Category Archives: Gene therapy

By the end of this year, Amryts Filsuvez could become the first approved treatment for the rare skin disorder epidermolysis bullosa. Despite mixed pivotal data, regulators in the US and Europe might be swayed by the lack of available treatments.

More durable options could come with gene therapies, and phase 3 studies of these will start to report later this year. However, gene therapyprocedures can be complex, and Amryt will hope that the convenience ofFilsuvez, a topical gel that can be applied at home, could make it the first-line treatment of choice.

First mover advantage

Epidermolysis bulllosa (EB) is a group of inherited skin disorders that cause fragile, blistering skin. Current treatments aim to help alleviate symptoms, including bandaging and bathing open wounds and trying to manage patients pain.

Late last month, Amryt completed the US rolling submission for Filsuvez for junctional and dystrophic forms of EB, and the EMA began its reviewof the project.

The pivotal Ease study was not an emphatic victory, however.The trial met the primary endpoint, the complete closure of the target wound within 45 days, but several secondary measures were missed. These included90-day target wound closure, and the pain endpoint.

Driving the primary treatment effect was a group of patients with the recessive form of dystrophic EB. This was the main patient group in the study, which also recruited junctional and dominant dystrophic EB patients.

Lack of available treatment options means that regulators might look favourably on Filsuvez, but restricting its use torecessive dystrophic patients cannot be ruled out.

According to Stifel analysts the recessive form makes up 15% of patients; they reckon that even if Filsuvez only gets the nod in this population it could still bring in$342m in 2027. This rises to $421m that year with a broader label.

Gene therapy race

As EB is an inherited condition, gene therapy could be the way forward in treating the underlying cause. Amryts own topical gene therapy, AP103, is due to start clinical studies next year, but others are much further ahead, with Krystal, Abeona and Castle Creek all in phase 3.

By the fourth quarter, pivotal data should be available with Krystal Biotechsberemagene geperpavec(B-Vec), atopically applied gene therapy designed to induce local collagen production.

If the project can show strong signs of durability it could differentiate itself from Filsuvez. There are some questions about B-Vec's longevity, however, as in itsphase 1/2 studysome lesions that had closed later reopened (Gene therapys duration is less than Krystal clear, October 29, 2019).

The primary endpoint of the upcoming pivotal Gem-3 trial is complete wound healing at weeks 22 and 24, or weeks 24and 26; only wounds that are completely healed for at least two weeks count as a positive response.

Approximately 30 adult and paediatric patients are being enrolled with recessive or dominant dystrophic EB.Subjects will act as their own controls, receiving either B-Vec or placebo once weekly on different wounds.

B-Vec does not represent a "once-and-done" gene therapy; due to the rapid turnover of skin cells, it still requires frequent applications.

In Gem-3, the project was administered in the clinic butKrystal is hopeful that, if approved,B-Vec could be given at home. If clinic visits are required, thiscould prove problematic as travel can be hard for EB patientsdue to their extremely fragile skin.

Complicated procedures

Further behind are Abeona and Castle CreeksEB-101 and D-Fi respectively. Both offer a personalised treatment approach that is a far cry from the ease of topical application.

With both therapies, biopsiesare taken and skin cells are corrected by gene transfer. Abeonas treatment involves surgically transplanting sheets of cells onto patients wounds while Castle Creeks uses intradermal injections.

Top-line data from Abeonas open-label phase 3 Viital study, inlarge chronic wounds, are due in mid-2022. The trial will enrol 10-15 recessive dystrophic EB patients with wound sites that are larger than 20cm2, which have been present for more than six months.

The primary endpoint is the proportion of wound sites with 50% or greaterhealing from baseline at 24 weeks. This differs from the complete wound closure measure for competitors' treatments, presumably due to the large size of the wounds being treated.

In an earlier study run by Stanford University, wound healing of 50% or greater was present in 95% of wounds treated with EB-101 versus 0% of untreated control wounds at 24 weeks. At two years, 71% of treated wounds had 50% or greater healing compared with 17% of control wounds.

Lastly to Castle Creeks D-Fi, which was previously known asFCX-007 andoriginated at Fibrocell. Aphase 1/2 trial found that after a single dose, 63% of treated woundshad completely closed at 12 weeks, versus none in the untreated cohort.

The phase 3 trial, DeFI-RDEB, is enrolling patients with recessive dystrophic EB, and wounds will be treated for two or more sessions. The primary endpoint is complete wound closure of the first wound pair at week 24, and the study has a primary completion date of April next year.

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Epidermolysis bullosa gene therapies wait in the wings - Vantage

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 28, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that five data presentations, of which two are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14.

"Our presentations at ASGCT showcase our gene therapy expertise in hemophilia and Huntingtons disease, as well as advantages of the AAV5 vector in dosing through pre-existing neutralizing antibodies, stated Ricardo Dolmetsch, Ph.D.president of research and development at uniQure. In addition, we look forward to presenting preclinical data demonstrating the potential of the miQURE technology used in our CNS product candidates to be effective in liver-based diseases as well.

Specific details on uniQures presentations at ASGCT include:

About uniQure uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

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uniQure Announces Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting - GlobeNewswire

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

When Bluebird Bio secured European approval for its gene therapy to treat beta thalassemia in 2019, the biotech entered pricing negotiations confident that a shared risk plan could convince governments to pay $1.8 million per patient for the one-time, potentially curative treatment.

But two years later, Bluebird is pulling its gene therapy, called Zynteglo, from Germany after government officials would not budge from an offer to pay less than half of the companys list price. The stalemate is a significant setback for Bluebirds business ambitions in Europe, but it also raises broader questions about whether Europe can ever become a viable commercial market for other expensive gene therapies.

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Bluebird talks in Germany may be bad omen for gene therapy in Europe - STAT

Bayer's cell and gene therapy ambitions have quickly taken flightas the German conglomerate locks up new partners and pumps cash into promising up-and-comers. Now, the company is looking to beef up its cell offerings at the Berkeley campus where it has separately blueprinted a $1.2 billion, 30-year expansion.

Bayer has drawn up plans for a $200 million cell therapy plantin Berkeley, where a separatecell culture facility and cell and gene therapy labs are due to come online later this year, Bioprocess International reports.

Called the Cell Therapy Launch Facility, the new plant is the latest in a series of cell and gene therapy movesby Bayer, as the company rapidly carves out a foothold inthe bustling field. In the past few monthsalone,it bought out adeno-associated virus (AAV)specialist Asklepios BioPharmaceutical, launched a cell and gene therapy platform and plugged millions of investment dollarsintogene therapy players Metagenomi and Senti Biosciences.

As for the latestfacility, the 98,000-square-foot plantand first production moduleshould be ready to goby mid-2023, a Bayer spokesperson told the news outlet.

To start, the factory will turn out clinical materials for Bayer's cell therapy pipeline, though both the launch facility and the cell culture plant, dubbed theCell Culture Technology Center (CCTC), will eventually chip in on "global commercial launches of multiple products," the spokesperson said.

RELATED:Bayer unveils first look at its post-Xarelto, post-Eylea lifeand it's better than expected

A second production module isalready planned for the site, which could eventually produce up to four cell therapy products at the same time, Jens Vogel, SVP and global head of biotech at Bayer, said in a recentinterview with San Francisco Business Times.

Bayer counts a number of early-stage cell and gene hopefuls among its pipeline, includinggene therapiesfor congestive heart failure, Parkinson's and Pompe disease, as well as a CAR-T cell therapy candidate forhigh mesothelin-expressing tumors.

Bayer didn't say when construction on the launch facility would begin, and the company didn't immediately respond to Fierce Pharma's request for comment. The 40,000-square-foot, $150 million technology centeris expected to be up and running in late 2021.

The launch facility is separate from Bayer's bigger expansion plans in Berkeley, the company told Bioprocess International. In July, Bayer proposed a site development plan that would addabout 1 million square feet of new work space and double its workforce in Berkeleyby some1,000 employees.

The CCTC, expected to open later this year, will "combine automation, digital capabilities and single-use bioprocessing technologies to streamline production" on Bayer's cardiology and oncology pipeline,Judy Chou, former global head of Bayer Biotech, said in a May 2019 statement.

RELATED:Bayer looks for pharma growth in 2021 as new drugs start to take shape

Bayer has been a tenant in Berkeley, where it cranks out hemophilia A treatments, since the 1970s. The site received a $100 million facelift in 2017but was also victim ofjob cuts in 2018 as the companyreorganized manufacturing of its hemophilia factor VIII replacement therapiesKogenate, Kovaltry andJivi.

Meanwhile, Bayer has taken a page out of the M&A playbook as it looks to solidify its place in the cell and genefield. In October, the company agreed to pay $2 billion upfront and potentially $2 billion more in milestones to snap upadeno-associated virus (AAV) gene therapy specialist Asklepios BioPharmaceutical.

Asklepios, also known as AskBio, boasts a pipeline ofpreclinical and clinical candidates for neuromuscular, central nervous system, cardiovascular and metabolic diseases, and came on board withgene therapy manufacturing capabilities, which the company now uses in a CDMO capacity.

On the cell therapy front, Bayer in December teamed up withAtara Biotherapeutics to work on anoff-the-shelf T-cell immunotherapy fortough-to-treat lung cancers. That same month, the company launched a cell and gene platform to assist its swelling Rolodex of partners across the product lifecycle. And amid all that, Bayer co-leda $65 million funding round forgene editing player Metagenomi and a$105 million series Bfor next-gen gene therapy startup Senti Biosciences.

A little more than a year before that, Bayer laid out$240 million to nab theremaining stake in its cell therapy joint venture, BlueRock Therapeutics.

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Bayer, intent on its cell therapy ambitions, plots $200M plant at overhauled Berkeley campus: report - FiercePharma

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 -...

ARLINGTON, Va. (PRWEB) April 27, 2021

The market for selected technologies and supplies commonly used in cell and gene therapy (CGT) research, development, clinical trial, and product manufacturing exceeded $2.1 billion in 2020. This was the finding made in Cell & Gene Therapy Technologies and Supplies, a market research report by Strategic Directions International, part of Science and Medicine Group.

SDi's report breaks the market into specific categories with defined markets. The market is led by cell expansion products, driven by costly GMP-grade consumables that are necessary for the culture of cells that are destined for infusion into humans. Currently, clinical trials account for a greater portion of cell expansion revenues than approved clinical use, but this dynamic will shift as an increasing number of CGTs gain approval and enter the market.

Gene therapies can target any gene in the human genome and transfer modified genetic material to specific cells. It requires editing the cellular genetic code to change specific target cells. A viral vector like adeno-associated virus (AAV) carries genetic instructions to the cell of interest. Non-viral transport vectors can also be employed for the purpose of treating or curing disease. Typically, diseases treated with gene therapy have a single gene or protein aberration responsible for the disorder. Once in, the genetic instructions find the appropriate cells and gene addition, inhibition, correction, reprogramming, and/or cell elimination is induced. These types of gene therapies are common for immune diseases caused by protein production abnormalities, like arthritis, connective tissue disorders, and rare genetic disorders such as sickle cell.

In vivo gene therapy involves direct injection, grafting, or intravenous administration of a vector with therapeutic genetic material into the body. The process can be systemic or localized to a specific cell type or tissue of interest. Ex vivo gene therapy genetically modifies the cells of interest outside of the patients body, which is the basis of many cell therapies.

Hundreds of suppliers participate in the market for cell and gene therapies technologies and supplies. While the leading vendors are broad-based companies with offerings in all product categories, most participants provide more niche products. The top suppliers in the market in 2020 were Thermo Fisher, MilliporeSigma, Gibco (Thermo Fisher), and Cytiva (Danaher). The rapid development of the market has driven much innovation of new and existing technologies. This has created opportunities for market participants, and numerous new analytical instrumentation and supply companies have been formed to serve the CGT market.

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.

The report can be found at: https://strategic-directions.com/product/2021-cell-gene-therapy-technologies-and-supplies-report/

About Strategic Directions InternationalStrategic Directions International (SDi), part of Science and Medicine Group, is the leading business intelligence firm in the highly specialized field of analytical and life science instruments. Its client list includes virtually every major analytical instrumentation company in the United States, Europe, and Japan. Founded in 1981, the Los Angeles-based company has published hundreds of market reports and provided proprietary consulting services for a multitude of clients.

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2 Billion Dollar Tools Market for Cell and Gene Therapy, Says Report - PR Web

SAN DIEGO, April 28, 2021 (GLOBE NEWSWIRE) -- Otonomy, Inc.(Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced the upcoming presentation of preclinical proof-of-concept data for OTO-825 at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on May 13, 2021. OTO-825 is the clinical candidate targeting the gap junction beta-2 (GJB2) gene developed under the companys collaboration with Applied Genetic Technologies Corporation (Nasdaq: AGTC). Mutations in the GJB2 gene are the most common cause of congenital hearing loss and typically result in moderate to severe hearing impairment.

"We are excited to present these preclinical results for OTO-825 that build on our previous presentations demonstrating gene expression in support cells of the cochlear, which are the target cells for GJB2 gene therapy, using novel AAV vectors identified through our collaboration with AGTC, said Alan C. Foster, Ph.D., chief scientific officer of Otonomy. Based on these encouraging results that demonstrate hearing recovery and improved cochlear morphology following OTO-825 administration, the companies have initiated IND-enabling activities and look forward to providing additional details of the program in the next several months.

The oral presentation entitled AAV-mediated GJB2 gene therapy rescues hearing loss and cochlear damage in a mouse model of congenital hearing loss caused by conditional Connexin26 knockout by Mathur et al., will be presented on May 13 at 6:30 p.m. EDT.

About Otonomy

Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs with a focus on hearing loss and tinnitus. For additional information please visit http://www.otonomy.com.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or the future financial or operating performance of Otonomy. Forward-looking statements in this press release include, but are not limited to, statements related to plans and expectations regarding OTO-825; and statements by Otonomys chief scientific officer.

Otonomys expectations regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: delays and disruption resulting from theCOVID-19pandemic; Otonomys ability to obtain additional financing; the uncertainties inherent in the clinical drug development process, including, without limitation, Otonomys ability to adequately demonstrate the safety and efficacy of its product candidates and the nonclinical and clinical results for its product candidates, which may not support further development; the risks of the occurrence of any event, change or other circumstance that could impact the performance under or give rise to the termination of Otonomys collaboration, co-promotion or license agreements, including its collaboration agreement with AGTC, or that could impact Otonomys ability to repay or comply with the terms of the loan provided by Oxford Finance LLC; side effects or adverse events associated with Otonomys product candidates; competition in the biopharmaceutical industry; Otonomys dependence on third parties to conduct nonclinical studies and clinical trials, and for the manufacture of its product candidates; Otonomys ability to protect its intellectual property in the United States and throughout the world and to ensure compliance with various laws and regulations in countries in which it conducts clinical trials; expectations regarding potential therapy benefits, market size, opportunity and growth; Otonomys ability to manage operating expenses; implementation of Otonomys business model and strategic plans for its business, products and technology; general economic and market conditions;and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in Otonomys Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 11, 2021, and Otonomys future reports to be filed with the SEC. The forward-looking statements in this press release are based on information available to Otonomy as of the date hereof. Otonomy disclaims any obligation to update any forward-looking statements, except as required by law.

Contacts:

Media InquiriesSpectrum ScienceChlo-Anne RamseyVice President404.865.3601cramsey@spectrumscience.com

Investor InquiriesWestwicke ICRRobert H. UhlManaging Director858.356.5932robert.uhl@westwicke.com

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Otonomy and AGTC to Present Preclinical Proof-of-Concept Results for OTO-825 Gene Therapy at ASGCT Annual Meeting - GlobeNewswire

Nobel Laureate James P. Allison, PhD, Carl H. June, MD and Pulitzer-prize winning author Siddhartha Mukherjee, MD will be the keynote speakers at the virtual Alliance for Cancer Gene Therapy Summit 20

STAMFORD, Conn. (PRWEB) April 28, 2021

WHO: April 29 marks a pivotal moment in cancer research when the worlds brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers. The scientists and companies driving the latest advances in cancer cell and gene therapy will gather online for a virtual Summit hosted by Alliance for Cancer Gene Therapy, who envision a cancer free future and want to change the C-word from Cancer to Cure. The Summit is open to the public, to medical professionals, scientists and companies interested in cell and gene therapy to fight cancer. To register for the Summit, visit acgtfoundation.org.

Summit 2021 is being held online on Thursday, April 29, 2021, from 10:00 a.m. until 6:00 p.m (ET), and features eight (8) panel discussions with leading researchers developing the next generation cancer cell and gene therapies, biotech companies who are bringing new treatments through the clinic, and investors who are funding this burgeoning pipeline of solid tumor breakthroughs.

The keynote features a conversation with Nobel Laureate James P. Allison, PhD, executive director of the Immunotherapy Platform at MD Anderson Cancer Center, and Carl H. June, MD, director of the Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania. The conversation will be moderated by Siddhartha Mukherjee, MD, founder, Myeloid Therapeutics, Pulitzer-prize winning author of "The Emperor of All Maladies.

WHAT: Barbara Lavery, chief program officer with Alliance for Cancer Gene Therapy notes, Its so rare to have the opportunity to hear from the worlds top cancer researchers, biotechs, investors and patients all in the same room. Were excited to have Alliance for Cancer Gene Therapy be a catalyst for these important conversations that will help not only other scientists working on new cell and gene therapy approaches, but companies seeking investments to advance their clinical pipelines, and patients and their families looking for potentially life changing therapeutic options in their fight against cancer.

WHEN: THURSDAY, APRIL 29, 2021 (ALL TIMES ARE IN ET)

10 AM: Opening Remarks & Evolving Regulatory and Manufacturing Processes to Match the Speed of Innovation -- Panelists: Asthika Goonewardene, MBA, Amy DuRoss, MBA, Rafael Amado, PhD, Andr Choulika, PhD, Ke Liu, MD, PhD, Bruce Levine, PhD

11 AM: Innovating Science - from the Lab to the Clinic -- Panelists: Clodagh O'Shea, PhD, Jenna Foger, PhD, Yvonne Chen, PhD, Brian Brown, PhD, Franco Marincola, MD

12 PM: Tackling the Toughest Challenges - Pancreatic Cancer -- Panelists: Joseph Fraietta, PhD, Andrew Rakeman, PhD, Michael T. Lotze, MD, Sidi Chen, PhD, Mark O'Hara, MD

1 PM: Tackling the Toughest Challenges - Glioblastoma -- Panelists: Nduka Amankulor, MD, Crystal Mackall, MD, Noriyuki Kasahara, MD, PhD, Klaus Veitinger, MD, PhD, Samantha Bucktrout, PhD

2 PM: New Approaches to Solid Tumor Breakthroughs -- Panelists: Daniel Getts, PhD, Luke Timmerman, Garry E. Menzel, PhD, MBA, Ken Drazan, MD

3 PM: Patients, Caregivers, Doctors, Advocates, Researchers, you, Me - We All Have Cancer in Common -- Panelists: Gregory C. Simon, Tom Whitehead, Robert Levis, Caroline Corner, PhD, Callum Miller

4 PM: Innovating Finance - Non-Traditional Funding Sources -- Panelists: Ken Schaner, Marc Hurlbert, PhD, Luke Evnin, PhD, Anna Turetsky, PhD, Jay Campbell

5 PM: Keynote: Does Cancer Have a Future? Whats Next? Where Will We Be in 2025? -- Panelists: Siddhartha Mukherjee, MD, Carl H. June, MD, James P. Allison, PhD

WHERE: The Alliance for Cancer Gene Therapy Summit 2021 is VIRTUAL. To register visit Alliance for Cancer Gene Therapy Summit 2021 visit https://web.cvent.com/event/66b028f2-2d12-4ebf-86c9-bd2fca898e11/summary or acgtfoundation.org.

WHY: For 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit http://www.acgtfoundation.org, call 203-358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube @acgtfoundation.

NOTE: Members of the media who are interested in attending the event should contact summit@acgtfoundation.org.

The rest is here:
April 29 Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs - PR Web

SOLANA BEACH, Calif., April 28, 2021 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the Company), a global therapy-enabling platform company providing navigation and delivery to the brain, today congratulates Voyager Therapeutics on receiving FDA clearance of the IND application for their gene therapy candidate VY-HTT01 for the treatment of Huntingtons disease.

Huntingtons disease is a rare inherited neurodegenerative disorder impacting approximately 30,0001 people in the United States alone, with symptom onset commonly appearing between 30 and 50 years of age. Voyagers anticipated VYTAL Phase 1/2 clinical trial is a dose escalation study to evaluate the safety and tolerability of VY-HTT01 in patients with early manifest Huntingtons disease. The investigational gene therapy has been designed to be delivered throughout the brain via a one-time MRI-guided neurosurgical delivery.

The ClearPoint team is thrilled to continue supporting Voyagers clinical program development with products and clinical services for this important planned Phase 1/2 trial, stated Jeremy Stigall, Vice President, Biologics and Drug Delivery. We look forward to playing our part in providing hope to the Huntingtons disease community through our continued innovation and customer centric approach including patients, physicians and our biologics and drug delivery partners.

About ClearPoint Neuro

ClearPoint Neuros mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics, and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and is installed in over 60 active clinical sites in the United States, Canada, and Europe. The Companys SmartFlow cannula is being used in partnership or evaluation with over 25 individual biologics and drug delivery companies in various stages from preclinical research, to late-stage regulatory trials. To date, more than 4,000 cases have been performed and supported by the Companys field-based clinical specialist team, which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Forward-Looking Statements

Statements herein concerning the Companys plans, growth and strategies may include forward-looking statements within the context of the federal securities laws. Statements regarding the Company's future events, developments and future performance, as well as management's expectations, beliefs, plans, estimates or projections relating to the future, are forward-looking statements within the meaning of these laws. Uncertainties and risks may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to: the impact of COVID-19 and the measures adopted to contain its spread; future revenues from sales of the Companys ClearPoint Neuro Navigation System products; and the Companys ability to market, commercialize and achieve broader market acceptance for the Companys ClearPoint Neuro Navigation System products. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section in the Companys Annual Report on Form 10-K for the year ended December 31, 2020, which has been filed with the Securities and Exchange Commission, and in the Companys Quarterly Report on Form 10-Q for the three months ended March 31, 2021, which the Company intends to file with the Securities and Exchange Commission on or before May 17, 2021.

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1https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-receives-fda-clearance-ind-application-gene

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ClearPoint Neuro, Inc. Congratulates Voyager Therapeutics on FDA Clearance of IND Application for Gene Therapy Candidate VY-HTT01 for Treatment of...