Novartis (NYSE: NVS) and Sarepta Therapeutics (NASDAQ: SRPT) to design superior versions of the adeno-associated viruses, or AAVs, commonly used in gene therapies, and the company says the deals may preclude it from needing to raise outside funds again. Thats a rarity for a biotech that is already considering advancing its own product candidates in addition to striking R&D arrangements with other biotechs and pharmaceutical companies.

The first gene therapy in the US was approved in 2017. Finding better AAVs in which to insert genetic material has proven challenging because tweaking the complex protein shells to improve one property, such as targeted delivery, can impede others, such as their ability to evade the immune system.

With small molecules and antibodies, the drug is the small molecule or the antibody, and sometimes youll require a delivery system but thats not the important part, Polaris partner Alan Crane, one of Dynos founders, said in an interview. Its almost flipped in gene therapy, because everyone knows what gene to deliver, generally, for a particular disease, but they dont know how to deliver it, and we really need these better vectors.

Dyno will work with Novartis to use its CapsidMap platform to design superior AAV vectors for gene therapies for eye diseases; Sareptas area of interest is muscle disease.

Under the terms of the deals, the larger companies will be responsible for taking any gene therapy product candidates created within the collaborations through preclinical and clinical testing and commercialization. In the Novartis agreement, Dyno gets an undisclosed amount of money up front plus research funding and license fees. If any products advance, it will be eligible for payments tied to clinical, regulatory, and sales milestones, plus royalties on sales.

In the Sarepta agreement, during the research phase of the collaboration, Dyno will be eligible for more than $40 million in payments. If candidates are developed, it will be eligible for additional payments tied to development milestones, plus royalties. The company wouldnt disclose additional financial details, but said if everything goes according to plan, the deals it has struck to date could collectively bring the startup more than $2 billion.

CEO Eric Kelsic said that when he joined Churchs lab, he aimed to use his experimental and computational biology background to combine the latest technologies in high-throughput biology, advanced machine learning, and protein engineering. He was exposed to gene therapy through Churchs work with the gene editing technology CRISPR-Cas9, one of the tools being deployed to address genetic diseases.

It just seemed like the perfect application of the technologies we had been working on, which are certainly going to transform all of protein engineering, Kelsic said in an interview.

The company takes available data, plus more it generates itself using high-throughput measurement technologies, and uses it to build machine-learning models that suss out the most optimal synthetic capsids. Using machine learning allows Dyno to take into account a number of important propertiesdelivery, immunity, packaging size, and manufacturing, for examplewhile weighting those most important to the particular disease indication. Thats compared to todays efforts, which generally can only tweak one property at a time.

Its been the case in the past that when you only select for one property, for example the efficiency of delivery, that might be improved, but the manufacturability might actually get more challenging, Kelsic said. Thats a tradeoff which has really limited engineering efforts in this space, but now we can overcome that using our platform, optimizing for both the efficiency and the manufacturability and so on, across all the different properties that are important.

Machine-learning tools also allow each experiment to build upon the findings of previous iterations.

When Crane and Kelsic first met, in June 2018, the scientist presented the investor with a spreadsheet of companies that were interested in what the startup had developed. That was before Dyno had published any of its results.

Before joining Polaris in 2002, Crane headed corporate development at Millennium Pharmaceuticals. (Takeda Pharmaceutical (NYSE: TAK) acquired Millennium in 2008 for $8.8 billion). Since joining the VC firm in 2002, Crane has been founder, chairman, or CEO of seven of the companies it has started.

We and a lot of folks in the VC world have been looking at AI applications to healthcare, and increasingly to life sciences and biology, but this was by far the best application to biology that I have ever seen, Crane said. Ive seen a lot of business development, and Ive never seen such robust interest in a young company platform.

Crane says Dyno is currently in talks with other companies that will likely lead to one or two more partnerships like those it has struck with Novartis and Sarepta. The company says it wont allow any company to use its tech to explore a certain disease area exclusively so that it can potentially be used across the industry. In addition to eye and muscle diseases, company is also looking at how to design better vectors for disorders of the central nervous system and the liver.

Along with Kelsic, Church, and Crane, Dynos co-founders are Sam Sinai, its lead machine learning scientist, Adrian Veres, and Tomas Bjorklund.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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Dyno Debuts With Plan, Partnerships For Better Gene Therapy Vectors - Xconomy