StemCell Therapy

TipRanks

Investors have been fixated on growth companies over the past year, and one segment which has been on the rise is the fledgling cannabis industry. The sector offers a unique proposition and the prospect of further growth, as there is still a major catalyst on the horizon which will completely alter the industry. As expected, a Democrat led senate has been good news for those banking on marijuana reform at the federal level; And it looks like the anticipated changes could happen faster than initially expected. Backed by Senate majority leader Chuck Schumer, Democratic Senators have stated that they will push for federal-level legalization of marijuana, promising a unified discussion draft on comprehensive [cannabis] reform in the first half of this year. The statement feeds expectations that the Democratic Congressional majority will pass and that President Biden will sign a bill to legalize marijuana. Investors are also looking at further state-level legalization moves; one key state in this regard is New York. So, the cannabis industry is looking up. There is an expanding network of state legalization regimes, and expectations of a change in federal policy; both are putting upward pressure on cannabis shares. Against this backdrop, we used TipRanks database to find two cannabis stocks that have been earmarked as 'Strong Buys' by the analyst consensus. Both have posted impressive year-to-date performances, and stand to rise even more in the year ahead. Village Farms International (VFF) We will start with Village Farms International, a company that has long been involved in the niche agricultural business. The company started out as a farmer, producing high-quality greenhouse vegetables year-round for sale in the North American market. That background fit the company well for a transition to the cannabis industry Village Farms has experience in greenhouse production and industrial-scale growing. Village Farms shares are showing a tremendous growth profile, up 327% in the past 12 months with a strong spike in recent days. Two important pieces of news precipitated the surge since the end of January. First, the company has fully repaid ahead of schedule the $15 million debt it incurred during its November acquisition of the cannabis growing company Pure Sunfarms. And second, Village Farms increased its investment in the Asian cannabinoid company Altum by 50%, to hold a 10% stake in the company. The move increases the international reach of Village Farms, and its ability to increase Altum holdings in the future. The company was able to fund these moves because it had a successful equity sale in January, putting an additional 10.8 million shares on the market, and raising US$135 million in new capital. In addition to its strong capital and expansion positions, Village Farms has been reporting solid financial results. The company saw US$43 million in revenue for 3Q20, a gain of 12.5% year-over-year. EPS came in at 1 cent per share, a turnaround from the US$0.10 loss in the year-ago quarter. Covering Village Farms for Craig-Hallum, 5-star analyst Eric Des Lauriers writes: Village Farms has clearly established itself as the leading cannabis producer in Canada with #1 brand share and industry-leading profitability. Canadian cannabis sales in 2020 through October (latest available) were up 128% y/y, and dispensary counts are set to accelerate through 2021, providing a tailwind to VFF revenues. Turning to the US markets, and VFFs position in Canadas larger neighbor, the analyst goes on to add, With 5.7M SF of greenhouses in TX, the company also has real US optionality, which is finally being appreciated by investors after the GA election. VFF has historically been undervalued compared to less profitable peers, but we expect shares to continue working higher as the prospect for US reform increases throughout the year. To this end, Des Lauriers rates VFF a Buy, and his $25 price target suggests the stock has room for ~26% upside in the coming year. (To watch Des Lauriers track record, click here) Overall, there are 3 recent reviews on VFF shares, and all are Buys, giving the stock a Strong Buy analyst consensus rating and showing a general agreement on Wall Street about the companys strengths. Shares are priced at $19.90, and the $24.33 average price target implies an upside of ~23% for the year ahead. (See VFF stock analysis on TipRanks) TerrAscend Corporation (TRSSF) The next cannabis stock were looking at, TerrAscend, is another major cannabis producer in both the US, Canada, and Europe. The company is involved in both the medical and recreational sides of the market, and both grows and produces cannabis and markets a range of products through numerous brand names. TerrAscends US operations are located in California, Pennsylvania, New Jersey, and Utah, and the company looks to expand as more states legalize cannabis. In a strong sign of the cannabis industrys strength, TRSSF shares are up a sky-high 624% over the past 12 months. Growth has been fueled by expansion of the cultivation operations in California and Pennsylvania, and by the move into the adult-use recreational market in New Jersey. Last month, TerrAscend closed a non-brokered private placement stock sale, putting more than 18 million common shares on the market. The sale price was C$12.35 (US$9.72), and the offering grossed C$224 million (US$176.3 million). The bulk of the proceeds some 80% of the total was put up by four large US-based institutional investors. The funds raised will be used to continue expansion of the companys cultivation operations (TRSSF has plans to expand growing and manufacturing ops in New Jersey), as well as to pursue merger & acquisition activities. TerrAscends rapid growth and strong future prospects have attracted attention from top-rated analysts, including 5-star analyst Eric Des Lauriers of Craig-Hallum (stated above). "TerrAscend is a leading multi-state operator (MSO) in the US cannabis market with top-tier management, assets, and access to deal flow. We have been bullish on the company since initiating coverage last year and are happy to say the TRSSF team has exceeded our expectations, generating rapid increases in margins and operating leverage that have earned them a place solidly in the Top Tier of MSOs," Des Lauriers noted. The analyst summed up, "[With] US$280M+ raised since the elections and federal reform moving quicker than expected, we think TRSSF does deserve a premium to peers." In line with his bullish comments, Des Lauriers rates TRSSF shares a Buy, and has a $20 price target that implies a ~31% upside potential for the next 12 months. Once again, were looking at a stock with broad agreement from Wall Streets analysts the Strong Buy consensus rating is unanimous, based on 7 recent reviews. Shares are selling for $15.30, and their recent appreciation has pushed that price almost up to the $15.43 average price target. (See TRSSF stock analysis on TipRanks) To find good ideas for cannabis stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance

Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, has launched CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye."The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer, Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform."Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer, CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020) discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."The abstract in Nature is available here.Access to the NEJM article is available here.

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Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma

DetailsCategory: DNA RNA and CellsPublished on Monday, 08 February 2021 16:09Hits: 412

- Phase 1/2 trial expected to commence in first half of 2021

- Company has three INDs cleared for rare monogenic CNS disorders

PHILADELPHIA, PA, USA I February 08, 2021 I Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy being studied for the treatment of early infantile Krabbe disease (Globoid Cell Leukodystrophy). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a childs life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

As part of our commitment to deliver a transformative, one-time gene therapy to the children and their families who suffer from the devastating effects of Krabbe disease, we are excited to advance toward clinically evaluating the potential life-changing benefits of PBKR03, said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. The FDA clearance of our IND for PBKR03 is an important milestone for Passage Bio, paving the way for the start of our third clinical program in rare monogenic CNS disorders in the first half of 2021. Having solidified our clinical trial preparedness and manufacturing readiness during the past year, we are well-positioned to move with urgency to advance PBKR03 into the clinic.

PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C). Low GAL-C activity results in accumulation of psychosine which is toxic to the myelin-producing oligodendrocytes of the CNS and Schwann cells in the periphery, resulting in damage to both the central and peripheral nervous systems. PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.

Compelling preclinical data support advancement into clinical trials

PBKR03 is supported by extensive preclinical studies, conducted by our collaborator, the University of Pennsylvanias Gene Therapy Program, showing meaningful transduction of both the central and peripheral nervous system in animal models, with restoration of myelination in the brain and peripheral nerves. In a naturally occurring Krabbe animal model, a single ICM injection of an AAVhu68 capsid containing the normal canine GALC gene showed normalization of GALC activity, reduction of cerebral spinal fluid psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation and increased survival.

Phase 1/2 study anticipated for 1H21

Passage Bio expects to initiate a Phase1/2 clinical trial for PBKR03 in the first half of 2021. The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease. The primary endpoint of the Phase 1/2 study is safety and tolerability; secondary endpoints include CSF and serum GALC levels, disease biomarkers, and clinical outcome measures. Initial data from the trial is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.

PENN Financial Disclosure

The University of Pennsylvania (Penn) and its Gene Therapy Program receives sponsored research funding from Passage Bio, and Penn has licensed intellectual property to Passage Bio that may result in future financial returns to Penn.

About Krabbe Disease

Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues. The early infantile form of the disease is the most severe and common, typically manifesting before six months of age and accounting for 60 percent to 70 percent of diagnoses. In these patients, the disease course is highly predictable and rapidly progresses to include loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, deafness and eventual death by two years of age. Late infantile patients, defined by onset between seven to twelve months of age, present similar symptoms and have a median survival of approximately five years from onset of symptoms. The estimated worldwide incidence of Krabbe disease is 2.6 in 100,000 births, which is higher than reported due to lack of adequate screening at birth.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

SOURCE: Passage Bio

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FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare...

PROVIDENCE, R.I.[Brown University] New research provides insights into the treatment of Christianson syndrome (CS), an X-linked genetic disease characterized by reduced brain growth after birth, intellectual disability, epilepsy and difficulties with balance and speech.

One of the major challenges in developing treatments for human brain disorders, like CS, is developing an experimental system for testing potential therapeutics on human neurons, said study senior author Dr. Eric Morrow, an associate professor of molecular biology, neuroscience and psychiatry at Brown University. In recent years, advanced stem cell therapies that use tissues from patients have provided powerful new approaches for engineering human neurons from the patients themselves, who may undergo the treatment in the future.

For the study, published in Science Translational Medicine on Feb. 10, 2021, Morrow and his colleagues obtained blood samples from five CS patients and the patients unaffected brothers. They then reprogrammed these blood cells into stem cells, and these stem cells were converted into neurons in a petri dish. As a result, they obtained neurons that were representative of those from CS patients, and they used these neurons to test treatments.

Morrow who directs the Center for Translational Neuroscience at the Carney Institute for Brain Science and the Brown Institute for Translational Science said the team also used a new gene-editing approach that employs CRISPR-Cas9 technologies to correct patient mutations back to a healthy gene sequence.

CS is caused by a mutation in a gene encoding for NHE6, a protein that helps regulate acid levels within cell structures called endosomes. Past research suggests that the loss of NHE6 causes endosomes to become overly acidic, which disrupts the abilities of developing neurons to branch out and form connections in the growing brain.

Loss of this important protein can arise from a variety of gene mutations in patients. The majority of CS mutations are called nonsense mutations, which prevent NHE6 from being produced at all; four of the five CS patients involved in this study exhibited this class of mutation. However, some CS patients exhibit missense mutations. Individuals with missense mutations still have some NHE6, but it is produced in smaller amounts, and the protein fails to function as it should.

The research team tested two main forms of treatment on the stem-cell-derived neurons: first, gene transfer, which involves adding a healthy NHE6 gene into the cell; and second, administration of trophic factors, which are substances that promote neuron growth and encourage neurons to develop connections with other neurons. The researchers found that the neurons response to treatment depended on the class of mutation present.

The gene transfer studies, which may represent the first steps toward developing gene therapy, were successful in neurons with nonsense mutations. After the researchers inserted a functional NHE6 gene into nonsense-mutation CS neurons, the neurons branched out properly. In neurons with missense mutations, however, gene transfer failed completely. Further tests suggested that the abnormal NHE6 produced as a result of missense mutations may interfere with normal NHE6, thereby rendering gene transfer therapy ineffective in patient cells with these mutations.

In contrast, administration of trophic factors, such as brain-derived neurotrophic factor (BDNF) and insulin-like growth factor-1 (IGF-1), successfully promoted proper branching in all the CS neurons studied, regardless of mutation type.

While these initial results are encouraging, Morrow hopes that future studies will examine these treatments in animal models.

Our results provide an initial proof-of-concept for these treatment strategies, indicating that they should be studied further, he said. However, we may ultimately need to pay close attention to the class of mutation that a patient has when we choose a specific treatment.

In addition to Morrow, the research team included scientists from Brown University, the University of South Carolina and the Icahn School of Medicine at Mount Sinai. The study was supported by multiple grants from the National Institutes of Health as well as a number of awards from foundations and academic institutions.

This news story was authored by contributing science writerKerry Benson.

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Neurons from patient blood cells enable researchers to test treatments for genetic brain disease - Brown University

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

Breyanzi (lisocabtagene maraleucel) was approved on the 54 percent complete remission rate achieved in diffuse large B-cell lymphoma trials.

Breyanzi (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or relapsed, after at least two other types of systemic treatment has been approved by the US Food and Drug Administration (FDA).

According to the agency, Breyanzi is the third gene therapy approved in the US for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma (DLBCL). Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.

Todays approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes, commented Dr Peter Marks, director of the FDAs Center for Biologics Evaluation and Research. Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens.

DLBCL is the most common type of non-Hodgkin lymphoma in adults. Approximately 77,000 new cases of non-Hodgkin lymphoma are diagnosed in the US each year, with DLBCL accounting for around a third of newly diagnosed cases.

Breyanzi is customised for each patient; their T cells, a type of white blood cell, are collected and genetically modified to include a new gene that facilitates targeting and killing of the lymphoma cells. Once the cells are modified, they are infused back into the patient.

The safety and efficacy of the treatment were established in a multi-centre clinical trial of more than 250 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Breyanzi was 54 percent.

The treatment can cause severe side effects, including cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T cells, causing high fever and flu-like symptoms and neurologic toxicities. Both CRS and neurological events can be life-threatening, so the therapy is being approved with a risk evaluation and mitigation strategy (REMS) which includes elements to assure safe use (ETASU).

The requirements include, among other things, that healthcare facilities that dispense Breyanzi be specially certified, with staff involved in the prescribing, dispensing or administering of the treatment being trained to recognise and manage the risks of CRS and neurologic toxicities.

Other side effects include hypersensitivity reactions, serious infections, low blood cell counts and a weakened immune system. According to the FDA, side effects generally appear within the first one to two weeks following treatment, but some side effects may occur later.

To further evaluate the long-term safety, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Breyanzi.

The approval was granted to Juno Therapeutics Inc., a Bristol-Myers Squibb Company.

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FDA approves third gene therapy for large B-cell lymphoma - European Pharmaceutical Review

REDWOOD CITY, Calif., Feb. 10, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). These data were previously presented as part of a corporate update on Saturday, November 14, 2020.

Oral Presentation Title: Intravitreal Gene Therapy for Exudative AMD and Diabetic RetinopathyDate and Time: Saturday, February 13, 2021 at 9:30 am ETSession V: Gene Therapy for Exudative AMD and Diabetic RetinopathyPresenter: Arshad M. Khanani, M.D., M.A., Director of Clinical Research, Sierra Eye Associates

A copy of this presentation will be available as the presentation begins in the publications and presentation section of Adverums website.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visitwww.adverum.com.

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Adverum to Present Data from the OPTIC Phase 1 Trial with ADVM-022 Intravitreal Gene Therapy in Wet AMD at the Angiogenesis, Exudation, and...

Gene therapy companies have been under pressure lately, but Orchard Therapeutics got a lift yesterday from promising early data with its mucopolysaccharidosis type I candidateOTL-203.

The company is seeking to supersede the current standard of care, enzyme-replacement therapy or bone marrow transplant. But other gene therapy contenders are not too far behind, notablyRegenxbio, which in December started a proof-of-concept study of its rival project, RGX-111.

Good IDUA

Both projects seek to deliver the -l-iduronidase (IDUA) gene, which is mutated in MPS-I, leading to a deficiency of the IDUA enzyme. This enzyme usually breaks down glycosaminoglycans (GAGs), so in MPS-I patients these build up, causing tissue and organ damage. Symptoms of MSP-I, also known as Hurler syndrome, include cognitive impairment and skeletal deformity; if left untreated, patients rarely survive beyond the age of 10.

And both OTL-203 and RGX-111 are designed as one-time therapies, whereas the current enzyme replacement, Biomarin/Sanofis Aldurazyme, is given intravenously once a week.

However, the gene therapy candidates go about restoring IDUA enzyme activity in different ways. OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused.

RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot cross the blood-brain barrier.

Getting into the brain should not be a problem for OTL-203 either, Orchards head of medical affairs, Leslie Meltzer, told Evaluate Vantage. She explained that hematopoietic stem cells naturally cross the blood-brain barrier and, once in the CNS, differentiate into a microglial-like cell.

This claim appears to be supported by the latest data, which admittedly come in just a handful of subjects. The eight-patient phase I/II trial, presented at the World Symposium yesterday, found increases in the IDUA enzyme in patients blood and cerebrospinal fluid. There was also a decrease in GAGs in the CSF and urine.

Encouragingly, this activity appears to have translated into a clinical benefit: all eight patients showed stable cognitive scores and stable motor function versus baseline, as well as growth in the normal range for patients age.

Its a progressive disease, so youd expect these things to worsen over time, but the fact they continued to be stable is very promising, Ms Meltzer said.She admitted that the data were early, with only around a year of follow-up on most of the clinical endpoints.

Orchard plans to start a registrational study by the end of this year.Ms Meltzer would not give any details ondesign, saying this would be finalised after feedback from regulators.

Regenxbios proof-of-concept study of RGX-111 is due to complete in November, putting the project about a year behind OTL-203.

One candidate that will go no further is Sangamos SB-318. The company reported disappointing data with the in vivo zinc finger nuclease genome-editing project two years ago, and has since said it would focus on second-generation zinc finger projects.

Still, even two gene therapies might be too many for an ultra-rare disease like MPS-I, which affects just one in 100,000 people. Asked whether this market could support more than one gene therapy, Ms Meltzer said newborn screening recently implemented in countries including the UScould lead to a revision of that estimate.

But, as in other rare disorders that have attracted several gene therapy players, a battle over a limited patient pool could be shaping up.

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World Symposium Orchard leads the crop of Hurler syndrome hopefuls - Vantage

Article reflects Companys leadership and innovation in scalable, reproducible manufacture of adeno-associated virus (AAV)-based gene therapies

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, announced that Sue Washer, President & Chief Executive Officer and Dave Knop, Vice President of Process Development, have been awarded first place in the BioProcess International (BPI) magazine inaugural Readers Choice Awards program, cell and gene therapies category, for their article, Viral-Vectored Gene Therapies: Harnessing Their Potential Through Scalable, Reproducible Manufacturing Processes.

High-productivity approaches to AAV manufacturing processes, like AGTCs HSV-helper based platform, will be crucial if we are to address the unmet clinical need growing across a variety of indications, said AGTC President and CEO, Sue Washer. There is no question that investing in the manufacturing process is imperative and our early commitment in this area has put AGTC in a strong position with respect to the purity and quality needed for late stage development and commercialization.

Concentrating on articles published from September 2019 through June 2020, and using rankings based on views, engagement, and download rates, BioProcess International identified the four most popular articles within each of its six pillars of bioprocessing coverage. The AGTC authors article received the highest number of votes from BPI readers, who ranked the nominees in terms of their innovativeness, presentability and applicability.

The eBook featuring the first-place article by Washer and Knop, as well as summarized versions of the second- and third-place articles, are available by visiting: https://bioprocessintl.com/wp-content/uploads/2020/11/18-11-eBook-RCA-CellGeneTherapies.pdf.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its preclinical programs build on the Companys industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS:David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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AGTC Executives Awarded First Place in the BioProcess International Reader's Choice Awards, Cell & Gene Therapies Category - GlobeNewswire

NEW YORK, Feb. 11, 2021 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today highlighted its presence at the 2021 Transplantation & Cellular Therapy (TCT) Annual Meeting, which is being held virtually from February 8th 12th. The TCT meeting organizes thousands of transplant professionals from over five hundred transplant centers worldwide and is a seminal event for Actinium given its focus on targeted conditioning for bone marrow transplant (BMT), CAR-T and other adoptive cell therapies and gene therapy. At TCT, Actinium's pivotal Phase 3 trial SIERRA trial for Iomab-B was featured in 2 oral presentations, as well as CME event focused on AML and BMT and in investigator interactions led by Actinium's clinical development and medical affairs teams.

Dr. Mark Berger, Actinium's Chief Medical Officer, said, "TCT is the ideal venue to showcase Actinium's Iomab-B and Iomab-ACT targeted conditioning programs given the concentrated audience of thought leaders in these fields that TCT brings together. The timing of TCT is also ideal as it follows shortly after ASH resulting in a data rich period for Actinium that drives investigator interest. This is particularly the case this year as we have built strong momentum in SIERRA following positive data from 75% enrollment featured in 2 oral presentations at ASH and now in 2 oral presentations at this year's TCT, which has driven high levels of investigator and referring physician interactions. We have coupled this with bolstered outreach efforts, which will continue beyond TCT, that have resulted in new site activation despite the advanced stage of SIERRA and robust enrollment rates that give us great confidence in completing SIERRA enrollment rapidly."

Summary data presented in TCT oral presentations include:

-100% BMT and engraftment rate for patients receiving a therapeutic dose of Iomab-B compared to 18% of patients receiving physician's choice of salvage therapy on the control arm- 79% of all patients enrolled on SIERRA were able to proceed to BMT despite being a patient population not considered eligible for BMT with standard approaches-Iomab-B delivers high amounts of targeted radiation to the bone marrow with minimal impact on other organs resulting in lower rates and severity of adverse events

TCT Oral Presentation: Targeted Radioimmunotherapy with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] in Older Patients with Active, Relapsed or Refractory (R/R) Acute Myeloid Leukemia Results in Successful and Timely Engraftment Not Related to the Radiation Dose Delivered

Phase 3 SIERRA 75% Enrollment Results

Baseline Characteristics

Iomab-B Arm(N=56)

Conventional Care (CC) Arm(N=57)

Age (yrs, median, range)

63 (55-77)

65 (55-77)

Cytogenetic and Molecular Risk1, 2

Favorable: 4%Intermediate: 35%

Adverse: 61%

Favorable: 5%

Intermediate: 32%

Adverse: 63%

% TransplantedIntent-to-Treat Group

88% (49/56)

18% (10/57)

64% (30/47)

Results

Underwent Iomab-B based Conditioning and HCT (N=49)3

Achieved CR and received standard of care HCT (N=10)

Randomized to Conventional Care and Crossed Over to Iomab-B with HCT (N=30)4

Cross-over Rate

n/a

n/a

Received Therapeutic Dose of Iomab-B (N=30)

Transplanted (N=30)

64% (30/47)

% Transplanted

100% (49/49)

18% (10/57)

100% (30/30)

% Marrow Blast @ randomization (median, range)

29% (4-95)5

20% (5-97)

28% (6-87)

Days to ANC Engraftment

14 (9-22)6

17 (13-83)7

14 (10-37)8

Days to Platelet Engraftment

18 (4-39)6

22 (8-35)7

19 (1-38)8

Days to HCT (Post Randomization)

30 (23-60)

67 (52-104)

62 (36-100)9

Myeloablative Dose Delivered to Bone Marrow

14.7 (4.6-32) Gv

n/a

15.5 (6.3-42) Gv

592 (313-1013) mCi

646 (354-1027) mCi

100-day non-Relapse Transplant-Related Mortality

4%

(2/45 Evaluable)

20%

(2/10 Evaluable)

10.7%

(3/28 Evaluable)

1) Iomab-B arm: data unavailable (4) and patient was excluded (1)

2) Per NCCN guidelines version 3. 2020

3) No therapy dose (7) due to: declining KPS (4), Infusion reaction (1), unfavorable biodistribution (1), post- randomization eligibility (1). Two (2) did not receive DI and five (5) received DI without proceeding to TI.

4) Thirteen (13) patients ineligible for crossover due to: hospice care/progression (4), declined/ineligible for HCT (5), died pre-crossover (4). Additionally, four (4) patients were eligible for crossover and did not receive Iomab-B due to declining KPS.

5) One (1) patient with 4% blasts in the marrow had circulating AML blasts

6) ANC engraftment data not available (4), platelet engraftment data not available (7)

7) ANC and platelet engraftment data not available (1)

8) ANC engraftment data not available (1), platelet engraftment data not available (2)

9) One (1) patient at 161 days had delayed transplant due to infection & respiratory failure, received Iomab & transplant when stable, not included in range

https://tct.confex.com/tct/2021/meetingapp.cgi/Paper/16878

TCT Oral Presentation: Myeloablative Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Spares the GI Tract and Has Low Incidence of Severe Mucositis, Febrile Neutropenia and Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Adverse Event

Iomab-B Arm (N=56)

Conventional Care Arm (N=57)

Received Iomab-B/HCT

(N=49)1

Achieved CR and received Std HCT (N=10)

No CR Crossed over to Iomab-B/HCT (N=30)

Sepsis

% (N)

4.2 (2)*

30.0 (3)

23.3 (7)

Febrile Neutropenia Gr 3-4

% (N)

41.7 (20)

50.0 (5)

40.0 (12)

Mucositis Gr 3-4

% (N)

10.4 (5)

30.0 (3)

16.7 (5)

Day +100 Non-Relapse Mortality3

2/45(4.4%)

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Actinium Showcases Targeted Conditioning Program with 2 Oral Presentations Highlighting Iomab-B and Pivotal Phase 3 SIERRA Trial at 2021...

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- In-depth analysis and data-driven insights on the impact of COVID-19 included in this Europe cell and gene therapy market report.

The Europe cell and gene therapy market is expected to grow at a CAGR of over 23% during the period 20202026.

Key Highlights Offered in the Report:

Key Offerings:

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Europe Cell and Gene Therapy Market Segmentation

Europe Cell and Gene Therapy Market by Product

Europe Cell and Gene Therapy Market by End-user

Europe Cell and Gene Therapy Market by Application

Europe Cell and Gene Therapy Market Dynamics

Cell and gene therapy is revolutionizing the global healthcare segment. Although various new cell and gene therapies are approved, there are various hurdles that limit the penetration of new therapies, such as high cost, multiple regulatory hurdles, and other manufacturing challenges. These cell and gene therapy developers need reliable, efficient, and cost-effective manufacturing services with the flexibility to scale up production as the demand increases. Cell and gene therapy products are very complex, and their manufacturing requires skilled labor, developed infrastructure for limited patients. Such huge investments will affect vendors and contract manufacturing organizations (CMOs) work with companies to overcome these challenges.

Key Drivers and Trends fueling Market Growth:

Europe Cell and Gene Therapy Market Geography

European countries such as Germany, France, the UK, Italy, and Spain play a significant role in the cell and gene therapy market. However, clinical trials and the number of manufacturing facilities are increasing slowly in Europe. Europe has become a major R&D destination for many vendors as the funding for cell and gene therapies is increasing across many European countries. Europe stands next to North America in the global cell and gene therapy market. Initially, Europe led the cell and gene therapy market due to first product approvals. France, Germany, and Italy had a greater contribution globally and in Europe. However, from the past decade, the US has competed and increased its market share globally. Europe stands second in the market, with the increasing prevalence of cancer and rare genetic disorders that are not effectively solved by the conventional therapies are increasing in the region. This increased target population is driving the demand for cell and gene therapy in the region.

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Europe Cell and Gene Therapy Market by Geography

Major Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

Explore our healthcare & lifesciencesprofile to know more about the industry.

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AriztonAdvisory and Intelligence is an innovation and quality-driven firm, which offers cutting-edge research solutions to clients across the world. We excel in providing comprehensive market intelligence reports and advisory and consulting services.

We offer comprehensive market research reports on industries such as consumer goods & retail technology, automotive and mobility, smart tech, healthcare, and life sciences, industrial machinery, chemicals and materials, IT and media, logistics and packaging. These reports contain detailed industry analysis, market size, share, growth drivers, and trend forecasts.

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Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire

Global Retinal Gene Therapy Market: Overview

The retinal gene therapy market is estimated to expand at an exponential growth rate. For the use of gene therapy, retina is considered a highly desirable target as it an irreplaceable part of a body. The global retinal gene therapy market is likely to be influenced by the promise its holds for the treatment of various forms of inherited and non-inherited blindness. Furthermore, this therapy can also be used in the treatment of rare genetic retinal diseases, such as Leber's congenital amaurosis, which is likely to augur well for the development of the global retinal gene therapy market during the forecast period, from 2020 to 2030. It is expected that the global retinal gene therapy market is anticipated to witness the entry of new players, with the presence of promising candidates in the phases of drug approval process.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Type, application, and region are the three important parameters based on which the global retinal gene therapy market has been classified. Such detailed analysis of the market comes with the sole purpose to provide stakeholders with a detailed and clear analysis of the global retinal gene therapy market.

Read Report Overview - https://www.transparencymarketresearch.com/retinal-gene-therapy-market.html

Global Retinal Gene Therapy Market: Notable Developments

One of the important market developments that give a quick view of the dynamics pertaining to the global retinal gene therapy market is mentioned as below:

There is only one player in this global retinal gene therapy market, which is mentioned as below:

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Global Retinal Gene Therapy Market: Key Trends

The global retinal gene therapy market is characterized by the presence of the following restraints, drivers, and opportunities.

Advent of High-end Technologies to Support Development of the Market

Mostly in the cases of inherited retinal diseases, retinal gene therapy is performed. Gene therapy is capable of bettering vision impairment through mutation in RPE65 gene. Luxturna, a recently introduced gene therapy is utilized for the treatment of patients suffering from type 2 Leber's congenital amaurosis. This disease is a form of inherited disease that causes impairment in vision at the time of birth, which leads to a highly progressive degeneration. At present, there are many retinal gene therapy at the clinical trial phase and those are utilizing recombinant viruses. This factor is likely to increase the scope of growth for the global retinal gene therapy market over the period of assessment, from 2020 to 2030.

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In addition to that, the emergence of new market players together with the advent of high-end technological developments is likely to encourage growth of the global retinal gene therapy market during the forecast period. It is estimated that retinal gene therapy is likely to come up as a standard form of treatment for such retina-related diseases.

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Global Retinal Gene Therapy Market: Geographical Analysis

North America is clearly at the forefront of the growth of the global retinal gene therapy market at the very moment. It is estimated that the region will continue to retain its dominance over the period of forecast, from 2020 to 2030. So far, the product that has been approved for use is from a manufacturer from this region. Europe is likely to emerge as another lucrative region in the global retinal gene therapy market over the period of forecast.

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Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace

CHICAGO, Feb. 2, 2021 /PRNewswire/ -- Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, today announced the launch of CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye.

"The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer of Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."

CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.

The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform.

"Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer of CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.

CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020)discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."

The abstract in Nature is available here: https://www.nature.com/articles/s41586-020-2201-4

Access to the NEJM article is available here: https://www.nejm.org/doi/full/10.1056/NEJMcibr2027602

About CiRC Biosciences CiRC Biosciences is a privately held cell therapy company dedicated to developing treatments for serious diseases with high, unmet needs with an initial focus on the eye. Currently it is pre-clinical phase for Geographic Atrophy Age-Related Macular Degeneration (Dry AMD) and advanced Retinitis Pigmentosa (RP). CiRC Biosciences is a portfolio company of Paragon Biosciences. Visit our website: https://circbiosciences.com/.

About Paragon Biosciences Paragon is a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the replicable Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contact:

Evelyn M. O'Connor Paragon Biosciences 312-847-1335 [emailprotected]

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Paragon Biosciences Launches CiRC Biosciences to Expand Cell and Gene Therapy Platform - PRNewswire

London, Feb. 02, 2021 (GLOBE NEWSWIRE) -- Roots Analysis has announced the addition of Gene Therapy Market (4th Edition), 2020-2030 report to its list of offerings.

Success of approved gene therapies has resulted in a surge in interest of biopharmaceutical developers in this rapidly evolving domain. Presently, the ability of gene therapies to treat diverse disease indications is considered among the most prominent drivers of this market. In addition, promising clinical results of pipeline candidates are anticipated to draw in more investments to support product development initiatives.

To order this 720+ page report, which features 220+ figures and 375+ tables, please visit this link

Key Market Insights

Around 800 gene therapies are currently being developed across different stages Apart from 10 approved products, most of the aforementioned therapies (65%) are in the early stages of development (discovery / preclinical), while the rest are being evaluated in clinical trials. It is worth mentioning that more than 40% of clinical stage candidates are intended for the treatment of oncological disorders.

Over 65% of innovator companies focused on gene therapy development, are based in North AmericaInterestingly, more than 75 players based in the same region, are start-ups, while over 35 are mid-sized players, and 10 are large and very large firms. Since the majority of gene therapy developers are headquartered in the US, it is considered a key R&D hub for such advanced therapy medicinal products.

There are 400+ registered gene therapy focused clinical trials, worldwideClinical research activity, in terms of number of trials registered, is reported to have increased at a CAGR of 12% during the period 2015-2020. Of the total number of trials, close to 25% have already been completed, and 35% claim to be actively recruiting.

USD 25.4 billion has been invested by both private and public investors, since 2015So far, a significant proportion of the capital raised has been through secondary offerings (USD 12.9 billion). On the other hand, around USD 5 billion was invested by venture capital investors, representing 20% of the total amount.

Close to 20,000 patents have been filed / published related to gene therapies, since 2016Around 30% of the total number of applications were related to gene editing-based therapies, while the remaining were associated with gene therapies. Further, majority of the patent assignees were industry players, however, the contribution of non-industry players in the overall patent filing activity has increased considerably (CAGR of 16%), over the past few years.

There have been several mergers and acquisitions in this market during the period 2015-2019 In fact, M&A activity is reported to have increased at a CAGR of more than 40%. Key drivers of the acquisitions mentioned in the report include, therapeutic area expansion, access to a novel technology / platform, drug class consolidation and drug class expansion.

North America and Europe are anticipated to capture over 90% of the market share, in terms of sales revenues, in 2030In vivo gene therapies currently represent a significant share of the market, and this trend is unlikely to change in the foreseen future, as several such candidates are being evaluated in late stages. In addition, more than 130,000+ patients are projected to use gene therapies in 2030 and the demand for gene therapies is expected to grow at an annualized rate of 29% and 31% during the periods 2020-2025 and 2025-2030, respectively.

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Key Questions Answered

The USD 14.6 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom, gene therapies exhibit the potential to become a promising alternative for the treatment of genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research includes brief profiles of key players (listed below) engaged in the development of gene therapies; each profile features an overview of the therapy, current development status, clinical trials and its results (if available), target indication, route of administration, and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html or email sales@rootsanalysis.com

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The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -...

NOIDA, India, Jan. 29, 2021 /PRNewswire/ -- A comprehensive overview of the gene therapy market is recently added by UnivDatos Market Insights to its humongous database. The gene therapy market report has been aggregated by collecting informative data of various dynamics such as market drivers, restraints, and opportunities. This innovative report makes use of several analyses to get a closer outlook on the gene therapy market. The gene therapy market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth. Furthermore, this statistical market research repository examines and estimates the gene therapy market at the global and regional levels. The Global Gene therapy Market is expected to grow at a CAGR of 29.7% from 2021-2027 to reach US 20.9 billion by 2027.

Market Overview

Gene therapy is the next trend of curative transformation in the life sciences industry. Globally, around 2,600 clinical trials in gene therapy have been performed, are underway, or have been approved to date. More than ever, the field of gene therapy seeks to identify a route to the clinic and the market. Approximately 20 gene therapies have now been licensed and over two thousand clinical trials of human gene therapy have been published globally. Aging populations worldwide and socio-economic risk factors are among the primary influences driving this surge.

As per Alliance for Regenerative Medicine (ARM) Quarterly Regenerative Medicine Global Data Report Q12019, 372 gene therapy clinical trials were in progress as of the end of Q1. Remarkably, a margin (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%). The number of gene therapy clinical trials edged up by 10 from the 362 recorded as of the end of 2018.

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Covid-19 Impact:

The COVID-19 pandemic has dislocated global management attempts across gene therapies. The manufacture and delivery of treatments, research and clinical development, and commercial operations are the three areas within the gene therapy sector that have been most interrupted amid the COVID-19 crisis. The development of gene therapies has been less affected. For instance, Peter Marks, Director of FDA's Center for Biologics Evaluation and Research (CBER) stated that with the arrival of therapies for cell and gene therapies over the last five years, it should have doubled in size while it is only modestly larger, 15-20% larger in size.

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Global Gene therapy Market report is studied thoroughly with several aspects that would help stakeholders in making their decisions more curated.

By Vector, the market is primarily bifurcated into

The viral vector segment dominated the gene therapy vector market in 2019 and will grow at 29.2% CAGR to reach US$ 17.9 billion by the year 2027.

By Viral Vector, the market is primarily sub-segmented into

Amongst viral vector types, adeno-associated virus accounted for the largest share and is expected to grow at 29.3% CAGR during the forecast period 2021-2027. In 2019, the adeno-associated virus segment accounted for a revenue share of almost 34%.

By Gene Type, the market is primarily studied into

In 2019, the antigen segment dominated the global gene therapy market with nearly 19.2% of the market share and it is anticipated by 2027, the segment will garner US$ 3.9 billion of the market.

By Indication, the market is primarily studied into

In 2019, the oncology segment dominated the global gene therapy market by indication with nearly 48.6% of the market share and it is anticipated to grow at 27.6% CAGR during the forecast period 2021-2027.

By Delivery Method, the market is primarily segmented into

Amongst delivery method, In vivo accounted for the largest share and is expected to grow at 28.6% CAGR during the forecast period 2021-2027. In 2019, the ex vivo segment accounted for a revenue share of 12.5%.

Gene therapy Market Geographical Segmentation Includes:

Based on the estimation, the North America region dominated the gene therapy market with almost US$ 1.7 billion revenue in 2019. At the same time, the Asia-Pacific region is expected to grow remarkably with a CAGR of 28.7% over the forecast period on account of owing increasing government initiative to improve healthcare infrastructure and rise in healthcare expenditure and surging cancer incidence rate in the region.

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The major players targeting the market includes

Competitive Landscape

The degree of competition among prominent global companies has been elaborated by analyzing several leading key players operating worldwide. The specialist team of research analysts sheds light on various traits such as global market competition, market share, most recent industry advancements, innovative product launches, partnerships, mergers, or acquisitions by leading companies in the gene therapy market. The leading players have been analyzed by using research methodologies for getting insight views on global competition.

Key questions resolved through this analytical market research report include:

We understand the requirement of different businesses, regions, and countries, we offer customized reports as per your requirements of business nature and geography. Please let us know If you have any custom needs.

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Gene Therapy Market to Reach US$ 20.9 Billion by 2027, Globally |CAGR: 29.7%|UnivDatos Market Insights - PR Newswire India

How many aging-promoting genes are there in the human genome? What are the molecular mechanisms by which these genes regulate aging? Can gene therapy alleviate individual aging? Recently, researchers from the Chinese Academy of Sciences have shed new light on the regulation of aging.

In this study, the researchers conducted genome-wide CRISPR/Cas9-based screens in human premature aging stem cells and identified more than 100 candidate senescence-promoting genes. They further verified the effectiveness of inactivating each of the top 50 candidate genes in promoting cellular rejuvenation using targeted sgRNAs.

Among them, KAT7 encoding a histone acetyltransferase was identified as one of the top targets in alleviating cellular senescence. It increased in human mesenchymal precursor cells during physiological and pathological aging. KAT7 depletion attenuated cellular senescence whereas KAT7 overexpression accelerated cellular senescence.

Altogether, this study has successfully expanded the list of human senescence-promoting genes using CRISPR/Cas9 genome-wide screen and conceptually demonstrated that gene therapy based on single-factor inactivation is able to delay individual aging. This study not only deepens our understanding of aging mechanism but also provides new potential targets for aging interventions.

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Delay aging and extend our lifespans? Gene therapy might be able to do that - Genetic Literacy Project

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006

Enrollment of approximately 20 total study participants is ongoing

PHILADELPHIA, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the dosing of the first participant in the Phase 1/2 RESOLUTESM trial of SPK-3006, an investigational liver-directed adeno-associated viral (AAV) vector gene therapy for late-onset Pompe disease (LOPD), a rare, inherited lysosomal storage disorder.

Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and first step to what we hope will ultimately allow us to bring an innovative gene therapy to these patients, said Gallia G. Levy, M.D., Ph.D., chief medical officer of Spark Therapeutics. We are deeply appreciative of the ongoing collaboration of the Pompe disease community as we continue to enroll participants in this Phase 1/2 study.

The RESOLUTE trial is an open-label Phase 1/2, dose-escalation gene transfer study designed to evaluate the safety, tolerability and efficacy of a single intravenous infusion of investigational SPK-3006, an AAV vector-based gene therapy, developed in collaboration with Genethon, in adults with clinically moderate LOPD currently receiving enzyme replacement therapy. The study is expected to enroll approximately 20 participants receiving the investigational gene therapy in sequential, dose-level cohorts. Additional details are available on ClinicalTrials.gov (NCT04093349).

We are honored to have the first participant dosed in this clinical trial, which we hope will lead us to introduce a novel therapeutic option for patients living with late-onset Pompe disease, said Principal Investigator Tahseen Mozaffar, M.D., University of California Irvine Health.

The International Pompe Association has been proud to collaborate with Spark Therapeutics to enhance the Pompe disease communitys understanding of gene therapy research, said Tiffany House, International Pompe Association Board Chairman. We look forward to the progress in the Phase 1/2 RESOLUTE trial, as well as the ongoing work aimed at developing gene therapies that have the potential to help individuals living with genetic diseases.

Pompe disease is a rare, inherited lysosomal storage disorder. It is a progressive, often life-limiting disease caused by the buildup of a complex sugar, glycogen, in the bodys cells. Mutations in the gene encoding acid alpha-glucosidase (GAA) result in deficiencies of the GAA enzyme and limit the breakdown of glycogen. For patients living with LOPD, the respiratory system, locomotion and maintenance of gait are the most critically impacted. These symptoms commonly result in patients becoming wheelchair bound and requiring respiratory support, which may result in reduced life-expectancy.

About SPK-3006 for Pompe diseaseSPK-3006is an investigational liver-directed AAV gene therapy for the potential treatment of late-onset Pompe disease (LOPD).SPK-3006has been engineered to produce a modified enzyme (secretable GAA) that is produced by the liver, which may result in sustained GAA plasma levels and could potentially provide greater uptake in muscle tissue. The transgene integrates technologies designed at and licensed from Genethon, where the in-vivo proof of concept in pre-clinical models was demonstrated. Spark Therapeutics retains global commercialization rights toSPK-3006.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanocommunications@sparktx.com(215) 294-9942

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Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace

Nottingham, UK 2nd February 2021 Life Science Newswire Albumedix Ltd. (Albumedix), an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with the Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

This agreement reflects the continued efforts of Albumedix to engage with the industry and expand upon its knowledge in the field, and the CGT Catapults mission to drive the growth of the UK cell and gene therapy industry by helping cell and gene therapy organisations across the world translate early-stage research into commercially viable and investable therapies.

Albumedix Chief Executive Officer; Jonas Skjdt Mller commented on the collaboration:

With a mission to empower excellence in advanced therapies, we are committed to continuously playing an integral part in enabling our customers to advance in a fast-moving industry. For us to do so, we continuously look at other industry leaders to establish collaborations. Continuing to learn from each other allows innovation in the market to advance, and Albumedix to support our customers with in-depth knowledge of how rAlb can uniquely benefit their therapies. Cell and Gene Therapy Catapult is the ideal partner; located in our own backyard here in the UK and with incredible skills, knowledge and drive to advance the cell and gene therapy industry we are excited about this collaboration.

Matthew Durdy, Chief Executive Officer at Cell and Gene Therapy Catapult commented:

The opportunity to collaborate with a leading company like Albumedix Ltd in order to assess and drive the potential of their latest technology is something that we embrace. The prospect of improving manufacturing of viral vectors such as Adeno-associated virus (AAV) through this exciting technology is something which could significantly benefit and advance the wider cell and gene therapy field.

Activities under this agreement will be carried out both at the CGT Catapult facility in Braintree and at Albumedix new R&D center, with state-of-the-art laboratories specifically designed for the process optimization, characterization and formulation development of advanced therapies.

Get in touch with Albumedix today by emailing communications@albumedix.com to learn more about their Recombumin range of world leading recombinant human albumin products. Reach out to Cell and Gene Therapy Catapult by emailing communications@ct.catapult.org.uk to learn more about how they can help your organisation to translate early stage research into commercially viable and investable therapies.

About Albumedix Dedicated to Better Health

Albumedix is a science-driven, life-science company focused on enabling the creation of superior biopharmaceuticals utilizing our recombinant human albumin products. We believe in empowering excellence to enable advanced therapies and facilitate otherwise unstable drug candidates reach patients worldwide. We are proud to be recognized as the world leader in recombinant human albumin with products and technologies used in clinical and marketed drugs by pharmaceutical and medical device companies worldwide. Headquartered in Nottingham, England with more than 100 people all committed to improving patient quality of life. We are just as passionate about albumin and albumin-enabled therapies today as we were when we started more than 35 years ago. For more information, please reach out to Albumedix at communications@albumedix.com or visit http://www.albumedix.com

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

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Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com

Gene silencer: Monkeys injected with a modified virus that includes the binding sequence of miRNA183 (bottom) express significantly lower levels of a gene delivered for gene therapy than do those injected with an unmodified virus, with or without steroid treatment (top).

A novel method for delivering gene therapies to the brain and spinal cord reduces nerve damage in primates, a new study shows. The approach could improve the safety of gene therapies under development for conditions related to autism, such as Angelman syndrome, Rett syndrome and fragile X syndrome.

Gene therapy typically involves replacing or repairing a faulty gene with a functional version, using a harmless adeno-associated virus (AAV). In primates, however, AAV-based gene therapies can damage the axons neuronal projections that transmit signals to other cells of neurons in the dorsal root ganglion, a bundle of nerves close to the spinal cord that relay information to the brain. In severe cases, degeneration in these cells leads to poor motor coordination.

Cell death in the dorsal root ganglion is associated with high levels of expression of the artificially introduced gene, the new work shows. To prevent it, the researchers devised a way to limit this gene expression only in the dorsal root ganglion nerves and not where it is needed.

The method takes advantage of short regulatory RNAs, known as microRNAs, that dampen gene expression by binding to messenger RNA, the intermediary between genes and proteins. The team identified four microRNAs miRNA96, miRNA145, miRNA182 and miRNA183 that are mainly expressed in the dorsal root ganglion. For each microRNA, they added its binding sequence to an AAV carrying the gene for a green fluorescent protein and injected it into mice. After 21 days, they imaged tissue slices from the spinal cord and various organs, including the brain, to gauge levels of the protein.

Viruses carrying the binding sequence of miRNA183 most effectively prevented expression of the green protein in the dorsal root ganglion, imaged tissue slices revealed. The findings were published in November in Science Translational Medicine.

Next, the team tested the viruses in macaques. In one experiment, they injected two animals with a virus carrying only the gene for the fluorescent protein, and another four with a version carrying miRNA183s binding sequence.

The researchers also made a virus carrying hIDUA, a gene that codes for an enzyme lacking in people with the condition mucopolysaccharidosis. They injected this virus into six macaques, half of which also received steroids to lower immune responses, a common practice in gene therapy trials. Three additional macaques received a modified version of the hIDUA virus that included the miRNA183 binding sequence.

After 14, 60 or 90 days, the team examined tissue slices from the animals spinal cord, brain and other organs. Adding miRNA183s binding sequence to the virus significantly prevented expression of either the fluorescent protein or hIDUA in the dorsal root ganglion, the researchers report. Elsewhere in the body, however, the genes expression levels were unaltered, or even increased.

The modified viruses that included miRNA183s binding sequence also caused less toxicity: Macaques injected with these versions had few, if any, damaging lesions in the dorsal root ganglion. By contrast, the steroid treatment did not reduce toxicity, suggesting that the immune response does not explain the damage.

The microRNA technique could be used to mitigate toxicity for a variety of gene therapies that target the central nervous system, the researchers say. It may also enable scientists to further investigate gene therapies for autism.

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Method may improve safety of gene therapies targeting the brain - Spectrum

Second Product Candidate Expected to Enter Clinic in First Half of 2021

Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options

PHILADELPHIA, Jan. 28, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies.

We are pleased to be advancing our second therapy into clinical development in our quest to bring transformative medicines to patients who need them, said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. FTD can have a devastating impact on a persons quality of life and create a substantial caregiving and economic burden for families. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.

FTD is one of the more common causes of early-onset (midlife) dementia, causing impairment in behavior, language and executive function, and occurs at similar frequency to Alzheimers disease in patients younger than 65 years. In approximately 5 to 10 percent of individuals with FTD 3,000 to 6,000 in the United States the disease occurs because of mutations in the GRN gene, causing a deficiency of progranulin (PGRN). PGRN is a complex and highly conserved protein. The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRNs role in lysosomal function. The rapid progression of FTD results in an average survival of eight years after onset of symptoms.

Passage Bio is developing PBFT02 to treat FTD-GRN as a single dose delivered via intra-cisterna magna (ICM) injection. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient's cells. The goal of this vector and delivery approach is to provide higher than normal levels of PGRN to the central nervous system to overcome the progranulin deficiency in GRN mutation carriers, who have been observed to have reduced cerebrospinal fluid PGRN levels ranging from 30% to 50% of the PGRN levels observed in normal, mutation non-carriers.

Clinical Development of PBFT02 Supported by University of Pennsylvanias Gene Therapy Program (GTP) Pre-Clinical Data

Passage Bio is advancing PBFT02 into the clinic supported by preclinical data generated by its collaborator, University of Pennsylvanias Gene Therapy Program (GTP). The data, published in the peer-reviewed scientific journal Annals of Clinical and Translational Neurology, showed that a single administration of an optimized AAV containing the GRN gene resulted in elevated levels of PGRN in the brain and cerebral spinal fluid (CSF), reduced lysosomal storage lesions, normalized lysosomal enzyme expression and corrected microgliosis in a mouse model of progranulin deficiency. A single administration of PBFT02 via the optimized AAV1-GRN vector demonstrated transduction broadly across the brain, including a very high transduction of ependymal cells that line the ventricles of the brain and are involved with CSF production, resulting in CSF progranulin levels of more than 50-fold normal.

The FDA has granted an Orphan Drug designation for PBFT02 for the treatment of FTD-GRN.

Phase 1/2 Study Initiation Anticipated for 1H21

Passage Bio expects to initiate a Phase1/2 clinical trial for PBFT02 in the first half of 2021. The trial is designed as a dose-escalation study of a single ICM dose of PBFT02 in subjects with FTD and heterozygous mutations in the GRN gene. The primary endpoint of the Phase 1/2 study is safety and tolerability; secondary endpoints include CSF progranulin levels, disease biomarkers, and clinical outcome measure. Initial data from the trial is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Passage Bio Investors:

Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com Zofia.mita@sternir.com

Passage Bio Media:

Gwen FisherPassage Bio215-407-1548gfisher@passagebio.com

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Passage Bio Receives FDA Clearance of IND Application for PBFT02 Gene Therapy Candidate for Treatment of Patients with Frontotemporal Dementia with...