Category Archives: Gene therapy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Be Biopharma (Be Bio), whose mission is to pioneer the emerging new class of engineered B cells as medicines, today announced that Krishnan Viswanadhan, Pharm.D., MBA, has been appointed President and Chief Operating Officer and Brad Hartman has joined as Chief People Officer. They join a rapidly growing team of scientists, drug developers, manufacturing experts, and business leaders whose track records have led to the creation and development of some of the industrys most impactful gene and cell therapies.

I am thrilled to welcome both Krishnan and Brad to Be Bio. Krishnan brings an exceptional track record building, rapidly scaling, and effectively leading operations that have delivered multiple highly impactful cell therapy products to patients, said Joanne Smith-Farrell, PhD, Chief Executive Officer at Be Bio. At Be Bio, we believe people are our most important asset, and Brads track record of building cell and gene therapy enterprises with innovative, inclusive cultures will help us push the boundaries of whats possible to create an unparalleled work experience. added Dr. Smith-Farrell. Both of these industry leaders have come to Be Bio to build something truly unique a fantastic team and a bespoke operating environment designed especially to unleash the breadth and depth of our leading engineered B cell medicines platform on behalf of patients who need options.

Prior to joining Be Bio, Krishnan served as Senior Vice President, Global Cell Therapy Franchise Lead at Bristol Myers-Squibb (BMS). In this role, he was responsible for setting the vision and developing the integrated, enterprise-wide cell therapy franchise strategy, building core capabilities, and driving key investments to support long-term growth. He oversaw the program team leaders responsible for the cross-functional development, registration, and lifecycle management of Breyanzi (liso-cel), a CD19 CAR T in large B cell lymphoma, and Abecma (ide-cel), the first BCMA CAR T in relapsed/refractory multiple myeloma. Prior to BMS, Krishnan held senior roles in Business Development & Global Alliances as well as Global Project Leadership at Celgene Corporation. He co-founded Advyzom, a boutique consulting company, and held multiple roles in leading development teams and global regulatory strategy at Hoffman-La Roche. Krishnan is a registered pharmacist and received his Pharm.D. from Rutgers University and holds a MBA from Cornell University. He currently serves as a non-executive director on the board of JW Therapeutics, a leading cell therapy company in China.

What is remarkable about Be Bio is the unprecedented opportunity afforded by this novel B cell therapy platform to tackle a broad set of therapeutic applications that cannot be addressed by other modalities, said Dr. Viswanadhan. The possibilities for transforming patient lives in such a wide range of serious and life-threatening diseases, the passion of the team, and the focus on patients are truly inspiring. I am honored to join this incredible team to build a portfolio of disruptive therapies for patients and to create a truly special place to come to work each day.

Brad joins Be Bio from FerGene where he served as the Chief People Officer leading the rapid expansion of the company in support of a first product commercialization effort for a novel gene therapy. He has spent the past 15 years building small to mid-sized biotech companies from early research stages through commercialization and has played a significant role in launching multiple innovative therapies, including Kalydeco (cystic fibrosis), Kalbitor (hereditary angioedema), and Incivek (hepatitis C), which was one of the fastest blockbuster drug launches of all time. He also founded and built out his own Executive Search firm, ConnectedSearch. Brad received his BS in Neuroscience from the University of Rochester and worked at both the National Cancer Institute and the Graduate School of Pharmacology at the University of Rochester in cellular pharmacology.

There is a deeply held belief at Be Bio in the paramount importance of people, culture, and service to patients, to each other, and to the community, said Mr. Hartman. I am equally awestruck by the enormous potential of this B cell platform to transform patient lives across a wide variety of serious, life-threatening diseases as I am by this teams character, heart, and values. I am incredibly honored to join this distinguished team in building a remarkable place to work together and endeavoring to reshape the lives of so many patients and their families.

About Be Biopharma

Be Biopharma is a leader in developing B cells as medicines, treating disease with the human bodys native protein factories. We precisely engineer B cells to harness their intrinsic drug-like properties remarkable protein production, selective tissue targeting, and fine control of their cellular environment to forge a new category of cell therapy. These medicines are designed to be durable, allogeneic, re-dosable, and administered without toxic conditioning, creating new avenues to halt or reverse severe diseases like cancer, autoimmune conditions, and enzyme deficiency. Founded by Longwood Fund and B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., Be Biopharma is re-imagining medicine based on the power of B cell therapy. Be Bio was founded in October 2020 by Longwood Fund with a $52 million Series A investment led by Atlas Ventures and RA Capital, joined by Alta Partners and Takeda Ventures. For more information, please visit Be Biopharma.

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Be Bio Appoints Leading Cell & Gene Therapy Executives, Krishnan Viswanadhan as President & Chief Operating Officer, and Brad Hartman as Chief...

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SIOX - Market Data & News

Sio Gene Therapies Inc (NASDAQ: SIOX), a 11 12 St Jamess Square, London, company, fell to close at $1.95 Monday after losing $0.08 (3.94%) on volume of 431,354 shares. The stock ranged from a high of $2.02 to a low of $1.94 while Sio Gene Therapiess market cap now stands at $142,235,939.

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Its current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. The company is also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson's disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Visit Sio Gene Therapies Incs profile for more information.

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Sio Gene Therapies (SIOX) falls 3.94% for August 16 - Equities.com

PHILADELPHIAToo many exhausted T cells left in the wake of aggressive chemotherapy regimens for patients with advanced chronic lymphocytic leukemia (CLL) make it more challenging for chimeric antigen receptor (CAR) T cell therapy to do its job. Now, a new study from researchers in the Perelman School of Medicine at the University of Pennsylvania shows how to overcome this type of resistance and reinvigorate these T cells with an experimental small molecule inhibitor.

Reporting online today in the Journal of Clinical Investigation, the team shows how the drug, known as JQ1, improved CAR T cell function by inhibiting what is known as the bromodomain and extra terminal (BET) proteins. BET, the researchers showed, can disrupt CAR expression and key acetylated histone functions in T cells in CLL.

The findings demonstrate, for the first time, this mechanism of resistance and present a much-needed target for CLL when treating patients with cellular therapies like CAR. Only a small subset of patients with advanced CLL respond to CAR T cell therapycompared to 80 percent of acute lymphocytic leukemia patients with advanced disease.

Why CAR T cells fail to fully attack cancer cells in so many CLL patients is an important question that needs to be answered in order to expand the use of these immunotherapies in CLL and other cancers, said senior author Joseph A. Fraietta, PhD, an assistant professor of Microbiology at Penn, and member of the Center for Cellular Immunotherapies. Treating these war weary T cells during the CAR T cell engineering process has the potential to boost responses, weve shown here. Its setting the stage for a very promising set of next steps that rationalize further studies, including clinical trials, to prove this approach is safe and feasible.

Using the small molecule inhibitor and the T cells and CD19 CAR T cells from multiple previously treated patients, the researchers demonstrated that the BET protein plays a role in downregulating CAR expression, and that, if blocked, can diminish CAR cell T cell exhaustion and increase the production of CAR T cells from CLL patients with poor lymphocytes.

Treatment with JQ1 also increased levels of various immunoregulatory cytokines and chemokines previously reported to be produced by CAR T cells in CLL during successful therapy. The array of native immune and CAR cells mirrored those found more typically in patients who do respond.

Given this observed reinvigoration of dysfunctional CLL patient CAR T cells by BET inhibition, the authors suggest that incorporating JQ1 into cellular engineering and expansion processes could lead to a generation of less defective and more potent final CAR T cells for patients.

To what extent the above pathways contribute to the effects of JQ1 on CAR T cells is a focus of ongoing investigations for the research group.

This work shows us that T cells can be taught new tricks, said Bruce Levine, PhD, the Barbara and Edward Netter Professor in Cancer Gene Therapy in Penns Perelman School of Medicine, and co-author on the study.That is to say that the methods of manufacturing can be adapted to improve CAR T cell function, so that what would have been exhausted or dysfunctional cells can now be reinvigorated, and potentially lead to better clinical responses in more patients than before.

This work was supported by the Bob Levis Funding Group, along with the National Institute of Allergy and Infectious Diseases (T32 AI007632), National Cancer Institute (P01 CA214278 575, R01 CA241762 U54 CA244711 576, P30 CA016520-44S3, and P30 CA016520-44S4), National Institute on Aging (U01 AG066100), the National Institute of General Medical Sciences (R01 GM118501), an Emerging Cancer Informatics Center of Excellence award from the Penn Institute for Biomedical Informatics and Abramson Cancer Center,Gabrielles Angel Foundation, an Alliance for Cancer Gene Therapy Investigator Award in Cell and Gene Therapy for Cancer, and Novartis.

Editors note: Fraietta is a co-founder of DeCART Therapeutics, Inc. and Levine is a co-founder of Tmunity Therapeutics, Inc. The University of Pennsylvania has licensed certain study-related technologies to Novartis. Penn and the inventors of these technologies receive significant financial benefits as a result of this licensing relationship with Novartis.

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.9 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $496 million awarded in the 2020 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 44,000 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2020, Penn Medicine provided more than $563 million to benefit our community.

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Existing Drug May Help Improve Responses to Cellular Therapies in Advanced Leukemias - pennmedicine.org

On track for three programs in the clinic by year end; trial site initiation for FLT180a for Hemophilia B and FLT201 for Gaucher disease Type 1 expected by year end; program for FLT190 for Fabry disease progressing

Completed first dose cohort for FLT190 Phase 1/2 dose-finding trial and advancing to next dose level; data presented by year end

Updated durability data from FLT180a Phase1/2 dose-finding trial presented by year end

Defined Company strategic priorities to accelerate value creation; near-term efficiencies extend cash runway into first quarter of 2023

LONDON, Aug. 16, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that Michael J. Parini has succeeded Theresa Heggie as Chief Executive Officer (CEO). The Company also reported financial results for the second quarter of 2021 and provided updates on its lead program FLT180a for Hemophilia B, FLT190 for Fabry disease, FLT201 for Gaucher disease Type 1 and FLT210 for Hemophilia A.

Today we are pleased to announce that Freelines Board of Directors has appointed Michael J. Parini as CEO and Executive Director of the Company, effective immediately, said Dr. Chris Hollowood, Chairman of Freeline. Michael has a long track record of building strong executive leadership teams and has had a significant positive impact on Freeline since he joined the Company from Vertex Pharmaceuticals. In a short period of time, he has mobilized program execution across our pipeline, increased focus on our core scientific and platform technology capabilities, and driven efficiencies across the organization that have extended our cash runway. In doing so, Michael has demonstrated his ability to unlock value at Freeline and deliver on the promise of its therapies and science to patients.

Dr. Hollowood continued, I wish to thank Theresa for leading the Company through the successful completion of its initial public offering last year and the extraordinary challenges of the COVID-19 pandemic. We wish her well in her future endeavors.

Freeline is at the forefront of gene therapy, leveraging a platform innovation engine that holds the potential to deliver functional cures to patients who suffer from debilitating diseases, said Mr. Parini, CEO of Freeline. I joined Freeline to deliver on the differentiated promise of our pipeline and technology, and am honored and excited to take the helm at this critical time for the Company.

Mr. Parini continued, I see three immediate strategic priorities as we turn the page on a new chapter for the Company. First and foremost is our commitment to advancing our pipeline with urgency for patients. Our recent execution in the clinic is a testament to this commitment, and we remain on track to deliver all expected data and developmental milestones across our four clinical and pre-clinical stage gene therapy programs before year end.

Our second strategic priority is to drive increased focus, financial discipline and operational efficiency in our business. Based on a thorough review of our operations, we have identified initial savings to extend our cash runway by nearly two quarters into the first quarter of 2023, funding operations through multiple value-generating milestones. We will continue this work to optimize our business, operational and platform strategy.

Finally, our third priority is to unlock the full value in our proprietary scientific and platform technology. We are refreshing our scientific strategy to leverage our unique combination of manufacturing quality, capsid potency and protein engineering capabilities, which we believe can drive lower and safer doses for high therapeutic effect, into the next wave of pipeline programs. We plan to focus our research and platform efforts where our differentiated capabilities allow us to address unmet needs and create transformative treatments for patients.

Mr. Parini added, We believe gene therapy holds tremendous promise. With our three strategic priorities in place, I look forward to building a world-class team and organization to realize the full potential of Freeline.

Key Pipeline and Operational Updates

Hemophilia B

Fabry Disease

Gaucher Disease (Type 1)

Hemophilia A

Platform Technology

Corporate

Q2 2021 Financial Highlights

Cash Position: Cash and cash equivalents were $164.7million as of June 30, 2021, as compared to $230.0million as of December 31, 2020. Based on the Companys revised operating plan, Freeline expects that its current level of cash and cash equivalents will enable the Company to fund its operating expenses into the first quarter of 2023.

R&D Expenses: Research and development (R&D) expenses for the six months ended June 30, 2021 were $48.1million, as compared to $29.4million for the same period in 2020. The increase of $18.7million was driven by increased investment in activities related to the current and proposed clinical trials for FLT201 and FLT210 and overall research and development, which includes earlier pipeline programs and further development of the Freeline platform.

G&A Expenses: General and administrative (G&A) expenses for the six months ended June 30, 2021 were $24.6million, as compared to $11.7million for the same period in 2020. The increase of $12.9million was driven primarily by an increase in personnel expenses, primarily due to an increase in headcount in corporate, legal, general and administrative functions to support the Companys growth initiatives and public company requirements, as well as an increase in non-cash share-based compensation expense, primarily due to equity grants to employees related to the completion of the Series C financing and the IPO.

As of June 30, 2021, the Company had 35,802,840 ordinary shares outstanding.

About Michael J. Parini

Michael Parini joined Freeline in March of 2021 to serve as the Companys President and Chief Operating Officer. Before coming to Freeline, Mr. Parini was the Executive Vice President and Chief Administrative, Legal and Business Development Officer at Vertex Pharmaceuticals, Inc. from March 2020 until February 2021, among other roles he held in his over five years with the company. Prior to Vertex, Michael Parini spent over a decade at Pfizer Inc. serving in multiple leadership roles within the company's global legal team, including Senior Vice President and Associate General Counsel.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing protein into the patients bloodstream. The Companys integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in HemophiliaB and Fabry disease, as well as preclinical programs in Gaucher disease Type 1 and HemophiliaA. Freeline is headquartered in the UK and has operations in Germany and the US.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include, among other topics, discussion of the Companys strategies, anticipated operating and financial performance and financial condition, including its review of strategy and operations; the Companys expectations regarding its use of cash and cash runway; statements regarding the initiation, timing, progress and results of the Companys preclinical studies and clinical trials, including the initiation and full enrollment of the Phase1/2 dose confirmation trial for FLT180a and data readouts from that trial, progress with respect to the dose-escalation for the Phase1/2 dose-finding clinical trial of FLT190 and data readouts from that trial, commencement of the Phase 1/2 dose-finding clinical trial of FLT201, and completion of pre-clinical IND/CTA-enabling studies of FLT210; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Companys product candidates. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; the recent departures of a number of executive officers of the Company, and the Companys ability to fill open positions, implement an orderly transition process and retain key talent; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on theSECwebsite atwww.sec.gov.

Contact

David S. ArringtonVice President Investor Relations & Corporate CommunicationsFreeline Therapeuticsdavid.arrington@freeline.life+1 (646) 668 6947

Freeline Therapeutics Holdings plcUnaudited Condensed Consolidated Statements of Operations Data(in thousands of U.S. dollars, except per share data)

Freeline Therapeutics Holdings plcUnaudited Condensed Consolidated Balance Sheet Data(in thousands of U.S. dollars, except per share data)

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Freeline Appoints Michael J. Parini as Chief Executive Officer and Reports Second Quarter 2021 Financial Results - GlobeNewswire

NOVATO, Calif., Aug. 04, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President will participate in a panel titled Miss Con-GENE-iality - Updates in Gene Tx on Wednesday, August 11, 2021 at the Wedbush PacGrow Healthcare Conference at 12:00 PM ET.

The live and archived webcast of the presentation will be accessible from the companys website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: http://www.ultragenyx.com.

Contact Ultragenyx Pharmaceutical Inc.Investors & MediaJoshua Higa415-475-6370

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Ultragenyx to Participate in Gene Therapy Panel at Wedbush PacGrow Healthcare Conference - Yahoo Finance

According to ResearchAndMarkets.com's new report, Cell And Gene Therapy Manufacturing Market Size, Share & Trends Analysis Report by Therapy Type, by Scale (R&D, Commercial), by Mode, by Workflow (Vector Production, Cell Banking), by Region, and Segment Forecasts, 2021 - 2028", the global cell and gene therapy manufacturing market size is expected to reach $57.4 billion by 2028.The market is estimated to grow at a CAGR of 20.3% from 2021 to 2028 due to an exponential rise in clinical pipeline along with a rising number of regulatory approvals for advanced therapies.With this growth are promising opportunities for contract development of cell and gene therapies. Sponsors are forming strategic alliances with contract manufacturers to help accelerate R&D of their candidate programs, increasing demand for CMOs/CDMOs services.Several new methods are being introduced to help advance cell and gene therapy manufacturing, including the potential of single-use technology in production workflows. This method is increasingly gaining attention to help speed the development process while reducing the overall cost and production timeline. Such technological advancements are expected to further drive market growth over the next few years.

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Global Cell & Gene Therapy Mfg. Market Could Reach $57.4B By 2028 - Contract Pharma

As of late June 2021, it was announced that researchers have effectively treated a genetic disorder inside humans using CRISPR therapy placed into patients bloodstreams. CRISPR is an acronym for clustered regularly interspaced short palindromic repeats, a naturally-occurring genome editing system in bacteria, repurposed by scientists.

The news has made shockwaves around the medical field as the technology has the potential to dramatically improve human health.

Genetic mutations account for more than 6000 human diseases. Curing these have been extremely difficult and expensive, and not all diseases are curable with even the most recent advancements within gene therapy.

Currently, there are two main ways gene therapy is conducted: somatic cell gene editing and germ-line gene editing.

Somatic cell gene editing involves editing matured cells within a human body. This kind of editing would not become hereditary, but does cure diseases for the afflicted individual. This is the only method to alter cells within a developed human body.

Germ-line gene editing involves editing cells such as sperm, eggs or embryos. This technique would allow any genetic modifications to be passed down to any future generations. This means that the editing process only needs to occur among a few independent cells, rather than trillions of cells found within an adult human.

Before the recent breakthrough in gene editing, CRISPR was used by removing affected cells from a patient, editing out the mutations in a lab and placing them back into the body to replicate. An example of this is curing sickle cell anemia through editing and infusing bone marrow cells.

The alternative method is to use a process known as Adeno Associated Virus, or AAV gene therapy. This method actually takes an artificially created virus with the healthy genes implanted inside, so that the virus itself infects and delivers the healthy genes to a patients cells. This is the current method for curing diseases such as Spinal Muscular Atrophy.

What makes this new advancement in CRISPR technology so novel is the method of deployment. This process injects CRISPR therapy directly into the bloodstream, so that it can make edits directly to the affected cells without invasive surgery or using AAV gene therapy.

The difficulty with direct CRISPR therapy inserted into the bloodstream has been attempting to figure out how CRISPR can correctly target and edit only the affected cells necessary. This new medical trial was successfully able to inject CRISPR into the patients bloodstream to target and edit the affected cells in their body from a genetic disorder, paving way to what could be an entirely new process to cure genetic diseases.

The ability to genetically edit human individuals is at the core of ethics within the medical industries, and, while the recent advancement is something to be celebrated, there are concerns about approaches to ethical gene editing and deploying such technology in the right hands for the right reasons.

One end of an extreme involved a scandal involving He Jiankui, a Chinese researcher who made claims of a successful birth with the first gene-edited twin girls. He has since been jailed and received international condemnation for the dangerous and unethical research experiment.

Another concern involving gene editing revolves around self-proclaimed biohackers, individuals without prior educational experience who perform genetic editing techniques either on themselves or on other living species, such as plants.

Both situations signify the most important question to consider with gene editing: regulation of use.

Dr. Bryan Cwik, a bioethics philosophy professor at Portland State University, spoke with Vanguard to discuss the ways scientists can approach gene editing in an ethical way.

The first thing to ask is, which ways work best? Cwik said. Are there advantages or disadvantages when treating this class of disease? These are ways to think about responsible gene editing.

Advancements in gene therapy are still extremely elementary, and cannot cure all types of genetic diseases as we currently understand them. Cwik explains that diseases with no deterministic link and complex etiologies should not be considered for this kind of research.

Deterministic link is a technical term used by the medical industry to explain the types of connections certain genes have with diseases. Spinal Muscular Atrophy is an example of a disease with a deterministic link; if an individual has a specific genome inside their cells, they will have the disease. These types of genetic disorders are ones suited for current advancements within gene therapy treatments.

Other diseases, however, have complex ways with which they might be constructed in the body, or the ways in which a disorder is afflicted within an individual, known as etiology. Schizophrenia is a prime example of a disease that has genetic roots, but has complicated factors; scientists dont fully understand what triggers it.

These advancements mean great news for medicine and individuals who suffer from genetic disorders such as Huntingtons disease. Others still require much more research before enough can be done. If we are to advance in gene therapies, we must be extremely cautious as to how research should be continuously conducted, and who has access to tools that constitute gene therapies.

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CRISPR advancements and the ethics of gene editing - Vanguard - Psuvanguard.com

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TSHA - Market Data & News

Today, Taysha Gene Therapies Inc Incs (NASDAQ: TSHA) stock fell $0.36, accounting for a 2.00% decrease. Taysha Gene Therapies opened at $17.77 before trading between $18.11 and $17.38 throughout Wednesdays session. The activity saw Taysha Gene Therapiess market cap fall to $668,485,049 on 76,140 shares -below their 30-day average of 164,130.

Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, Taysha aims to rapidly translate our treatments from bench to bedside. Taysha Gene Therapies has combined its teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, Taysha Gene Therapies leverages its fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives.

Visit Taysha Gene Therapies Inc's profile for more information.

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UMass Medical School researchers Guangping Gao, PhD, and Dan Wang, PhD, are working with ASC Therapeutics, a privately held biopharmaceutical company developing in-vivo gene replacement, gene editing and allogeneic cell therapies, to bring a gene therapy for maple syrup urine disease (MSUD) to the clinic.

Our significant progress developing an AAV-based therapy for MSUD will strongly benefit from this partnership by accelerating the progression of this much-needed treatment into the clinic. ASC Therapeutics IND clearance by the FDA of a novel gene therapy for hemophilia A confirms their excellence, commitment and expertise in translating preclinical discoveries into clinical programs, said Dr. Gao, thePenelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research.

Maple syrup urine disease is a rare genetic disorder affecting degradation of the branched-chain amino acids (BCAA) leucine, isoleucine and valine and their ketoacid derivatives. MSUD is caused when a mutated form of the BCKDHA, BCKDHB or DBT gene is inherited from both parents. As a result of this mutation, the body is unable to breakdown certain parts of proteins. This leads to the build-up of toxic substances that can cause organ and brain damage. There are several forms of MSUD. The most common is the classic or infantile form. Symptoms of the classic form of MSUD start in early infancy and include poor feeding, irritability, extra sleepiness and muscle spasms. If untreated, respiratory failure may occur. The symptoms of other forms of MSUD start in adolescence or adulthood.

A protein restricted diet and supplements are the mainstay of treatment but have insufficient efficacy and afford no protection against episodic and life-threatening encephalopathic crises.

In some cases, liver transplantation may be considered and is considered a cure.

Often diagnosed based on the results of a newborn screening test, MSUD gets its name because the urine of infants with MSUD smells like maple syrup.

Under the agreement, the research group at UMMS, led by Gao and Dr. Wang, assistant professor of RNA therapeutics, will develop multiple animal models for MSUD, as well as conduct pre-clinical testing using such animal models and proprietary AAV constructs. ASC Therapeutics receives an exclusive option on the selected constructs and intellectual property resulting from this partnership. ASC Therapeutics and UMMS will jointly conduct further IND-enabling studies, regulatory and manufacturing activities to obtain IND clearance and advance this program into clinical stage.

We look forward to working with the UMMS team led by Dr. Gao and Dr. Wang, leading experts for the development and application of AAV technology. This partnership solidifies our gene therapy pipeline and will provide novel genetic therapies to patients with severe unmet medical needs worldwide, said Ruhong Jiang, PhD, CEO for ASC Therapeutics.

Related stories on UMassMed News:UMMS establishes gene therapy collaborative research agreement with PfizerH-ABC Foundation partners with UMass Medical School and Yale to study gene therapy for H-ABCGuangping Gao makes list of Nature Biotechnology Top 20 translational researchers

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Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease - UMass Medical School