Archive for the ‘Gene Therapy Research’ Category

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.

Effective gene therapy targeting MECP2 for the treatment of Rett syndrome has been elusive due to the inability to properly regulate transgene expression, said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. The built-in self-regulatory feedback loop mechanism in TSHA-102, work that was initiated in my laboratory in 2007, is a completely novel approach that allows for regulated expression of MECP2 on a cell-by-cell basis. These results published today are highly encouraging and allow us to conceive additional novel approaches using miRARE in conditions with dose-sensitive genes.

The complexities of developing an efficacious and well-tolerated gene therapy for the treatment of Rett syndrome are highlighted by phenotypic variability, mosaicism and the need to regulate MECP2 such that it does not cause overexpression-related toxicity. Todays data give us confidence that we can achieve appropriate MECP2 expression in all cells in a genotype-dependent manner, which we believe significantly de-risks the developmental program in its translation to humans, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. Historically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein. With the built-in regulatory element, miRARE, TSHA-102 provided a statistically significant survival extension by 56% in 4- to 5-week-old knockout Rett mice. We see the potential for broadening the miRARE platform to other CNS diseases requiring regulated gene expression. These positive data support our intent to file an IND/CTA for TSHA-102 in the second half of this year, followed by initiation of a Phase 1/2 trial by year-end 2021. TSHA-102 has the potential to address a significant unmet need for an estimated 25,000 patient with Rett syndrome across the United States and in Europe.

The preclinical study was conducted by the UT Southwestern Medical Center (UT Southwestern) laboratory of Sarah Sinnett, Ph.D., and evaluated the safety and efficacy of regulated miniMECP2 gene transfer, TSHA-102 (AAV9/miniMECP2-miRARE), via intrathecal (IT) administration in adolescent mice between four and five weeks of age. TSHA-102 was compared to unregulated full length MECP2 (AAV9/MECP2) and unregulated miniMECP2 (AAV9/miniMECP2).

TSHA-102 extended knockout survival by 56% via IT delivery. In contrast, the unregulated miniMECP2 gene transfer failed to significantly extend knockout survival at either dose tested. Additionally, the unregulated full-length MECP2 construct did not demonstrate a significant extension in survival and was associated with an unacceptable toxicity profile in wild type mice.

In addition to survival, behavioral side effects were explored. Mice were subjected to phenotypic scoring and a battery of tests including gait, hindlimb clasping, tremor and others to comprise an aggregate behavioral score. miRARE attenuated MECP2-mediated aggravation in wild type aggregate phenotype severity scores. Mice were scored on an aggregate severity scale using an established protocol. AAV9/MECP2- and AAV9/miniMECP2-treated wild type mice had a significantly higher mean (worse) aggregate behavioral severity score versus that observed for saline-treated mice (p <0.05; at 630 and 727 weeks of age, respectively). TSHA-102-treated wild type mice had a significantly lower (better) mean aggregate severity score versus those of AAV9/MECP2- and AAV9/miniMECP2-treated mice at most timepoints from 1119 and 920 weeks of age, respectively. No significant difference was observed between saline- and TSHA-102-treated wild type mice.

Of note, miRARE-mediated genotype-dependent gene regulation was demonstrated by analyzing tissue sections from wild type and knockout mice treated with AAV9 vectors given intrathecally. TSHA-102 demonstrated regulated MECP2 expression in different regions of the brain. In the pons and midbrain, miRARE inhibited mean MECP2 gene expression in a genotype-dependent manner as indicated by significantly fewer myc(+) cells observed in wild type mice compared to knockout mice (p<0.05), thereby demonstrating that TSHA-102 achieved MECP2 expression levels similar to normal physiological parameters.

It has been a challenge finding an approach that can appropriately regulate MECP2 expression in Rett syndrome but the preclinical data for TSHA-102 published today support the miRARE approach, said Sarah Sinnett, Ph.D., Assistant Professor in the Department of Pediatrics at UT Southwestern. It is clear that the disease is reversible, and I am encouraged that this novel strategy may enable us to make a difference in the management of this disease.

The publication is available by clicking on the following link: https://academic.oup.com/brain/advance-article-abstract/doi/10.1093/brain/awab182/6265600.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our 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, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Drs. Gray and Sinnett have intellectual property interest in Taysha and UTSW has a financial interest in the company.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science...

NEW YORK, and RESEARCH TRIANGLE PARK, N.C., April 28, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021.

The AXO-AAV-GM1 presentation will include a review of patient-level data on safety and efficacy at 6 months follow up from the low-dose cohort of the Companys ongoing clinical study. Additionally, Dr. Cynthia Tifft, the lead investigator for the study, will present 6-month biomarker data from cerebrospinal fluid (CSF) in the 5 children who received intravenous AAV9 gene therapy.

Oral Presentation Details:

Presentation Title: AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase I-II trialAbstract Number: 162Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Cynthia Tifft, MD, PhD, Deputy Clinical Director, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 6:15 PM 6:30 PM EDT

Presentation Title: AXO-Lenti-PD gene therapy for Parkinsons disease: efficacy, safety, and tolerability data from the second cohort in open-label dose evaluation study SUNRISE-PD at 6 months post administrationAbstract Number: 163Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Gavin Corcoran, MD, Chief R&D Officer Presentation Date and Time: Thursday, May 13, 2021 6:30 PM 6:45 PM EDT

Presentation Title: Immune Modulation Preceding AAV9-GLB1 Gene Therapy Preserves the Possibility for Re-Dosing in Children with GM1 GangliosidosisAbstract Number: 179Session: Immunotherapy and VaccinesPresenting Author: Precilla DSouza, DNP, MSN, CRNP, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 7:00 PM 7:15 PM EDT

Presentation Title: A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow RateAbstract Number: 256Session: Pharmacology/Toxicology Studies or Assay DevelopmentPresenting Author: Thomas Pack, PhD, Sio Gene TherapiesPresentation Date and Time: Friday, May 14, 2021 from 1:45 PM 2:00 PM EDT

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFood and Drug Administrationand is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

In 2018, Sio licensed exclusive worldwide rights from theUniversity of Massachusetts Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About AXO-Lenti-PDAXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinsons disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration.Axovantexpects to dose the first patient in EXPLORE-PD, a randomized, sham controlled study in 2021.

AboutSio Gene TherapiesSio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our 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. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons 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. For more information, visitwww.siogtx.com.

Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as believe, "estimate," may be and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing of its upcoming clinical milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for Axo-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononFebruary 9, 2021, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officer investors@siogtx.com

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Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

BOSTON--(BUSINESS WIRE)--SOLA Biosciences, LLC, today announced positive preclinical data in mice from a study designed to evaluate the efficacy of SOL-257, an innovative gene therapy candidate selectively targeting pathogenic TDP-43, which is found in approximately 97% of ALS patients. The data was presented during the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular Disease on April 29, 2021, by Akinori Hishiya, Ph.D., Founder/CSO of SOLA.

SOLA has developed an innovative chaperone technology (JUMP70) to selectively eliminate disease-causing misfolded proteins using the patients' own chaperones. SOLA has developed nine gene therapy candidates incorporating JUMP70 to address conformational diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington's disease, Parkinson's disease, and Alzheimer's disease. These candidates demonstrated preclinical activities and safety to prove the mechanism of the JUMP70 technology. SOL-257 is a flagship compound to selectively target misfolded and neurotoxic TDP-43 proteins without affecting healthy TDP-43.

At the conference, SOLA presented unprecedented in vivo efficacy data using the NEFH-hTDP-43NLS mouse model, which expresses a doxycycline-repressible form of human TDP-43 lacking a nuclear localization signal (NLS). The transgenic mice accumulate cytoplasmic insoluble TDP-43 in neurons of the brain and spinal cord soon after pathogenic TDP-43 proteins are expressed. The mice develop many features reminiscent of ALS, including motor deficits, denervation of neuromuscular junctions, motor neuron loss, and rapid loss in viability. The mice treated with control gene therapy (placebo) started dying 3 weeks after pathogenic TDP-43 expression, and by 5 weeks, 100% of male mice died. In contrast, all male mice treated with SOL-257 gene therapy survived.

"Now we know many diseases such as ALS are caused with protein folding issues. Repairing or removing the misfolded disease-causing proteins can be a fundamental treatment for the patients, although the treatment has not been realized yet. We learned the natural protein quality control system and carefully designed our novel therapeutics," says Dr. Akinori Hishiya. "Our designed therapeutic enables highly specific intervention, only misfolded disease-causing proteins can be targeted and removed."

"SOL-257 targeting only pathogenic TDP-43 to repair the specific problem has a significant potential for a broadly applicable ALS treatment," says Keizo Koya, Ph.D., Founder/CEO of SOLA. "I hope this groundbreaking animal data of SOL-257 will bring new hope for patients with ALS.

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SOLA Biosciences Presented Positive ALS Animal Efficacy Data of SOL-257 at the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular...

DUBLIN--(BUSINESS WIRE)--The "Cell & Gene Therapy Technologies and Supplies" report has been added to ResearchAndMarkets.com's offering.

Cell and gene therapy (CGT) is a rapidly evolving field producing powerful new treatments for cancers and genetic diseases, and expanding into autoimmune disease, cardiovascular disease, musculoskeletal disease, and many others. The CGT field has generated great interest and hope among researchers, patient groups, and regulators, which has led to huge investments in R&D.

Globally, many governments and regulatory bodies have adopted policy and patent environments supportive of CGT development. Since CGTs often target rare and underserved disease areas for which few other therapeutics exist, there is a sense of urgency within the pharma/bio space to develop CGTs.

As development and market entries of CGTs accelerate, the market for suppliers of laboratory and clinical tools within the CGT R&D and manufacturing spaces will see very rapid growth. The Cell & Gene Therapy Technologies and Supplies examines the global market for analytical technologies and products used throughout the various stages of CGT development and manufacturing, evaluating 21 technologies grouped into six categories.

The goal of this report is to provide demand growth projections by technique, region, and function, while also providing comprehensive views of the competitive landscape for each technology.

Report Overview

Participants include the following:

Key Topics Covered:

1. Introduction

2. Market Insights

3. Market Demand

3.1 Overall Demand

3.2 General Techniques

3.3 Gene Therapy Development

3.4 Transduction and Transfection

3.5 Cell Enrichment

3.6 Cell Culture and Cell Expansion

3.7 Market Demand by Technique (2020)

3.8 Cryopreservation

For more information about this report visit https://www.researchandmarkets.com/r/hrs0we

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Cell & Gene Therapy Technologies and Supplies Report 2021: Market Demand and Five-year Forecasts, Segmented by Technique, Region and Function -...

Xerostomia, a lack of saliva production leading to dry mouth, has a variety of causes, including radiotherapy and chemotherapy, the chronic use of drugs and rheumatic and dysmetabolic diseases. It is also a common side effect of ionizing radiation used to treat head and neck cancer.1

A proof of concept Phase 1 clinical trial sponsored by a third party showed that Aquaporin-1 (AQP1), a trans-membrane protein that facilitates water movement across lipid layers, restored saliva flow in a human population using an adenovirus-based vector encoding AQP1 to a single previously irradiated parotid gland.2 The results from this study led AHN to research using UAGT technology targeting the salivary gland, which combines the use of nonviral DNA vector and lipid microbubbles with a low-frequency acoustic field to create a sonoporation effect allowing gene transfer to the cells of the salivary gland without the introduction of viral antigens.3

According to Mark Trombetta, MD, Director of Clinical Program Development for the AHN Cancer Institute, a radiation oncologist and co-investigator in the networks study of UAGT, thousands of cancer patients suffer from radiation-induced xerostomia, which can cause severe tooth decay, chronic oral pain, loss of taste, inability to eat properly and increased oral infections. Once xerostomia begins, it is a permanent condition.

We believe that UAGT may provide long-term relief of radiation-induced xerostomia, with adjustable dosing and potential for booster doses over time, said Warren Swegal, MD, an AHN head and neck surgeon and clinical lead of the networks UAGT program. No other existing treatment offers a long-lasting solution for this life-altering condition, and we are thrilled to be able to further develop and improve the therapy by leveraging Lantheus proven microbubble technology.

We are excited to support AHN in its efforts to progress this innovative development program. Xerostomia is a chronic and debilitating condition with limited treatment options for patients, said Mary Anne Heino, President and Chief Executive Officer of Lantheus. We believe our microbubble combined with AHNs UAGT technology have the potential to make a difference in the lives of these patients.

Lantheus will supply its microbubbles and activation devices to AHN. AHN will be solely responsible for all development work, future regulatory submissions and commercialization. The deal terms include a transfer price and royalties.

About Lantheus Holdings, Inc.

Lantheus Holdings, Inc. is the parent company of Lantheus Medical Imaging, Inc., Progenics Pharmaceuticals, Inc. and EXINI Diagnostics AB and an established leader and fully integrated provider of innovative imaging diagnostics, targeted therapeutics and artificial intelligence solutions to Find Fight and Follow serious medical conditions. Lantheus provides a broad portfolio of products, including the echocardiography agent DEFINITY Vial for (Perflutren Lipid Microsphere) Injectable Suspension; TechneLite (Technetium Tc99m Generator), a technetium-based generator that provides the essential medical isotope used in nuclear medicine procedures; AZEDRA for the treatment of certain rare neuroendocrine tumors; and RELISTOR for the treatment of opioid-induced constipation, which is partnered with Bausch Health Companies, Inc. The Company is headquartered in North Billerica, Massachusetts with offices in New York, New Jersey, Canada and Sweden. For more information, please visit http://www.lantheus.com.

About Allegheny Health Network

Allegheny Health Network (AHN.org) is an integrated healthcare delivery system serving the greater Western Pennsylvania region. The Network is composed of 13 hospitals, ambulatory surgery centers, Health + Wellness Pavilions, an employed physician organization, home and community-based health services, a research institute and a group purchasing organization. The Network provides patients with access to a complete spectrum of advanced medical services, including nationally recognized programs for primary and emergency care, cardiovascular disease, cancer care, orthopedic surgery, neurology and neurosurgery, womens health, autoimmune diseases, diabetes and more. AHN employs approximately 21,000 people, has more than 2,600 physicians on its medical staff and serves as a clinical campus for Drexel University College of Medicine and the Lake Erie College of Osteopathic Medicine.

Safe Harbor for Forward-Looking and Cautionary Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, that are subject to risks and uncertainties and are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by their use of terms such as anticipate, believe, confident, could, estimate, expect, intend, may, plan, predict, potential, project, target, will and other similar terms. Such forward-looking statements are based upon current plans, estimates and expectations that are subject to risks and uncertainties that could cause actual results to materially differ from those described in the forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation that such plans, estimates and expectations will be achieved. Readers are cautioned not to place undue reliance on the forward-looking statements contained herein, which speak only as of the date hereof. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. Risks and uncertainties that could cause our actual results to materially differ from those described in the forward-looking statements include (i) expectations for future clinical trials, the timing and potential outcomes of clinical studies and filings and other interactions with regulatory authorities; (ii) the impact of legislative, regulatory, competitive and technological changes; (iii) AHNs ability to successfully launch its UAGT technology with our microbubble as a commercial product; and (iv) the risk and uncertainties discussed in our filings with the Securities and Exchange Commission (including those described in the Risk Factors section in our Annual Reports on Form 10-K and our Quarterly Reports on Form 10-Q).

1Pinna R, Campus G, Cumbo E, Mura I, Milia E. Xerostomia induced by radiotherapy: an overview of the physiopathology, clinical evidence, and management of the oral damage. Ther Clin Risk Manag. 2015; 11:171-188.2https://clinicaltrials.gov/ct2/show/NCT02446249 3Wang Z, Zourelias L, Wu C, Edwards PC, Trombetta M, Passineau MJ. Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion. Gene Therapy. 2015; 22:739-749.

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Lantheus Grants Allegheny Health Network Exclusive Rights for the Use of its Microbubbles in Combination with Ultrasound Assisted Gene Therapy for the...

New fund to support regenerative medicine discoveries with high potential impact for patients

TORONTO and MISSISSAUGA, ON, April 29, 2021 /CNW/ - New regenerative medicine discoveries, including cell and gene therapies, will never reach patients without access to the funding, expertise and other specialized support that are required to move along the commercialization pathway. To address this need, CCRM, a leader in developing and commercializing regenerative medicine-based technologies and cell and gene therapies, and Amgen, a global leader in the biotechnology industry, are announcing a multi-year fund, for early-stage regenerative medicine-based technologies and therapies to benefit patients and the health-care system. The collaboration is made up of equivalent investments from CCRM and Amgen.

To bridge the gap from bench to bedside, the program will identify, develop and commercialize promising technologies and therapies arising from research conducted in institutions that form CCRM's global network. CCRM and Amgen's contributions will range from financial support to in-kind technical services and expertise.

"CCRM's collaboration with Amgen to create this fund is a perfect example of how public-private partnerships can leverage resources and expertise to support development and commercialization, and change patients' lives," said Michael May, President and CEO, CCRM. "The regenerative medicineresearch ecosystemacross Canada offers a rich discovery pipeline and it is ready for such a program."

"There are few places in the world that have clustered all the necessary resources and talent to drive regenerative medicine from the bench to the bedside. Canada has consistently led the way for decades," said Alan Russell, Vice-President, Research, Amgen Inc."Amgen is delighted to have the opportunity to partner with CCRM and leverage an extraordinary platform to benefit patients."

To identify opportunities, a Joint Steering Committee, made up of representatives from both CCRM and Amgen, will assess proposals from CCRM's Canadian and international member institutions. Selected projects will possess high scientific merit, be developed by scientists and academics with established credentials and expertise, and demonstrate the greatest potential to have an impact in the industry.

Regenerative medicine, including cell and gene therapy, harnesses the power of (stem) cells, biomaterials, molecules and genetic modification to repair, regenerate or replace diseased cells, tissues and organs. It has the promise of creating revolutionary new treatments for devastating and costly conditions such as heart disease, diabetes and cancer.

About CCRM CCRM is a global, public-private partnership headquartered in Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding, and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About Amgen in CanadaAs a leader in innovation, Amgen Canada understands the value of science. With main operations located in Mississauga, Ontario's vibrant biomedical cluster, and its research facility in Burnaby, B.C., Amgen Canada has been an important contributor to advancements in science and innovation in Canada since 1991. The company contributes to the development of new therapies and new uses for existing medicines in partnership with many of Canada's leading healthcare, academic, research, government and patient organizations. To learn more about Amgen Canada, visitwww.amgen.ca.

SOURCE Amgen Canada

For further information: CCRM: Stacey Johnson, Director, Communications and Marketing, 647-309-1830, [emailprotected]; Amgen Canada: Natasha Bond, Head of Corporate Affairs, 905-285-3007, [emailprotected]

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CCRM and Amgen partner to advance emerging medical innovations - Canada NewsWire

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data presentations at the upcoming 24thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators will review new data from Rockets Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi Anemia (FA) gene therapy programs in oral and poster presentations.

Details for oral presentations are as follows:

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim ResultsSession: Genetic Blood and Immune DisordersPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesDate: Tuesday May 11, 2021Time: 6:15-6:30 p.m. EDTLocation: Room 7Abstract number: 39

Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric PatientsSession: Gene Therapies for HemoglobinopathiesPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainDate: Wednesday May 12, 2021Time: 6:45-7:00 p.m. EDTLocation: Room 7Abstract number: 83

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene Therapy Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday, May 13, 2021Time: 10:00-10:45 a.m. EDTLocation: Room 7Abstract number: 36

Select results from Dr. Buerens presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene TherapyPresenter: Paula Ro, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday May 13, 2021Time: 10:00-11:45 a.m. EDT

Details for poster presentation are as follows:

Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical TrialsSession: Hematologic and Immunologic DiseasesPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineDate: Tuesday, May 11, 2021Time: 8:00-10:00 a.m. EDTLocation: Digital GalleryAbstract number: 697

Abstracts for the presentations can be found online at: https://annualmeeting.asgct.org/

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Originally posted here:
Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

LONDON, April 27, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that the Company will give six poster presentations at the American Society of Gene and Cell Therapy Annual Meeting 2021, taking place May 11 14.

We are excited to be presenting six posters at the upcoming ASGCT conference, which together highlight the scientific foundation that underlies our gene therapy programs and broader platform technology, said Theresa Heggie, CEO of Freeline. These posters are reflective of steady progress at Freeline, which we expect will include three gene therapies in the clinic by year end.

Poster Presentation Details

#329:FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1Presenter: RomualdCorbau, PhD, Chief Scientific Officer

#509:GLA Uptake and Metabolic Cross Correction in Fabry Disease Relevant Cell Lines: A Rationale for Liver-Directed AAV Gene TherapyPresenter:Jey Jeyakumar, PhD, Scientific Director

#821:Development of a 96-Well Plate-Based High-Throughput System forrAAVManufacturing Platform Optimization and Candidate SelectionPresenter: BettinaPrieler, PhD Student Technology Development

#843:Development and Scale Up of a Suspension Cell-Based AAV Manufacturing ProcessPresenter:Ahmed Youssef, Team Leader USP Development

#878:Defining a Reliable Quantification Assay Strategy for Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:FeliciaThoennissen, PhD, Scientist Analytical Development

#894:Development of an Assay to Measure Transduction Efficiency of Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:Anita Heinlein, Scientist Analytical Development

Abstracts will be available on the ASGCT website starting today.

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 Hemophilia B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. 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 discussion of the Companys research, pipeline and clinical trial plans. 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 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; the Companys ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt 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. 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 the U.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on the SEC website at http://www.sec.gov.

Contact

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

Continue reading here:
Freeline to Present Data at the American Society of Gene and Cell Therapy Annual Meeting 2021 - GlobeNewswire

CRISPR Technology Market: Introduction

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CRISPR Technology Market: Rise in focus on gene therapeutics is projected to contribute to the growth of the market - BioSpace

The exciting potential of immunotherapy for cancer treatment continues its exploration and here, Drug Target Review investigates three of the latest pre-clinical developments in immuno-oncology research.

Scientists at McMaster University, Canada, have shown in new research that changing the metabolism of natural killer (NK) immune cells allows them to overcome the hostile conditions found inside tumours and destroy advanced ovarian and lung cancer.

In this study, we discovered that the metabolism, or energy hub, of NK cells is paralysed by tumours, causing the NK cells to undergo an energy crisis and lose their tumour-killing functions, said Sophie Poznanski, a PhD student and lead author of the study. With that understanding, we were able to reverse the dysfunction of NK cells by repurposing a pre-existing metabolism drug that restored their energy production.

Published in Cell Metabolism, the study shows that NK cells can also be modified to mimic the metabolism of tumours. These modified NK cells proved to be far better adapted for the hostile tumour environment, the team found.

We were just hoping that the modified NK cells would better resist suppression in tumours. We were astounded to see that not only did they show no suppression, but they paradoxically functioned better inside of the tumour than outside of it, said Poznanski.

This is the first report of an antitumour immune cell that exploits the hostility of tumours for their own advantage, said senior author Professor Ali Ashkar. Generating cytotoxic immune cells to have tumour-like metabolism is key for their antitumour functions in a very hostile environment of a solid tumour. This could be a paradigm shift for immune cell-based cancer immunotherapy.

So far, NK cells have only proven effective against blood cancers. However, re-programmed and trained NK cells could afford patients with otherwise terminal cancers with a safe and effective treatment option. Furthermore, immunotherapy with NK cells has already proven safe with few, if any, side effects.

This research revealed a novel targeted immunotherapy approach that employs new antibodies against genetically altered proteins to target cancers. The technique was developed at the Johns Hopkins Kimmel Cancer Center, US.

The researchers targeted their immunotherapy approach to alterations in the common cancer-related p53 tumour suppressor gene, the RAS tumour-promoting oncogene or T-cell receptor genes. Their findings are reported in three related studies published inScience Immunology, Science and Science Translational Medicine.

Although common across cancer types, p53 mutations have thus far not been successfully targeted with drugs. Genetic alterations in tumour suppressor genes often result in their functional inactivation.

Traditional drugs are aimed at inhibiting proteins. Inhibiting an already inactivated tumour suppressor gene protein in cancer cells, therefore, is not a feasible approach, said Dr Emily Han-Chung Hsiue, lead author on theSciencepaper.

Instead of drugs, the researchers set out to target these gene alterations with antibodies. Conventional antibodies require an antigen target on the cell surface most commonly a protein that looks like a foreign invader to the immune system. However, the proteins produced by mutant oncogenes and tumour suppressor genes are inside the cells, out of reach from conventional antibodies. However, proteins are routinely degraded within cells, generating protein fragments called peptides.

These peptides can be presented on the cell surface when complexed with the human leukocyte antigen (HLA) proteins, saidKatharine Wright, postdoctoral fellow and a lead author on theScience Immunologypaper. Mutated proteins in cancer cells can also be degraded and generate mutant peptides presented by the HLA molecules. These mutant peptide HLA complexes serve as antigens and mark cancer cells as foreign to the immune system.

They developed an approach in the form of bispecific antibodies, comprising one component that specifically recognises cancer cells and another component that recognises immune cells and brings both cells together. In laboratory and animal tumour cell models, it resulted in the destruction of tumour cells.

This therapeutic strategy is dependent on a cancer containing at least one p53 or RAS alteration and the patient having an HLA type that will bind to the mutant peptide to present it on the cell surface, said senior authorAssociate Professor Shibin Zhou, a study leader.

In theScience Translational Medicinepaper, the researchers report that the powerful bispecific antibody approach they developed could also be used for the treatment of T-cell cancers. In animal models, the researchers showed that their approach selectively killed the cancerous T cells while sparing the majority of healthy T cells.

The scientists say the next research steps are to assess whether the strategy can be applied to other gene alterations in p53, KRAS and other cancer driver genes.

We intend to develop a large number of bispecific antibodies that would target such genes, said Alex Pearlman, PhD student and co-author of the three studies. Although any individual bispecific antibody would target a small fraction of cancer patients, a suite of antibodies would allow for the treatment of many patients.

Researchers at Moffitt Cancer Center, US, have investigated how to combine and sequence new therapies to improve survival of patients with advanced melanoma. In a new article published inCancer Immunology Research, the team demonstrated that sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses in pre-clinical models and may be a potential treatment option for patients.

One of the most common genetic alterations in melanoma are mutations of the BRAF gene, which affect approximately 50 percent of patients. These alterations result in downstream signalling through the protein MEK and stimulation of cell growth, invasion and survival. Several therapies that target both BRAF or MEK have been approved and standard treatment for patients who have BRAF mutations is now combination therapy with BRAF and MEK inhibitors.

Given the success of both BRAF/MEK inhibitors and immunotherapies, researchers have attempted to combine these two classes of anticancer therapies. However, some of these combinations have not always been successful.

Initial attempts to develop targeted therapy/immunotherapy combinations clinically were not successful due to severe toxicity, said Dr Keiran Smalley, lead researcher.

These toxicities led the research team to determine whether using a sequential treatment approach rather than a simultaneous combination approach would lead to durable antitumour activity in BRAF and NRAS-mutated melanoma.

The researchers analysed sequential combinations of immunotherapy followed by either BRAF/MEK-targeted therapy or another targeted therapy combination (ceritinib/trametinib) and vice versa in pre-clinical mouse models. They discovered that immunotherapy followed by targeted therapy was more effective than either treatment approach alone. While using targeted therapy followed by immunotherapy was better than targeted therapy alone, it was not as effective as the immunotherapy-targeted therapy sequence.

The team discovered the immunotherapy-targeted therapy sequence modulated the environment to promote immune cell functions resulting in antitumour activity while simultaneously blocking activity that permitted tumour cell escape from immune detection. They also showed that the immunotherapy-targeted therapy sequence enriched the melanoma cells for proteins that promoted immune cell detection and suppressed signalling pathways associated with drug resistance.

sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses

Results from this research suggest that sequential treatment approaches with anti-PD-1 agents followed by targeted therapy may be clinically beneficial and several ongoing trials are currently investigating this hypothesis.

Together, our data demonstrates that sustained antitumour responses to targeted therapy are dependent upon a vigorous, sustained immune response and that upfront use of immunotherapy can augment this, said Smalley.

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New developments in the research field of immuno-oncology - Drug Target Review

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that the following poster, highlighting a preclinical research collaboration using its ARCUS genome editing platform for treatment of transthyretin amyloidosis (ATTR), will be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, scheduled for May 11-14, 2021.

Title: Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal modelsPoster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases, Abstract 497Date/Time: Tuesday May 11, 2021 8:00 AM - 10:00 AMPresenting Author: Jenny A. Greig, Ph.D., Senior Director, Gene Therapy Program, Perelman School of Medicine, University of PennsylvaniaCo-Authors: Cassandra Gorsuch2, Joanna K. Chorazeczewski1, Melanie K. Smith1, Thomas Furmanak1, Alexa N. Avitto1, Scott N. Ashley1, Wendy Sharer2, Hui Li2, Jeff Smith2, Peter Clark1, Camilo Breton1, Derek Jantz2, and James M. Wilson1

Transthyretin amyloidosis is a rare disease caused by the progressive accumulation of misfolded transthyretin (TTR) protein into amyloid fibrils, which leads to peripheral neuropathy and/or cardiomyopathy. Research to be presented at the annual ASGCT meeting, led by Dr. Jenny A. Greig at the Perelman School of Medicine, University of Pennsylvania, used an AAV vector for in vivo delivery of ARCUS gene editing nucleases to knock out the TTR gene, which is responsible for ATTR.

With this program, we are excited to continue building a dataset demonstrating in vivo gene editing in large animal models using ARCUS nucleases, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. In this study, use of an optimized ARCUS nuclease to knock out the TTR gene was found to be effective in both mice and nonhuman primates, where we observed a good correlation between TTR gene editing in the liver and reductions of TTR in the serum. This approach addresses the root cause of the disease and results in genomic edits that are expected to be permanent. These results continue to demonstrate the power and versatility of ARCUS nucleases, particularly for in vivo editing.

Abstracts for the ASGCT 2021 Meeting are available on the meeting website.

About ARCUSARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including more than 75 patents to date.

About Precision BioSciences, Inc.Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward Looking StatementsThis 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 statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon the transthyretin gene, transthyretin serum levels and transthyretin amyloidosis. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, believe, could, eligible, expect, expected, should, plan, intend, estimate, target, mission, goal, may, will, would, should, could, target, potential, potentially, promising, project, predict, contemplate, potential, or the negative thereof and similar words and expressions.

Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

1 Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA2 Precision BioSciences, Inc., Durham, NC, USA

Excerpt from:
Precision BioSciences Announces Poster Presentation at the Upcoming American Society of Gene & Cell Therapy Annual Meeting - Business Wire

As per the research report, Global Gene Therapy Market is expected to reach the market valuation of US$ 20.9 billion by 2027 expanding at a reasonable CAGR of 29.7% during the forecast period (2021-2027) from US$ 3.5 billion in 2019.

Gene therapy is a technique that involves inserting genetic material into cells in order to correct for abnormal genes or produce a desirable protein. The growing number of ongoing clinical trials, as well as significant mergers, acquisitions, and venture capital investments in the gene-therapy field, suggest that the future of such therapies is promising. About 2,600 gene therapy clinical trials have been completed, are in progress, or have been accepted around the world to date. Gene therapy researchers are working more than ever to find a path to the clinic and the market. About two thousand clinical trials in human gene therapy have been published worldwide, and about 20 gene therapies have been approved. Such progress raises the prospect of treating devastating rare and hereditary illnesses, as well as incurable diseases.

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As of the end of the first quarter of 2019, 372 gene therapy clinical trials were in development, according to the Alliance for Regenerative Medicines (ARM) Quarterly Regenerative Medicine Global Data Study. Surprisingly, trials in Phase II had the largest margin (217 or 58 percent), followed by Phase I (123 or 33 percent), and Phase III (123 or 33 percent) (32 or 9 percent). The number of gene therapy clinical trials increased by ten from the previous years total of 362 trials. In fact, from 319 trials in progress in Q1 2018, the number of clinical tests has increased by 17% year over year. Many gene therapy clinical trials have focused on treating cancers such as breast, gynaecological, skin, urological, neurological, and gastrointestinal tumours, as well as haematological malignancies and paediatric tumours. According to the International Agency for Research on Cancer, 1 in 5 people will grow cancer over their lifetime, with 1 in 8 men and 1 in 11 women dying from it. According to these recent estimates, nearly 50 million people are living within five years of a previous cancer diagnosis. Globally, ageing demographics and socioeconomic risk factors continue to be major factors driving this increase.

For a detailed analysis of the applications in the Gene Therapy Market browse through https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Insights Presented in the Report

Amongst vectors, Viral vectors segment holds the major share

The market is divided into viral and non-viral vectors based on the form of vector. Due to major advances in vector engineering, distribution, and protection, viral vectors dominated the industry in 2019 with an 88.6 percent share and are projected to continue their dominance over the forecast era.

Amongst viral vectors, adeno-associated virus vectors are anticipated to dominate the market during the analyzed period

Lentivirus, adeno-associated virus, retrovirus & gammaretrovirus, transformed herpes simplex virus, and adenovirus are the major segments of the viral vector market. Because of the fast penetration into the host genome, no viral genes, ability to transduce cells that are not actively dividing, a wide variety of host cells, and being non-inflammatory and non-pathogenic, the adeno-associated virus sub-segment accounted for a maximum market sales share of 34% in 2019 and is projected to remain dominant throughout the studied timeframe. The non-viral vector, on the other hand, is expected to rise at the fastest rate over the projected period.

Amongst gene type, antigen segment dominated the market during the forecast period

The market is divided into antigen, cytokine, tumour suppressor, suicide, deficiency, antibody, and others based on gene form. The antigen category has the highest market sales share of 19.2 percent in 2019 and is projected to remain dominant over the forecast period.

Amongst indication, oncology segment holds the major share

The industry is divided into oncology, rare disorders, respiratory, neurology, infectious diseases, among others based on indication. Thanks to the prevalence of cancer cases, the oncology segment accounted for the largest market share of 48.6% in 2019 and is projected to be the leading segment of the gene therapy market over the forecast period.

Amongst the delivery method, in-vivo segment dominated the market during the forecast period

The market is primarily divided into in-vivo and ex-vivo delivery methods, depending on the method of delivery. Due to the direct transfer of genes into patients, the in-vivo segment accounted for a maximum market revenue share of 87.5 percent in 2019 and is projected to remain dominant throughout the studied timeframe.

North America represents one of the largest markets of Gene Therapy market

A systematic study of the consumer dynamics of the gene therapy market was performed for various regions around the world, including North America (the United States, Canada, and the Rest of North America), Europe (Germany, France, Italy, Spain, United Kingdom and Rest of Europe), Asia-Pacific (China, Japan, Australia, South Korea, and the Rest of APAC), and the Rest of the World. Because of the high incidence of cancer, the presence of high disposable income, and a rise in support for R&D initiatives associated with gene therapy, North America led the industry and reported sales of US$ 1.7 billion in 2019.

Customization Options:

The Gene TherapyMarket can further be customized as per the requirement or any other market segment. Besides this, UMI understands that you may have your own business needs, hence feel free to connect with us to get a report that completely suits your requirements.

Table of Contents

1 Market Introduction

2 Research Methodology or Assumption

3 Industry Performance

4 Executive Summary

5 Top Start-Ups Under Gene Therapy Sector

6 COVID-19 Impact

7 Market Insights by Vector

8 Market Insights by Gene Type

9 Market Insights by Indication

10 Market Insights by Delivery Method

11 Market Insights by Region

12 Gene Therapy Market Dynamics

13 Legal & Regulatory Framework

14 Demand and Supply Side Analysis

15 Value Chain Analysis

16 Gene Therapy Market Opportunities

17 Gene Therapy Market Trends & Insights

18 Competitive Scenario

19 Company Profiled

20 Disclaimer

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Gene Therapy Market Worth US$ 20.9 billion UnivDatos Industry Analysis- by Size, Share, Growth, Trends, and Forecast 2021-2027 The Courier - The...

The US Food and Drug Administration (FDA) has removed a clinical hold on Dutch biotech uniQures haemophilia B gene therapy, following concerns over a case of cancer in a patient in a pivotal trial.

In December 2020, the FDA placed a hold on uniQures haemophilia B clinical programme following the diagnosis of hepatocellular carcinoma (HCC) a type of liver cancer in a patient in the HOPE-B trial evaluating AMT-061 (etranacogene dezaparvovec).

In a statement, uniQure said that the patient diagnosed with HCC had multiple risk factors associated with this type of cancer. This included a 25-year history of hepatitis C (HCV) as well as a history of hepatitis B (HBV). Chronic infections with both HCV and HBV are associated with around 80% of HCC cases.

Multiple analyses, following a surgical resection of both the patients tumour and adjacent liver tissue, showed that AAV vector integration in the tissue sample was extremely rare, accounting for 0.027% of the cells in the sample.

The integration events that were present were randomly distributed, the company added, with no signs of clonal expansion or any dominant integration event.

Whole genome sequencing of the tumour also confirmed that it had genetic mutations characteristic of HCC, independent of vector integration.

UniQure noted that a gene expression analysis of the tumour and adjacent tissue suggested a precancerous state in the liver that could have predisposed the patient to developing HCC.

Patient safety is our top priority, and we are grateful to our advisors and the FDA for their help in resolving this clinical hold, said Ricardo Dolmetsch, president of research and development at uniQure.

Our comprehensive investigation showed that AMT-061 is very unlikely to have contributed to the HCC in our patient. We look forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter, he added.

UniQure added that all patients in its haemophilia clinical programme have abdominal ultrasounds performed one year after dosing, with patients continuing to receive this test every six months.

AMT-061 has been granted breakthrough therapy designation by the FDA and a priority medicine (PRIME) designation by the European Medicines Agency (EMA).

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FDA removes clinical hold on uniQure's haemophilia gene therapy - PMLiVE

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announced today that it will host a key opinion leader (KOL) webinar on GJB2-Related Genetic Hearing Loss on Monday, May 10, 2021 at 10am Eastern Time.

The event will feature a presentation by KOL Thomas Lenarz, M.D. Ph.D., Medical University of Hannover, who will discuss the clinical aspects, current treatments landscape and unmet medical needs in treating patients with a pediatric onset of GJB2related hearing loss as well as the role of the GJB2 gene. Dr. Lenarz will be available to answer questions following the formal presentations.

Sensorion's management team will also discuss their internal capabilities dedicated to gene therapy development as well as their GJB2-GT gene therapy program, which aims to restore hearing in people living with hearing loss due to mutations in the GJB2 gene in both children and adult populations.

To register for the call, please click here.

Thomas Lenarz, M.D. Ph.D. is Professor of Otorhinolaryngology and Chair of the Department of Otorhinolaryngology at the Medical University of Hannover, Germany, and a pioneer in the development of surgical and pharmacological interventions for hearing loss. Under his leadership, the department has become the center of an internationally recognized world-leading network for diagnosis, treatment, and translational research on hearing loss.

Professor Lenarz and his team have developed the largest international cochlear implantation program in the world. His research activity on surgical approaches to hearing loss extends beyond cochlear implants and includes central auditory implants in the midbrain and brain stem for neural deafness and implantable hearing aids for middle ear and inner ear hearing loss.

Professor Lenarz received his Ph.D. in pharmacology of the auditory system in 1987 before undertaking postdoctoral research at the University of California in San Francisco in 1989. He is currently vice president of the German Society of Biomedical Technology and speaker of the expert group on health care technologies at Acatech (German National Academy of Technology).

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion has launched three gene therapy programs, currently at preclinical stage, aimed at correcting hereditary monogenic forms of deafness including deafness caused by a mutation of the gene encoding for Otoferlin, hearing loss related to gene target GJB2 as well as Usher Syndrome Type 1 to potentially address important hearing loss segments in adults and children. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.

http://www.sensorion.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2020 Half-Year financial report published on October 21, 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

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Sensorion Hosting Key Opinion Leader Webinar with Dr. Thomas Lenarz on the GJB2 Gene Related Hearing Loss on May 10, 2021 - Business Wire

TheHemophilia Gene Therapy Marketis expected to have a highly positive outlook for the next eight years 2020-2027. This Research Reports emphasizes on key industry analysis, market size, Share, growth and extensive industry dynamics with respect to with respect to drivers, opportunities, pricing details and latest trends in the industry.

The global Hemophilia Gene Therapy Market analysis further provides pioneering landscape of market along with market augmentation history and key development involved in the industry. The report also features comprehensive research study for high growth potential industries professional survey with market analysis. Hemophilia Gene Therapy Market report helps the companies to understand the market trends and future market prospective,opportunities and articulate the critical business strategies.

Request Exclusive PDF Of This Report: https://www.coherentmarketinsights.com/insight/request-pdf/2480

The Hemophilia Gene Therapy market analysis report speaks about the growth rate till 2027 manufacturing process, Growth, key factors driving this market with sales, revenue, and price analysis of lead manufacturers of Digital Mapping Cameras Market, distributors, traders and dealers of Hemophilia Gene Therapy.

The comprehensive value chain analysis of the Hemophilia Gene Therapy market will assist in attaining better product differentiation, along with detailed understanding of the core competency of each activity involved. The market attractiveness analysis provided in the report measures the potential value of the Hemophilia Gene Therapy providing business strategists with the latest growth opportunities.

The report studies the historical and present performance of the market to project future growth. For this market report, 2019 is considered as a base year. The historical data was studied for year 2020 to 2021.

Geographically Regions Covered:

Table of Content:

Chapter 1 Research Objective and Assumptions

Research ObjectivesAssumptionsAbbreviations

Chapter 2 Market Purview

Report DescriptionExecutive SummaryCoherent Opportunity Map (COM)

Chapter 3 Global Market Demand Productions Supply Sales Demand Market Status and Forecast

Chapter 4 Market Dynamics, Regulations, and Trends Analysis

RestraintsMarket OpportunitiesRegulatory ScenarioIndustry TrendMerger and AcquisitionsNew Product Approvals/LaunchPromotion and Marketing Initiatives

Chapter 5 Competitive Landscape

Heat Map AnalysisMarket Share AnalysisCompany Profiles

Some Major Players are BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics

Chapter 6 Conclusion

Key Features of the Hemophilia Gene Therapy Market Report:

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Hemophilia Gene Therapy reportcover complete modest view with the market stake and company profiles of the important contestants working in the market. Also it offers a summary of product specification, production analysis, technology, product type, considering key features such as gross, gross margin, revenue & cost structure. The report helps the user to strengthen decisive power to plan their strategic moves to launch or expand their businesses by offering them a clear picture of this market.

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Hemophilia Gene Therapy Market: Global Industry Insights, Growth Prospects, Business Overview, Growth Rate and Forecasts 2027 KSU | The Sentinel...

- CANbridge to lead development and commercialization in China; expands rare disease pipeline

- Mirum is entitled to receive up to an aggregate of $120.0 million in upfront and milestone payments, and significant double-digit tiered royalties

FOSTER CITY, Calif. & BEIJING--(BUSINESS WIRE)-- Mirum Pharmaceuticals (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan). Maralixibat, an investigational, orally administered medication, is being evaluated in Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210429005614/en/

Maralixibat targets the apical sodium dependent bile acid transporter (ASBT), ultimately resulting in lower levels of bile acid systemically, which could mediate liver damage. The U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with ALGS under priority review. The European Medicines Agency is reviewing maralixibat for the treatment of PFIC2. Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.

Under the terms of the licensing agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions for ALGS, PFIC, and BA. In exchange, Mirum is entitled to receive an $11.0 million upfront payment, R&D funding, and up to $109.0 million for the achievement of future regulatory and commercial maralixibat milestones, with significant double-digit tiered royalties based on product net sales.

In collaboration with Mirum, CANbridge has agreed to oversee Mirums clinical study sites in China, with the goal of accelerating enrollment of the global Phase 2b EMBARK study, which was recently initiated for patients with BA. CANbridge would also have the right to manufacture maralixibat in Greater China under certain conditions.

Maralixibat has the potential to be a transformative medication for certain cholestatic liver diseases and our goal is to ensure its availability to patients globally, said Chris Peetz, president and chief executive officer at Mirum. CANbridge is a leading rare disease company in China and with their track record of commercial success, we believe they will be a strategic partner to accelerate the global launch of maralixibat, if approved.

We are thrilled to add maralixibat to our rare disease portfolio, as we expand into liver diseases and strengthen our pipeline with a late-stage asset in indications for which there are no approved treatments, said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. We are looking forward to participating in the global development of maralixibat in BA by supporting the China sites for the global Phase 2b EMBARK study, in collaboration with Mirum, and to working closely with Mirum to bring this treatment to patients and families in Greater China, where the need is great.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirums marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of treatments for orphan diseases and rare cancers to address unmet medical needs. CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in rare diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome (also known as mucopolysaccharidosis type II), developed by GC Pharma and marketed in more than 12 countries worldwide. CANbridge also has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.

For more on CANbridge Pharmaceuticals Inc., please go to: http://www.canbridgepharma.com.

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirums expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential development and commercialization by CANbridge of maralixibat in Greater China, Mirums receipt of milestone and royalty revenue in connection with the license agreement with CANbridge, and the regulatory approval pathway for maralixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as will, could, would, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirums current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirums business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirums filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210429005614/en/

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Mirum Pharmaceuticals and CANbridge Pharmaceuticals Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat in Greater China...

The emergence of efficacious human gene therapy for life-limiting genetic diseases such as spinal muscular atrophy type 11 has raised hopes among families of children with previously incurable genetic disorders. However, a number of concerns have surfaced regarding the gene therapy trial process. Two bereaved parents, both deeply engaged in their disease communities, collaborated with an experienced gene therapy researcher to create patient education materials to aid parents in critical decision-making. In this Family Partnerships article, we present a summary of the module, as well the reflections of each of the 3 contributors describing the impetus for the Courageous Parents Network (CPN) unit Evaluating the Clinical Trial Option, and their hopes for its impact, as captured in an interview conducted after completion of the educational module.

The Evaluating the Clinical Trial Option unit (https://courageousparentsnetwork.org/topics/clinical-trial) offers an overview of the fundamental elements of clinical trial participation, written for a nonmedical audience to help families understand and evaluate their options. It is disease and therapy agnostic and does not advocate for any particular path or decision. Content areas include a glossary of terms; informed consent; clinical trial basics, such as efficacy; trial design, including the phases; participation criteria; and the emotional impact of decision-making. Its elements include a variety of media types: 85 short videos (average 24 minutes each, a total of 240 minutes); downloadable guides in English and Spanish; 3 podcasts; 4 blog posts written by parents; and a curated educational digital experience (Guided Pathway) that, through videos and text, walks parents

Address correspondence to Terence R. Flotte, MD, Medical School, University of Massachusetts and Deans Office, 55 Lake Ave North, Worcester, MA 01655. E-mail: terry.flotte{at}umassmed.edu

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Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives - American Academy of Pediatrics

The global gene therapy market is estimated to grow from USD 3.8 billion in 2021 to USD 13.0 billion by 2027, at a CAGR of 27.8%.

TheGene Therapy Marketresearch report thoroughly explains each and every aspect related to the Global Gene Therapy Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business.

A large-scaleGene TherapyMarket document offers an all-inclusive study about production capacity, consumption, import, and export for all the major regions across the world. Furthermore, the statistical and numerical data such as facts and figures are represented very neatly in the report by using charts, tables, or graphs. This market report also involves strategic profiling of the major players in the market, comprehensive analysis of their basic competencies, and thereby keeping the competitive landscape of the market in front of the client. The winningGene Therapy report covers all the market shares and approaches of the major competitors or the key players in the market.

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Scroll down 100s of data Tables, charts and graphs spread through Pages and in-depth Table of Content on Gene Therapy Market, By Vector (Non-Viral Vectors, Viral Vectors), Indication (Neurological Diseases, Cancer, Duchenne Muscular Dystrophy, Hepatological Diseases, Other Indications), Delivery Method ( In Vivo, Ex Vivo) and Geography Global Forecast to 2026. Early buyers will get 10% customization on study.

To Avail deep insights of Gene Therapy Market Size, competition landscape is provided i.e. Revenue Analysis (M $US) by Company (2018-2020), Segment Revenue Market Share (%) by Players (2018-2020) and further a qualitative analysis is made towards market concentration rate, product/service differences, new entrants and the technological trends in future.

Unlock new opportunities in Gene Therapy Market; the latest release fromMarketDigitshighlights the key market trends significant to the growth prospects, Let us know if any specific players or list of players needs to consider to gain better insights.

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The global gene therapy market is estimated to grow from USD 3.8 billion in 2021 to USD 13.0 billion by 2027, at a CAGR of 27.8%. The growth of this market is majorly driven by the high incidence of cancer and other target diseases, availability of reimbursements, and the increasing funding for gene therapy research. However, the high cost of gene therapies is expected to hamper market growth to a certain extent during the forecast period.

The demand for gene therapies for the treatment of cancer is estimated to grow at a high rate during the forecast period

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The cancer segment is estimated to grow at the highest CAGR during the forecast period owing to the approval of a growing number of gene therapies for the treatment of cancer and the rising incidence of hematologic cancers.

Non-viral vectors segment accounted for the largest share of the gene therapy market, by vector, in 2018

The market, by vector, has been segmented into viral and non-viral vectors. The non-viral vectors segment accounted for the largest share of the market in 2018. This can be attributed to the high market penetration of oligonucleotide-based non-viral vector gene therapies.

North America is the largest regional market for gene therapies

The global market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the market, followed by Europe. The rising prevalence of chronic diseases, high and growing healthcare expenditure, presence of advanced healthcare infrastructure, availability of reimbursements, and the presence of major market players in the region are the major factors driving the growth of the gene therapy market in North America.

Key Market Players

The prominent players operating in the gene therapy market include Biogen (US), Novartis AG (Switzerland), Gilead Sciences, Inc. (US), Spark Therapeutics, Inc. (US), MolMed S.p.A. (Italy), Orchard Therapeutics plc. (UK), SIBIONO (China), Shanghai Sunway Biotech Co., Ltd. (China), bluebird bio, Inc. (US), Human Stem Cells Institute (Russia), AnGes, Inc. (Japan), Alnylam Pharmaceuticals, Inc. (US), Sarepta Therapeutics (US), Jazz Pharmaceuticals, Inc. (Ireland), Akcea Therapeutics (US), and Dynavax Technologies (US).

Biogen was the leading player in the market in 2018. Strong business operations across all key regions, coupled with a strong brand image, have made Biogen a leading player in this market. Biogen adopted organic and inorganic growth strategies, such as agreements & collaborations and acquisitions, to maintain its leading position in the market. For instance, in 2017, Biogen entered into a collaboration with Ionis Pharmaceuticals to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy. In 2019, the company received the NMPA (National Medical Products Association) approval in China for SPINRAZA, which is used for treating 5q spinal muscular atrophy.

Recent Developments

What Porters Five Forces of Competitive Analysis Provides?

Competitive rivalry:-The main driver is the number and capability of competitors in the market. Many competitors, offering undifferentiated products and services, will reduce market attractiveness.

Threat of substitution:-Where close substitute products exist in a market, it increases the likelihood of customers switching to alternatives in response to price increases. This reduces both the power of suppliers and the attractiveness of the market.

Threat of new entry:-Profitable markets attract new entrants, which erodes profitability. Unless incumbents have strong and durable barriers to entry, for example, patents, economies of scale, capital requirements or government policies, then profitability will decline to a competitive rate.

Supplier power:-An assessment of how easy it is for suppliers to drive up prices. This is driven by the: number of suppliers of each essential input; uniqueness of their product or service; relative size and strength of the supplier; and cost of switching from one supplier to another.

Buyer power:-An assessment of how easy it is for buyers to drive prices down. This is driven by the: number of buyers in the market; importance of each individual buyer to the organisation; and cost to the buyer of switching from one supplier to another. If a business has just a few powerful buyers, they are often able to dictate terms.

Five forces analysis helps organizations to understand the factors affecting profitability in a specific industry, and can help to inform decisions relating to: whether to enter a specific industry; whether to increase capacity in a specific industry; and developing competitive strategies.

The country section of the report also provides individual market impacting factors & changes in regulation in the market domestically that impacts the current & future trends of the market. Data points such as consumption volumes, production sites & volumes, import export analysis, price trend analysis, cost of raw materials, down-stream & upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence & availability of global brands & their challenges faced due to large or scarce competition from local & domestic brands, impact of domestic tariffs & trade routes are considered while providing forecast analysis of the country data.

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Gene Therapy Market 2021-2026 to Witness High Growth in Near Future and Competitive Analysis | Leading Players Abeona Therapeutics, Audentes...

ALISO VIEJO, Calif.--(BUSINESS WIRE)--The international rare disease patient advocacy organization, Global Genes, is pleased to announce the 6th Annual RARE Drug Development Symposium (RDDS) in partnership with the Orphan Disease Center of the University of Pennsylvania. The June 9-11 virtual event will connect and educate hundreds of advocates, clinicians, and academic and industry researchers to explore the latest science, opportunities, and challenges to the advancement of therapies for more than 7,000 rare diseases.

New to this year's event is an optional preconference workshop targeted to attendees who are in the earlier stages of their research efforts or careers and looking to build knowledge in core competencies that will be expanded upon in the main RDDS program. The main program will address the current landscape of rare drug development and allow attendees to interact with subject matter experts and other rare disease stakeholders to help them better understand and develop their impact and role in advancing potential treatments.

This year, the RDDS keynote speaker will be David Fajgenbaum, M.D., MBA, MSc, co-founder and executive director of the Castleman Disease Collaborative Network (CDCN), assistant professor of medicine in Translational Medicine & Human Genetics at the University of Pennsylvania, associate director, Patient Impact for the Orphan Disease Center of the University of Pennsylvania, and author of the national bestselling book, Chasing My Cure: A Doctors Race to Turn Hope Into Action.

Dr. Fajgenbaum has been a leader in helping researchers to prioritize treatments for COVID-19 clinical trials and inform patient care through the CORONA (COvid19 Registry of Off-label & New Agents) Project. Were thrilled to have him share his insights during this critical time in health care and understand how we can apply these approaches to accelerate progress in rare disease research and treatments, said Craig Martin, CEO at Global Genes. The rare disease leaders who will be featured during the RDDS have tremendous depth of knowledge to share, and we look forward to sharing it with members of the rare community during this event.

RDDS will continue to host the CureAccelerator Live! For Rare Diseases 2021 event on June 10 in partnership with Cures Within Reach, a not-for-profit organization exclusively dedicated to using the speed, safety, and cost-effectiveness of already approved drugs, devices, diagnostics, nutraceuticals, and combination products to impact patients with unmet medical needs driving more treatments to more patients more quickly.

The emergence of therapeutic platforms creates unprecedented opportunities for treatments to improve the lives of those living with rare diseases, said Jim Wilson, M.D., Ph.D., director, Gene Therapy Program, and Rose H. Weiss, professor and director at the Orphan Disease Center of the University of Pennsylvania. We are delighted to collaborate with Global Genes to educate the rare disease community on research directed to these treatments.

Thank you to our gold sponsors, Horizon Therapeutics and Greenwich Biosciences, and silver sponsor, Pfizer, Inc., for their generous support of this important event.

For more information, visit http://www.globalgenes.org/rdds.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes and Orphan Disease Center of the University of Pennsylvania to Convene Patient Group Leaders, Researchers, Clinicians and Industry for...

Professor Naoto Hirano of the Temerty Faculty of Medicine and collaborators have developed a new technology that rigorously and robustly identifies the immune cells that are capable of recognizing and eliminating cancer cells.

The findings, published in Nature Biotechnology, pave the way for novel immunotherapies to help more patients, regardless of their genetic ancestry, live longer and healthier lives.

Adoptive cell therapy, a promising cancer treatment that uses our immune system to eliminate cancer cells, is effective only for a small subset of individuals with specific types of cancer and specific inherited genes. The new technology developed by Hirano, a professor in the department of immunology, allows researchers to develop new immunotherapies for cancer patients that are not limited by the differences or heterogeneity of tumour cells, expanding the potential impact of immunotherapy for patients around the world.

The technology applies to an immunotherapy approach called T cell receptor (TCR) gene therapy that is based on genetically-engineered immune cells (T cells) recognizing and binding to specific molecules, called peptide-loaded human leukocyte antigens (HLA), on the surface of cancer cells. Although there has been progress in TCR therapy, there are more than 28,000 different variations of HLA found in humans and current TCR therapies only work for a few of these variations.

Historically, TCR treatments have been developed for those who had the most common and well-studied HLA alleles, which often meant that these immunotherapies only worked for people from Caucasian ancestry, says Hirano, who is also a senior scientist at the Princess Margaret Cancer Centre and Ontario Institute for Cancer Research clinician scientist.

It was an important goal for us to develop a technology that could work for a broad range of HLA alleles. Were proud of what we developed because it could help many more cancer patients in the future.

The technology presented in this study involves a methodology that can in a single step, at a low expense form a functional protein structure, called a dimer, that is composed of any peptide and HLA molecule, regardless of type, and can bind to and identify a variety of T cells. The method improves the binding affinity between T cells and HLA molecules nearly 200-fold relative to prior methods, which could allow researchers to better identify and engineer the T cells for novel immunotherapies.

The technology has been licensed to TCRyption Inc., a company co-founded by Hirano,for further development, translationand large-scale implementation. In the future, it may be applied to fields other than cancer research and care, including autoimmune diseases such as rheumatoid arthritis and type 1 diabetes.

Im grateful for the cancer research communitys support over the years, which has enabled me to focus on important and challenging issues, says Hirano, who was named the University Health Networks Inventor of the Year last year for developing these analysis techniques. Only with the support for rigorous experimentation, deep expertise, and innovative thinking, were we able to make this breakthrough.

This article was originally published by the Ontario Institute for Cancer Research.

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New technology could allow more cancer patients to benefit from immunotherapy: U of T researchers - News@UofT

BioCentriq, the New Jersey Innovation Institutes cell and gene therapy development and manufacturing center, on March 23 announced it partnered with Kytopen, a Cambridge-based startup spun out of the Massachusetts Institute of Technology (MIT).

Our mission at BioCentriq is to work with innovative industry partners like Kytopen to advance the production and manufacturing of cell and gene therapies, making them accessible and affordable for the patients who so desperately need them, said Haro Hartounian, and SVP, general manager, BioCentriq. This partnership aligns perfectly with that mission.

Kytopen leadership team KYTOPEN

Kytopens proprietary Flowfect technology is a flexible, complete technology solution for non-viral cell engineering that integrates the discovery, development, and manufacturing of cell and gene therapeutics. The platform speeds therapies from the clinic to commercial use by enabling cell engineering without compromising functionality or viability. Kytopens technology reduces risk and provides maximum control and flexibility to drive higher yields, faster approvals, and better outcomes for curative cellular disease treatment.

The Flowfect platform is a transformative solution that eliminates the complexity of gene delivery for cell engineering and links discovery, development and manufacturing in one flexible scalable solution, stated Paulo Garcia, CEO and co-founder of Kytopen. Our goal is to enable simple and efficient non-viral manufacturing of cell therapies in days versus weeks to help patients; our partnership with BioCentriq accelerates that goal.

Our Flowfect technology utilizes a novel combination of electrical energy and continuous fluid flow to engineer cells, said Bethany Grant, head of research and development at Kytopen. Our ability to engineer billions of cells in minutes with minimal disruption unlocks new opportunities to enable curative therapies in autologous or allogeneic therapeutic applications.

In the initial phase of the collaboration, the Kytopen and BioCentriq teams will demonstrate the impact to both autologous and allogeneic cell therapies by integrating this novel transfection technology with other steps in the manufacturing process.

BioCentriq has a manufacturing facility in Newark and a pilot plant in South Brunswick.

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BioCentriq partners with Kytopen to advance production and manufacturing of cell and gene therapies - NJBIZ

The global cell and gene therapy drug delivery devices market was valued at $55.75 thousand in 2019, and is expected to reach $375.13 thousand by 2030, registering a CAGR of 16.61% during the forecast.

The global cell and gene therapy drug delivery devices marketis projected to reach $375.13 thousand by 2030, reveals the premium market intelligence study by BIS Research. The study also highlights that the market is set to witness a CAGR of 16.61% during the period between 2020 and 2030.

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BIS Research Study Highlights the Global Cell and Gene Therapy Drug Delivery Devices Market to Reach $10.82 Billion by 2030 investigated in the latest...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Maze Therapeutics, a company translating genetic insights into new precision medicines, today revealed its first three lead therapeutic candidates in the companys wholly owned pipeline. The candidates include:

Each of the three lead candidates was enabled by Mazes COMPASS platform, which uncovered important new findings for the genetic target, discerning which specific signals may be critical for the treatment of patients, and which are likely non-actionable. The Maze pipeline will have the potential to serve as precision medicines for rare diseases and mechanistically defined subsets of common diseases based on certain genetic drivers.

In addition, Maze is concurrently leveraging COMPASS to advance additional discovery-stage research programs across three main therapeutic areas of focus: metabolic, cardio/renal and neurological diseases. These programs will constitute a broad, diverse pipeline for Maze and will be a combination of wholly owned and partnership-led collaborations.

Maze was built by co-founders, including Charles Homcy and other preeminent thinkers in the field of genetics, on a bold vision to leverage growing knowledge of genetic drivers of disease in order to create precision medicines for the treatment of both rare and more common diseases, said Jason Coloma, Ph.D., president and chief executive officer of Maze. Since our founding, we have been leveraging insights from leading geneticists, combined with the growing availability of paired human genetic and clinical data, the evolution of functional genomic technologies and advances in computational power, to build our COMPASS platform in order to bring unique insights into efficient, genetics-based drug development. We are excited by the significant progress we have made with our platform and pipeline, bringing us an important step closer to our goal of delivering the right drug to the right patient at the right time.

Mazes therapeutic candidates are designed to: 1) target genes whose activity affects the phenotype associated with another, often distant, gene, referred to as genetic modifiers; 2) mimic the activity of protective genetic variants; 3) correct the effects of toxic genetic variants; or 4) leverage new genetic insights to address otherwise challenging drug targets.

COMPASS is a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. In addition, Maze is exploring applications of COMPASS in diseases of haploinsufficiency by identifying genetic mechanisms that increase levels of a deficient protein and translating them into therapeutics.

New findings using COMPASS helped fill in fundamental data gaps, turning known but challenging targets into exciting, differentiated approaches to the genetic drivers of disease for our first three programs, said Sarah Noonberg, M.D., Ph.D., chief medical officer of Maze. While it has been shown that targets with human genetic evidence are more likely to yield efficacious treatments, very few groups have had the capabilities to then turn genetic insights into viable drug programs. We believe our COMPASS platform, integrated with our extensive drug discovery capabilities, will allow us to accelerate the pace of therapeutic development, as well as increase the likelihood of producing therapies that provide meaningful clinical benefit for patients. We are excited to advance these initial programs and look forward to continued progress toward the clinic as efficiently as possible.

About Mazes Wholly Owned Programs

GYS1 Program for Pompe DiseasePompe disease is a rare, inherited autosomal recessive disorder with an incidence of approximately 1 in 40,000 live births in the U.S., and is estimated to affect 5,000 to 10,000 patients worldwide. It is caused by mutations in the GAA gene, which codes for an enzyme responsible for breaking down lysosomal glycogen into glucose. As a result of this mutation, glycogen accumulates in various tissues, particularly skeletal and cardiac muscle tissues, causing progressive weakness and respiratory insufficiency.

Maze is developing a novel, oral approach to treating Pompe disease by inhibiting the protein muscle glycogen synthase, which is encoded by the gene GYS1. Targeting this protein leads to reduction in the synthesis of glycogen, which is expected to restore glycogen balance through a mechanism called substrate reduction. While GYS1 has been a therapeutic target of interest, its attractiveness as a therapeutic target has been limited due to its structural complexity and uncertainties related to the tolerability of a long-term reduction in muscle glycogen levels. Critical insights derived from COMPASS have enabled Maze to overcome these challenges. Maze has interrogated the structurally complex protein to develop an oral inhibitor of muscle glycogen synthase, a target not previously addressable by small molecule therapies. Maze is rapidly progressing its GYS1 program toward an Investigational New Drug application and expects to initiate clinical trials in the first half of 2022.

APOL1 Program for Chronic Kidney DiseaseCKD affects approximately 37 million people in the U.S., including more than 700,000 patients who suffer from end-stage renal disease (ESRD), many of whom require chronic dialysis. Individuals of African ancestry are at an approximately 3.5-fold greater risk of developing ESRD than individuals of European ancestry. Previous studies have shown that two coding variants of the apolipoprotein L1 (APOL1) encoded by the gene APOL1 cause toxic gain-of-function variants and are important genetic drivers of kidney disease that are responsible for much of the increased risk for CKD and ESRD in individuals of African ancestry. There are currently no approved therapies that address the underlying causes of APOL1-associated CKD, and efficacious treatment options for individuals with APOL1 risk variants and CKD represent a significant unmet medical need.

Maze employed COMPASS to functionalize human genetic variants to uncover the underlying biology of the target and has designed a small molecule that corrects the effects of toxic gain-of-function variants to potentially enable a therapeutic solution. Maze plans to name the development candidate in early 2022.

ATXN2 Program for Amyotrophic Lateral SclerosisALS is a progressive and fatal neurodegenerative disease with a prevalence of approximately 16,000 patients in the U.S. Current available treatments for ALS primarily focus on providing symptomatic relief and have limited impact on disease progression. A high variability in disease phenotype and life expectancy is observed and believed to be related to the presence of genetic modifiers.

One of Mazes founders, Aaron Gitler, identified a potent genetic modifier, ATXN2, whose inhibition has been shown to limit the toxicity of a certain protein, TDP-43, which is involved in pathologic aggregates seen in up to 97% of all ALS cases. Maze is translating these important insights by developing a novel microRNA gene therapy that targets ATXN2 and has used the proprietary application of its functional genomics tools to optimize its properties. Maze plans to name the development candidate in early 2022.

About Maze TherapeuticsMaze Therapeutics is focused on translating genetic insights into new precision medicines for rare diseases and mechanistically defined subsets of common diseases. Maze has developed the COMPASS platform, a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using COMPASS, Maze is building a broad portfolio, including wholly owned programs targeting Pompe disease, chronic kidney disease and amyotrophic lateral sclerosis, as well as partnered programs in cardiovascular and ophthalmic diseases. Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn.

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Maze Therapeutics Reveals Its Initial Three Lead Programs Targeting Underlying Genetic Drivers of Life-Threatening Diseases - Business Wire

Company to use its novel gene editing technology to explore a potential in vivo gene therapy treatment for cystic fibrosis

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundationto identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF). The award will enable Life Edit to screen its library of proprietary base editors for a potential treatment targeting people with CF that are not able to be treated by existing small molecule treatments due to what are known as nonsense genetic mutations (also known as stop mutations). This award is part of the Cystic Fibrosis Foundations Path to a Cure initiative that was launched in October 2019 to address and treat the underlying cause of CF.

Due in large part to the efforts of the CF Foundations support for the development of new medicines, there are now effective therapies available to most people living with the disease, but there remain as many as 7% of patients with CF for whom recent medical advances are not effective, said Mitchell Finer, Ph.D., Chief Executive Officer, Life Edit Therapeutics. Were looking forward to working with the CF Foundation to leverage the unique benefits that our platform offers to develop a highly targeted gene editing approach for these individuals. We believe our science and this approach can be applied across a range of diseases, which will be our focus as we work to build a pipeline of life-changing therapies for severe genetic diseases like CF.

Dr. Allie Crawley, Principal Investigator for the project and member of the Life Edit team, continued by saying, We are thankful to be a part of the Path to a Cure initiative from the CF Foundation which is focused on curing cystic fibrosis by addressing the underlying cause of the disease. We believe our base editor technology has potential to make a great impact in the lives of cystic fibrosis patients with nonsense mutations and are excited about the opportunity to begin early research in this development.

Despite tremendous progress in advancing therapeutics to help people with CF live longer and healthier lives, there remain unmet needs to help all those living with this disease. Approximately 13% of people living with CF have nonsense mutations. These mutations cause the cells to stop the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein midway through the process, resulting in shortened, non-functional protein.

As part of the $400,000 award from the Foundation, Life Edit will explore its large collection of adenine base editors, or A-base editors, that can potentially be used to correct the six most common, Class I, cystic fibrosis nonsense mutations to restore CFTR function in vivo. A unique feature of the base editors under development by Life Edit is their small size which will allow in vivo delivery with Adeno-associated viruses (AAV) vectors to specific tissue types in the lungs. As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare genetic disease found in more than 30,000 people in the U.S. CF is a hereditary disease that affects the lungs and digestive system that is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. When the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell. This imbalance leads to thick, sticky mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.

About Genome Editing and Life Edit Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, Life Edit Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. Life Edit Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The Life Edit Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of gene therapies, Life Edit Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About Life Edit Therapeutics Inc.

Life Edit Therapeutics is a next-generation gene editing company that has built a highly innovative genome editing platform with one of the worlds largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. The company is continuing to strengthen the platform, developing a pipeline of in vivo gene therapies to address severe genetic disease, and sharing its expertise through strategic partnership. Life Edit is an ElevateBio portfolio company. For more information visit lifeeditinc.com.

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Life Edit Therapeutics Announces Award from Cystic Fibrosis Foundation - BioSpace

- Initial data from Cohort 1 of the PEARL-1 study shows safety and tolerability of repeat KB301 injections

- Dr. Bhushan Hardas M.D., MBA appointed President, Jeune, Inc.

PITTSBURGH, March 24, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced the launch of Jeune, Inc., a wholly owned subsidiary of Krystal Biotech, and initial safety data from the ongoing Phase 1 trial of Jeunes lead product candidate, KB301 for treatment of aesthetic skin conditions.

Jeune was formed to advance innovative aesthetic medicines and has an exclusive license to a portfolio of candidates derived from Krystals proprietary technology platform. Jeunes products are designed to directly address biological changes in the skin associated with intrinsic and extrinsic aging. The lead product candidate, KB301, delivers the human COL3A1 gene to increase production of normal type III collagen at the site of administration.

My initial clinical experience with KB301 injections has been highly encouraging, said Dr. Mark Nestor, director of the Center for Clinical and Cosmetic Research and the Center for Cosmetic Enhancement. Not only were the injections well-tolerated, but we see clear signs of new collagen generation which underscores the potential of this treatment to directly address the declining levels of collagen that lead to wrinkles and other skin changes.

Initial data from Cohort 1 in the PEARL-1 studyThe Phase 1, open-label, dose-ranging study is being conducted in adult subjects aged 18-75 (NCT04540900). The primary outcome measure in this first-in-human study was to assess the safety profile of KB301. Secondary outcome measures include COL3A1 transgene expression. In Cohort 1, three different dose levels of KB301 were evaluated in seven (7) healthy subjects who received two intradermal injections into healthy buttock tissue spaced 30 days apart (day 0, day 30). KB301 injected areas were compared to uninjected or saline injected control tissue within the same subject. Treatment and control sites were biopsied at day 2 or day 32. Initial results are as follows:

More detailed data from Cohort 1 will be presented as an e-Poster talk at the Society for Investigative Dermatology (SID) Annual Meeting, held virtually May 3-8.

The presentation will be available on-demand for those registered for the SID conference from May 3, 2021 May 31, 2021. The poster will also be available on the companys website at http://www.jeuneinc.com

The company plans to begin enrollment in the efficacy cohorts of the Phase 1 study in the second half of 2021.

Jeune, Inc. LeadershipJeune has assembled a veteran team of leaders and advisors, comprised of pharmaceutical and biotechnology executives who together have decades of experience developing products in the aesthetic medicine space. Dr. Bhushan Hardas M.D., MBA will join the company on March 29th, 2021 as President of Jeune. Before joining Jeune, Dr. Hardas served as Chief Scientific Officer, Executive Vice President, Global Head of Licensing at Almirall and previously served as Chief Medical Officer of Allergan's Dermatology and Medical Aesthetics business.

I am thrilled to be joining Jeune at such an exciting time. With the ability to deliver genes directly to skin cells, this platform has tremendous potential to address underlying biological changes in aging or photo-damaged skin, noted Dr. Hardas. We are starting with KB301 and type III collagen which I look forward to advancing through the clinic, and the team is already working on pipeline programs that will address additional proteins of interest.

Prior to joining Allergan, Dr. Hardas served as Senior Vice President, Global Head of Dermatology and Aesthetics R&D and Chief Scientific Officer of the North American Business at Merz Pharmaceuticals. Dr. Hardas received advanced training in clinical immunology and molecular biology at King's College at the University of London, in London, England. He also completed a research fellowship in the Department of Dermatology at the University of Michigan, and received his Master of Business Administration degree in healthcare management from the University of California - Irvine.

We are thrilled to welcome Bhushan to Jeune, said Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech. His expertise and development experience in aesthetics is an important asset presently and will help guide next steps for both the pipeline and Jeune overall.

Jeune, Inc. Board Krish Krishnan, Chairman and CEO at Krystal Biotech will serve as the Chairman of the Jeune Board. Joining Mr. Krishnan on the Board are Marc Forth, President and CEO of Aeon BioPharma and Suma Krishnan, Founder and COO of Krystal Biotech.

AboutJeune Inc. Jeune Inc., a subsidiary of Krystal Biotech, is a biotechnology company leveraging a clinically validated gene-delivery platform to fundamentally address and reverse the biology of aging and/or damaged skin. For more information, please visithttp://www.jeuneinc.com

AboutKrystal BiotechKrystal Biotech, Inc.(NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.

Forward-Looking StatementsAny statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., or its subsidiary Jeune, Inc., including but not limited to statements about the development of Krystals and Jeunes product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of KB301 the clinical utility of KB301, the ability of these candidates to fundamentally address and potentially reverse the biology of aging or damaged skin, plans to pursue research and development of other product candidates; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including KB301 and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

CONTACTS:

Investors:Whitney Ijemwijem@krystalbio.com

Media:Mary CoyleTellMed Strategiesmary.coyle@tmstrat.com

Source: Krystal Biotech, Inc.; Jeune, Inc.

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Krystal Biotech Announces Launch of Jeune, a Gene-Based Aesthetics Company, and Initial Phase 1 Safety Data for KB301 in Aesthetic Indications -...