StemCell Therapy

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.

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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).

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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:

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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...

A gene therapy targeting the inherited electrical heart disorder long QT syndrome has been developed by researchers at the Mayo Clinic and has shown early success in lab-based studies.

The therapy involves a two-stage process of silencing the genetic error that causes the electrical dysfunction and then replacing it with a functional protein using a short hairpin RNA vector.

As reported in the journal Circulation, the research is still at an early stage, but shows promise in a lab-based cardiac cell model.

Although the first gene therapy trials happened more than 30 years ago, several deaths and adverse effects from these original therapies, combined with sometimes limited efficacy, led to delays in further research and development in this area.

However, things have changed over the last few years. Since the eye gene therapy Luxturna was approved by the FDA in 2017, more than double the number of new therapies for different genetic diseases have gone into development than before and there have been notable successes in developing therapies for various retinal, neurological and immune disorders.

The possibility of developing gene therapies for heart disease has not been explored in depth until recently and is still in its early stages. More widespread heart diseases with polygenic causes are complicated to target with gene therapies and incur high costs. But many rarer arrythmias, such as long QT syndrome, have a genetic cause and are therefore potential gene therapy targets.

Gene therapy is an emerging area of interest for treating a variety of genetic heart diseases in general and long QT syndrome in particular, says Michael Ackerman, M.D. Ph.D., a Mayo Clinic cardiologist and director of Mayo Clinics Windland Smith Rice Comprehensive Sudden Cardiac Death Program, who led the research.

Long QT syndrome is caused by an electrical heart problem that can cause irregular heartbeat, and under conditions of stress, or exertion, can cause the heart to stop beating suddenly. It occurs in approximately 1 in 7,000 people and most often has a genetic cause, although in some cases it can be acquired as a side effect of certain medications or as a result of other illnesses.

Ackerman and team designed and developed the first suppression and replacement KCNQ1 gene therapy approach for the potential treatment of patients with type 1 long QT syndrome. The KCNQ1 protein is involved in potassium channel function and mutations in this gene are common in people with long QT syndrome.

The therapy designed by the Mayo Clinic team involved cloning two short hairpin RNAs one for silencing purposes and one to replace the faulty protein into a single gene therapy construct.

When tested in beating cardiac cells from long QT patients and controls in the lab, the therapy successfully normalized the electrical function of the long QT cells, providing proof of principle to continue development of the therapy.

If the therapeutic efficacy of this disease-in-the-dish gene therapy trial with KCNQ1 can be replicated in a nonhuman, animal model of long QT syndrome, then suppression-replacement gene therapy may be a promising strategy for long QT syndrome in general and in theory almost any sudden death-predisposing autosomal dominant genetic heart disease, says Ackerman.

Of course, we still have a long way to go from nearly curing a patients heart cells in the dish to effectively treating the whole person. Nevertheless, we are excited by this first critical milestone and look forward to the next step.

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Prototype Gene Therapy for Long QT Heart Condition Developed - Clinical OMICs News

REDWOOD CITY, Calif., Feb. 02, 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 publication of preclinical data on ADVM-022 intravitreal (IVT) gene therapy in Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO). ADVM-022 is in clinical trials for wet AMD and DME, and this preclinical study in NHPs is the longest safety and expression study to date, with measurements out 30 months following a single IVT injection.

There is a growing body of both clinical and preclinical data demonstrating durable efficacy and favorable safety profile following a single IVT injection of ADVM-022, said Laurent Fischer, M.D., chief executive officer at Adverum Biotechnologies. In this preclinical study, we saw long-term, sustained aflibercept expression out to 30 months following ADVM-022. The levels of aflibercept were sustained at therapeutic levels, with no measurable adverse effects on normal retinal structure and function. We are excited to work on developing ADVM-022 as a potential one and done IVT injection therapy that may dramatically reduce the treatment burden for patients living with wet AMD and DME.

Szilrd Kiss, M.D., academic retina specialist, added, Currently, patients with wet AMD are treated with frequent anti-VEGF intravitreal injections to maintain their vision. One of the highest priorities in research today is to develop therapies that extend the duration of efficacy following treatment, enabling patients to preserve sight for months or years following treatment. The preclinical data on ADVM-022 demonstrate long-term safety and aflibercept expression following a single intravitreal injection of this novel IVT injection gene therapy. We are excited to continue to assess ADVM-022 as it demonstrates the potential to improve real-world visual outcomes over intermittent anti-VEGF injections for patients living with wet AMD.

The publication, titled Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates, reported the following:

The full online publication can be accessed from the TVST website.

About ADVM-022 Gene TherapyADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection (IVT), designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for patients with wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).

In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for ADVM-022 for the treatment of wet AMD.

Adverum is currently evaluating ADVM-022 in the OPTIC Phase 1 clinical trial in patients with wet AMD and the INFINITY Phase 2 trial in patients with DME at 2 x 10^11 vg/eye and 6 x 10^11 vg/eye doses. The Company plans to begin a pivotal trial in mid-2021 for ADVM-022 in wet AMD.

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 visit http://www.adverum.com.

Forward-looking StatementsStatements contained in this press release regarding the events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding: the potential for ADVM-022 in treating patients with wet AMD and DME; the potential efficacy and safety of ADVM-022 in wet AMD and DME; Adverums expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for future complications or side effects in connection with use of ADVM-022. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with theSEConNovember 5, 2020under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - GlobeNewswire

Undaunted by the challenges the COVID-19 pandemic unleashed on the world the expected surge of cell and gene therapies already in the pipeline and on the horizon will continue to materialize, and with them the complexity of riding that wave of innovation.

Just two years ago the U.S. Food and Drug Administration (FDA) forecast that it would be flooded by 2020 with about 200 Investigational New Drugs a year on top of the more than 800 active cell-based or gene therapies it was already processing. The agency projected that by 2025 it would be approving anywhere between 10 to 20 new cell and gene therapy products a year. By 2024, the FDA and the pharmaceutical and biopharmaceutical industries expect more than 40 new and innovative cell and gene therapies will be available on the market.

Although the pandemic disrupted drug discovery and development efforts early in the crisis, the industry has been quick to respond and adjust. The CG&T market will likely slow from $6.68 billion in 2019 to $6.92 in 2020 because of the pandemic, yet it is forecast to recover and grow to an estimated $13.23 billion by 2023, according to Researchandmarkets.com.

There is more momentum than ever before to bring these innovative medicines to market, said Doug Cook, president of Commercialization Services and Animal Health at AmerisourceBergen. The influx of therapies offers tremendous promise and hope to patients with conditions where there are few treatment options and no cures. But these complex products introduce new considerations throughout the commercialization journey, so its critical that manufacturers work with a partner that can help them navigate challenges at each stepfrom pre-clinical and commercial logistics to market access strategies and patient support solutions.

Because CG&T are derived from a patients own cells, time and temperature have become critical factors from the moment they are extracted on through the manufacturing process and then returned as a curative life-saving therapy. As such, there is little room for failure or delay throughout the supply chain.

Given the narrow window of viability of these therapies they need to be shipped as quickly as possible to preserve the time the cells are active. With such a constraint on the time those cells are viable, the pressure on logistics providers has become even more acute. Clearly, supply chain companies that have larger networks and better access to more depots are more advantageous for manufacturers, but more importantly, for patients.

The complexity of these treatments can be staggering both from a development perspective and on into storage and transportation, Cook said. For the first time, the patient is now part of the supply chain where they used to be at the end of it, and thats really different than anything weve seen before.

Many, if not most, of CG&T require ultra-frozen storage from the development stage on through to the application to the patient. This is an element of the supply chain the public is becoming acutely aware of as a result of the COVID-19 pandemic. For example: Pfizer-BioNTechs COVID-19 vaccine must be stored in containers that can achieve between -80 to -60 degrees Celsius. C> require storage conditions from ultracold (-80 degrees Celsius) down to cryogenic temperatures (-135 to -150 degrees Celsius). To ensure the product remains viable throughout transport, the shipping containers must have the ability to keep a constant monitor of the temperatures as well as have real-time GPS tracking.

The shorter the shelf life of the cell therapy, the more intense the logistical challenges. To achieve successful outcomes in what are very patient-centric treatmentsoften referred to as a vein-to-vein supply chainrequires manufacturers to partner with experienced and technologically advanced wholesalers and distributors that have a global reach and ability to address issues with customs and country-specific regulatory requirements.

As the wave of these therapies begins to swell past the approval stage, the need for infrastructure that can handle CG&T has to be in place to avoid bottlenecks and delays that could limit patient access.

Because of all the complexity, handoffs are where mistakes happen, and you need a partner who focuses on all those small details and makes the process seamless, Cook said. This is where experience matters, and capabilities are essential.

In order to continue to meet and exceed its capabilities, early last year AmerisourceBergen strengthened its logistics offerings by integrating its global logistics provider, World Courier, with ICS, its third-party (3PL) provider. Now fully integrated, the service offers a complete cryogenic supply chain. World Courier and ICS offer vapor-charged cryogenic storage with fully automated technology and temperature-controlled transport from a manufacturers location to a storage facility and then to each point of care in dry shipment containers. The group has extensive experience in navigating international borders while maintaining temperature requirements.

With a global network of more than 140 offices, World Couriers has the ability to provide cryogenic shipping solutions that are close to patient and manufacturing locations, which provides much more flexibility as well as cutting response times for patient and hospital needs.

Its become clear as we navigated through COVID that everything has to be connected in ways they werent before, Cook said. As a result, weve invested in more technology services to better position ourselves to support CG&T and play the role of partner and connector more than ever before.

And we are always looking at ways to offer more cohesive capabilities.

To learn more about how AmerisourceBergen anticipates supply and demand and how we do business and the role of distributors in the supply chain check out:https://www.amerisourcebergen.com/pharmaceutical-distribution/value-of-the-distributor

Continued here:
Meeting the commercialization challenge of a surging gene and cell therapy market - FierceBiotech

NEW YORK, Feb. 3, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases,today announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to ARU-1801, a one-time investigational gene therapy for sickle cell disease (SCD).

"PRIME designation from EMAhighlightsthe importance of ARU-1801, administeredwith only reduced intensity conditioning,for the treatment ofindividuals with severe sickle cell disease,"said Will Chou, M.D., Aruvant chief executiveofficer."With PRIME,we will be able to work closely with EMAon the development of ARU-1801, with the goal of rapidlybringingthispotential cure toSCD patients in Europe."

PRIME was created by the European Medicines Agency (EMA) to enhance support for the development of innovative medicinesthat target an unmet medical needand demonstratethe potential to achieve relevant clinical outcomes on morbidity, mortality or underlying disease progression. The PRIME designation offersenhanced early interaction with companiesdeveloping promising medicines, to optimize development plans and speed up evaluation. PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments, or that benefit patients without treatment options.

ARU-1801 was designated PRIME status based on clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, that demonstrate meaningful,durable reductions in disease burden.

About ARU-1801ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM StudyAruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at http://www.momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromsickle cell disease(SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. For more information on the clinical study, please visit http://www.momentumtrials.comand for more on the company, please visitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About RoivantRoivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

SOURCE Aruvant Sciences

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Aruvant Announces the European Medicines Agency (EMA) Granted Priority Medicines (PRIME) Designation to ARU-1801 for the Treatment of Sickle Cell...

NEW YORK, Feb. 1, 2021 /PRNewswire/ --Welsh, Carson, Anderson & Stowe ("WCAS"), a leading private equity firm focused exclusively on the healthcare and technology industries, announced today that it is committing up to $250 million to a strategic partnership with Kiniciti, a newly-formed platform. Kiniciti will invest in non-therapeutic companies supporting cell and gene therapy ("CGT") innovation which have the potential to transform the cell and gene therapy ecosystem and deliver the promise of CGT to impact patients' lives.

Principal focus areas for investment include companies with: transformational capabilities in cell engineering and gene-editing; cell sources and other value-added starting materials; process science and scale-up tools and services; production technologies; and, source-to-patient delivery. Kiniciti plans to invest in a cross section of CGT opportunities, large and small, across multiple geographies.

Core to Kiniciti's strategy is its flexible investment model focused on ensuring that the ecosystem of companies supporting cell and gene therapeutics customers have access to the capital and strategic resources necessary to enable these advanced therapies to rapidly and reliably reach patients.This will include control, growth equity and significant minority stake structures intended to:

Kiniciti's leadership team includes Geoffrey Glass, Chief Executive Officer, and Jason Conner, Chief Strategy Officer. For more than 25 years, Mr. Glass has helped lead services and therapeutic companies in the life sciences sector. Mr. Conner has helped numerous high-growth life sciences and services companies in his senior strategy, corporate development, and legal roles over more than two decades. Kiniciti's core team has a total of five decades of experience in growing and scaling companies across the healthcare services, life sciences and tools and equipment sectors organically and through M&A.

Mr. Glass said, "The number of innovations, new companies and clinical trials in the cell and gene therapy space is at an all-time high and, ironically, this is exactly when challenges emerge. The pace of funding and therapeutic innovation is far outstripping the available human capital to design, execute and scale the uniquely demanding processes required by advanced therapies. Furthermore, many promising cell and gene therapy solutions providers lack the scale and capital to support this pace of industry growth. We aim to address these challenges."

"In forming Kiniciti, we are thrilled to partner with WCAS, a pioneer in the private equity industry with a 40-year track record of building strong, sustainable platforms working hand-in-hand with management teams," added Mr. Glass. "The firm has deep experience investing in high growth healthcare businesses that are at unique inflection points. WCAS has raised and successfully managed funds totaling over $27 billion of committed capital, and dozens of public healthcare companies can trace their roots to WCAS. We are pleased that two of WCAS's General Partners, Nick O'Leary and Brian Regan, will serve on our Board of Directors and we look forward to benefitting from their judgment and years of experience."

Nick O'Leary, General Partner at WCAS, said, "Partnering with Kiniciti to help realize the promise of cell and gene therapy represents a natural extension for WCAS's Healthcare franchise. We will pursue opportunities where operational improvements, organic growth initiatives and strategic acquisitions can unlock full potential, for both our investments and the patients these companies serve. In today's cell and gene therapy landscape, we believe that there are many exciting therapy innovators that possess the right science but need the supporting ecosystem essential to advancing their therapeutics at the pace they require and deserve. We look forward to working with the Kiniciti team to help address these critical pain points to help deliver CGT at scale and lower cost."

About KinicitiNewly-formed Kiniciti was established to partner with companies with the potential to transform and strengthen the cell and gene therapy ecosystem. With a highly tailored, collaborative and flexible investment and strategic support model, Kiniciti aims to ensure the promise of cell and gene therapeutics is delivered quickly and safely to patients in need worldwide. The company's leadership team includes professionals experienced in investing in and building successful companies across the life sciences sector. For more information, visit kiniciti.com.

About Welsh, Carson, Anderson & StoweWCAS is a leading U.S. private equity firm focused on two target industries: technology and healthcare. Since its founding in 1979, the firm's strategy has been to partner with outstanding management teams and build value for its investors through a combination of operational improvements, growth initiatives and strategic acquisitions. The firm has raised and managed funds totaling over $27 billion of committed capital. WCAS is currently investing an equity fund, Welsh, Carson, Anderson and Stowe XIII, L.P., which closed on $4.3 billion in commitments in 2019. For more information, please visit wcas.com.

Media and Investment Opportunity Contact:

Geoffrey Glass+1 (212) 650-4104[emailprotected]

SOURCE Welsh, Carson, Anderson & Stowe; Kiniciti

https://www.wcas.com/

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Welsh, Carson, Anderson & Stowe Commits $250 Million in a Strategic Partnership with Kiniciti, a Newly-Formed Platform Investing in Cell and Gene...

A Cambridge UK-Korea joint venture promising great things in nextgen cell and gene therapy technology has been rewarded with major cash backing in a Series A round.

Avactas JV with Daewoong Pharmaceutical AffyXell Therapeutics has secured $7.3 million to further develop its pipeline of next generation cell and gene therapies.

AffyXell was established in January 2020 to develop novel mesenchymal stem cell therapies. The business is combining Avactas Affimer platform with Daewoongs MSC platform such that the stem cells are genetically modified to produce and secrete therapeutic Affimer proteins in situ in the patient.

The Affimer proteins are designed to enhance the therapeutic effects of the MSC creating a novel, next generation cell therapy platform.

The Series A funding has been raised from a group of venture funds including Samsung Venture Investment Corporation, Shinhan Venture Investment, Smilegate Investment, Shinhan Investment Corporation, Kolon Investment, Stonebridge Ventures and Gyeongnam Venture Investment.

The proceeds will be used by AffyXell to further the development of MSCs engineered to produce Affimer molecules generated by Avacta that suppress immune response and restore immune balance.

While initially focusing on inflammatory and autoimmune diseases and prevention of organ transplant rejection, longer term goals could also include applications in regenerative medicine, infectious diseases and oncology.

Avacta's R & D costs associated with the generation of the Affimer proteins are funded by AffyXell whilst Avacta retains the rights to commercialise the Affimer proteins outside the field of cell therapies.

Avacta CEO Dr Alastair Smith said: The potential for AffyXells new class of MSC therapies to deliver improved treatments for a wide range of inflammatory and autoimmune diseases is significant, in a market estimated to be worth $16 billionn by 2025.

We expect these novel engineered MSCs to show a more powerful therapeutic effect than existing antibodies and stem cells and they therefore have the potential to lead the rapidly growing field of cell and gene therapy.

AffyXell is uniquely positioned to develop novel and powerful cell therapies through the combination of two world-class technologies: Avactas Affimer platform and Daewoongs proprietary technology for generating off-the-shelf allogeneic MSC therapies.

Completion of the Series A funding is a strong validation of this concept and moves us closer to providing these new therapies to the patients who need them.

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Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly - Business Weekly

Feb. 1, 2021 12:00 UTC

Expects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively

Plans to initiate a U.S. Phase 1/2 trial for TSHA-101 in GM2 gangliosidosis in second half of 2021 as well as Phase 1/2 trials for TSHA-118 in CLN1, TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end 2021

Anticipates advancement of four programs into IND/CTA-enabling studies: SLC13A5 haploinsufficiency, Adult Polyglycosan Body Deficiency (APBD), Lafora disease and GM2 AB variant

Expects IND/CTA submission from one of the following programs: SLC13A5 haploinsufficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

Anticipates advancement of four new undisclosed programs into preclinical development focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases

Intends to advance the development of next-generation technologies including miRARE platform, redosing strategy, mini-gene payloads and novel capsids, to optimize key components of the companys AAV-based gene therapies

Continues to make progress on internal 187,000 square-foot, 2,000-liter capacity, multi-product cGMP facility located in Durham, NC

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today highlighted its strategic priorities and provided a business outlook for 2021.

We enter 2021 having built a strong foundation on which to execute our corporate and pipeline objectives. Notably, we expanded our seasoned leadership team and esteemed board of directors steeped in gene therapy development and commercialization expertise, successfully raised funds in our initial public offering, transitioned from a preclinical- to a clinical-stage company, and achieved important progress on R&D initiatives and our three-pillar manufacturing strategy, said RA Session II, President, Founder and CEO of Taysha. 2021 will be a transformational year as we intend to rapidly advance multiple drug candidates to clinical proof-of-concept, further expand our platform-enabled pipeline and advance next-generation technologies. Specifically, we expect to report clinical data for our GM2 gangliosidosis program in the second half of this year and have multiple ongoing clinical studies by year end. We also anticipate several IND/CTA submissions across three CNS franchises and have multiple therapies in IND/CTA-enabling studies while advancing four new programs into preclinical development. In addition, we are excited to advance our next-generation platform technologies and further our efforts in redosing, transgene regulation and capsid development. We believe that our platform will drive future sustained innovation and value creation and look forward to highlighting the productivity of our platform in an R&D day later this year. Lastly, we continue to make progress on cGMP facility and process development capabilities with the completion of the design phase and initiation of procurement of long lead equipment.

Anticipated Milestones by Program

TSHA-101 for infantile GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes HEXA and HEXB intrathecally for the treatment of infantile GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for one of the most common genetic causes of severe intellectual disability, designed to deliver MECP2 as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a codon optimized transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

Pipeline programs advancing into IND/CTA-enabling studies

Discovery programs

Next-generation technology platform

Anticipated Corporate Milestones in 2021

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates and early-stage programs, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210201005298/en/

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Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook - BioSpace

Ground zero for disruptive growth in the healthcare sector is genomics through the ARK Genomic Revolution Multi-Sector Fund (CBOE: ARKG).

ARKG holds equity securities of companies across multiple sectors, including health care, information technology, materials, energy, and consumer discretionary, that are relevant to the funds genomics theme. The active management team behind the ARKG strategy combines a top-down and bottom-up research methodology to identify innovative companies and convergence across markets.

The second generation of cell and gene therapy is one of multiple frontiers ARKG provides exposure to. Its also lacking in many old-school biotechnology ETFs.

New cell and gene therapy innovations could increase the total addressable market for oncology therapeutics by more than 20-fold, according to ARK Research.

The actively managed ARKG offers investors a thematic multi-capitalization exposure to innovative elements that cover advancements in gene therapy bio-informatics, bio-inspired computing, molecular medicine, and pharmaceutical innovations.

ARKG includes companies that merge healthcare with technology and capitalize on the revolution in genomic sequencing. These companies try to better understand how biological information is collected, processed, and applied by reducing guesswork and enhancing precision.

Interestingly, ARKG marries one disruptive technology with others.

The US Food and Drug Administration (FDA) approved Gleevec, an oral chemotherapy, after ten years of trials, seven years of which were in solid tumors. This timeline suggests that the FDA could approve the first CAR-T therapy for solid tumors in 2025, notes ARK. Because of artificial intelligence (AI), gene-editing, and next generation sequencing (NGS), failure rates and time-to-market should fall, accelerating approval rates.

The evolution of gene therapies from ex vivo to in vivo is another scenario worth monitoring in the coming years.

Unlike ex vivo, in vivo therapies cannot check edited cells before transduction. That said, in vivo gene therapy is more cost effective and easier to manufacture and scale. It also enables more access to the liver, eye, central nervous system (CNS), and muscles, concludes ARK.

For more on disruptive technologies, visit our Disruptive Technology Channel.

The opinions and forecasts expressed herein are solely those of Tom Lydon, and may not actually come to pass. Information on this site should not be used or construed as an offer to sell, a solicitation of an offer to buy, or a recommendation for any product.

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Unlock the Long-Term Genomics Runway with 'ARKG' - ETF Trends

Press Release

4BIO Capitals review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery

Systematic review and meta-analysis of 149 clinical trials of AAV-based gene therapies demonstrates high safety and efficacy rates

Data reinforces potential of AAV-based gene therapies to become a mainstream and potentially curative treatment modality

25 January 2021

LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.

The review, led by Dr. Dima Kuzmin in collaboration with academics from the University of Oxford and Childrens Hospital of Philadelphia as well as several members of 4BIOs management and advisory teams, analyses 149 unique adeno-associated virus (AAV) gene therapy clinical trials, determining current key trends and depicting the impact of clinical trials on the gene therapy field. This is the first systematic review and meta-analysis that establishes transition probabilities, cumulative safety, and efficacy data for AAV gene therapies.

Gene therapy using AAV as a vector has emerged as a novel therapeutic modality with significant clinical developments made over the past 20 years, including the treatment of over 3000 patients, showing its potential for substantial disease modification in many monogenic disorders, and perhaps even cures. The most commonly used serotype, AAV2, has produced vast amounts of safety and efficacy evidence, indicating its ability to be readily used within the gene therapy space. In addition, decreasing clinical trial duration, which reflects increased comfort from regulators, and the growing number of successful clinical trials focusing on treating diseases of the central nervous system (CNS), reiterates the potential of AAV gene therapy as a safe, well tolerated and efficacious therapeutic modality.

The authors show that AAV gene therapy is generally safe and well tolerated, with no clinical trials failing to reach their primary safety endpoints prior to the cut-off date of the review. Additionally, whilst sample sizes are still relatively small, the existing data indicates the success rates of gene therapy development are significantly higher than the average for all modalities. For example, the transition probability for a drug progressing from an investigative new drug (IND) to a new drug application (NDA) is 31% for ophthalmology gene therapies as compared to 24% across all modalities; 43% vs. 16% in metabolic diseases; 56% vs 47% in haematology; and 30% vs. 19% in neurology.

However, the authors conclude that drawbacks remain and further innovations, such as better manufacturing, an ongoing switch to engineered capsids and synthetic promoters could lead AAV gene therapies to advance from the primary targets of the retina, liver, muscle and brain into other major organs and therefore into the next stage of clinical significance.

Dr. Dima Kuzmin, Managing Partner at 4BIO Capital, said: The analysis of vast amounts of clinical trial data has allowed us to establish the safety, tolerability and efficacy of AAV gene therapy, and consider it as a potential curative modality. Whilst certain challenges, such as uncertainty with regards to durability of response to gene therapy and potential viral vector liver toxicity are still yet to be overcome, it is clear that AAV gene therapy has the potential to become an effective mainstream therapeutic option.

-Ends-

Contacts

About 4BIO Capital

4BIO Capital is an international venture capital firm focused solely on the advanced therapies sector.

4BIOs objective is to invest in, support, and grow early stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome.

The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals including Nature, The Lancet, Cell, and the New England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space.

For more information, please visit http://www.4biocapital.com

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4BIO Capital's review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery - GlobeNewswire

Docket Number: FDA-2020-D-1137 Issued by:

Guidance Issuing Office

Center for Biologics Evaluation and Research

FDA plays a critical role in protecting the United States from threats such as emerging infectious diseases, including the Coronavirus Disease 2019 (COVID-19) pandemic. FDA is committed to providing timely guidance to support response efforts to this pandemic.

FDA is issuing this guidance to provide manufacturers of licensed and investigational cellular therapy and gene therapy (CGT) products with risk-based recommendations to minimize potential transmission of the novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). This guidance is intended to supplement the recommendations to drug and biological product manufacturers provided in FDAs Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry issued in June 2020 (Ref. 1) (June 2020 GMP Guidance). The recommendations in this guidance specifically consider the source material (cells and/or tissues) recovered from donors and how the CGT product will be manufactured (e.g., cell expansion in culture, viral reduction steps, formulation).

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets ManagementFood and Drug Administration5630 Fishers Lane, Rm 1061Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2020-D-1137.

01/19/2021

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Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency - FDA.gov

BEDFORD, Texas, Jan. 25, 2021 /PRNewswire/ --NANOSCOPE THERAPEUTICS Inc., a clinical-stage biotechnology company determined to change lives through the ocular gene therapy, today announced that it has received an Orphan drug designation from FDA for gene therapy-based treatment of Stargardt disease, a form of inherited retinal degenerative disease caused by gene mutation and passed on to children.

Central vision loss due to poor functioning RPE cells and loss of photoreceptors in the macula is the hallmark of Stargardt disease.According to Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S.The loss of vision is devastating to both children and adults, and significantly impacts their quality of life. There is an immense need to restore vision in these patients.

"Currently, there are no approved therapies for Stargardt Disease"said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope."We are excited by the potential of ambient light activatable Multi-Characteristic Opsin (MCO) based photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner."

"vMCO-010 expands the portfolio of orphan drug designations obtained in multiple pipeline programs, expanding its therapeutic potential from Retinitis Pigmentosa (RP) with peripheral retinal degeneration to macular degeneration as in Stargardt disease", said Al Guillem, Chairman of Nanoscope Therapeutics Board.

Sai Chavala, Chief Medical Officer of Nanoscope, commented "We look forward to developing our innovative optogenetic platform technologies for visually-challenged patients through continued interaction with the FDA. The orphan designation will aid in accelerating our clinical development program."

The Orphan Drug Act encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset clinical development costs, as well as eligibility for seven years of post-approval market exclusivity.

"We are extremely excited and pleased to announce a second orphan drug designation for MCO based treatment for retinal diseases. Since the pathology of degenerated macula in Stargardt disease is similar to that of dry-AMD, we are very excited about the opportunity to learn from our Stargardt program and advance MCO-based dry-AMD program"said Samarendra Mohanty, President and Chief Scientific officer of Nanoscope that received an orphan designation in 2017 for MCO for treatment of RP.

AboutNANOSCOPE THERAPEUTICS Inc.

Nanoscope Therapeutics is advancing gene therapy using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. We utilize an ambient light-sensitive MCO molecule to re-sensitize the retina toward ambient light level. Our pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with RP, Stargardt disease, and dry-AMD.

Contact:

Sulagna BhattacharyaInvestor Relations and Corporate Communications817-719-2692[emailprotected]

http://www.nanostherapeutics.com

SOURCE Nanoscope Therapeutics

http://www.nanostherapeutics.com

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Nanoscope Therapeutics Receives Orphan Drug Designation for gene therapy of blindness - PRNewswire

Large pharmaceutical companies have made gene therapy a priority with a series of acquisitions over the past several years, a stamp of validation for a field that's pushed through decades of ups and downs.

One of the latest buyers is German healthcare conglomerate Bayer, which in October inked a $2 billion deal for North Carolina gene therapy developer Asklepios Biopharmaceuticals, also known as AskBio.

For Bayer, the acquisition is part of a broader effort to build a gene and cell therapy division. But the deal is just as noteworthy for AskBio, an unusually large, privately held biotech based on the work of one of gene therapy's pioneers, Jude Samulski.

AskBio chose the security of a wealthy backer over independence and the chance to go public like several of its peers. And the deal helped the biotech quickly hire Katherine High, one of the few executives with experience shepherding a gene therapy through to regulatory approval. All of which makes the efforts of AskBio, now operating as an independent arm of Bayer, worth watching.

"I think we have the right people, the right chemistry, and the right amount of experience to make a difference here," Samulski said in an interview.

As 2019 drew to a close, so did a chapter in High's career. A hematologist by training, High, 69, has spent three decades working in gene therapy, a large portion of which was as a founder, president and chief scientific officer of Philadelphia biotech Spark Therapeutics.

At Spark, High had helped make history by steering the development of Luxturna, a treatment for a genetic form of blindness. When cleared by the Food and Drug Administration in late 2017, Luxturna became the first gene therapy for an inherited disease approved in the U.S. Roche swooped in soon after to acquire Spark, and closed the deal in December 2019.

Katherine High, president of therapeutics at AskBio

Permission granted by AskBio

High decided to take a year off from biopharma. But the coronavirus pandemic dashed her plans to conduct research at Rockefeller University. The institution reduced its staff to essential personnel, and the Harvard Club of New York City, where High, a Philadelphia resident, planned to stay during the week, closed its doors.

"My sabbatical turned into a virtual event, which was good; I got a lot of things done review articles written, book chapters written, things like that," High said in an interview. "I really needed a break."

She spent time with her first grandchild, swam, and, fulfilling a longtime desire, remotely studied German at Middlebury College's storied language program.

But High couldn't keep away from drug research. During periodic visits to North Carolina, where she has family, High dropped in on Samulski and fellow AskBio co-founder Sheila Mikhail.

High has over the years both collaborated and competed with Samulski, a University of North Carolina researcher and expert in gene therapy delivery tools known as adeno-associated viruses, or AAVs. He formed AskBio in 2001 with another gene therapy researcher, Xiao Xiao, and CEO Sheila Mikhail, a life sciences attorney.

"Our paths have crossed, our students have crossed, our sciences [have] definitely cross-pollinated," Samulski said, describing High's academic work at University of Pennsylvania and his at UNC.

By the time of their meeting, AskBio had grown to become one of the gene therapy field's most unique. Originally bootstrapped with angel investing and backing from the Muscular Dystrophy Association, AskBio had spun multiple gene therapy programs into companies that were later acquired. The returns from those buyouts were then used by AskBio to build its own manufacturing capabilities, a crucial step for gene therapy products.

During High's visits, Samulski and Mikail shared some of the progress the company had made advancing its technology. Among them: the acquisition of a Scottish biotech whose technology may allow the company to more tightly control how much protein a gene therapy can produce. Doing so could help overcome a critical limitations of gene therapy, which can have widely varying effects from patient to patient.

"We have a roadmap, how to get from A to B," Samulski told High. "If you want to come in and champion that, we would love to have you."

As AskBio was courting High, Bayer was eyeing AskBio, which had put in motion plans for an initial public offering a typical step for a biotech of its size.

Bayer had already announced plans to develop a cell and gene therapy division, acquiring Bluerock Therapeutics, a maker of "off-the-shelf" cell-based treatments, in 2019.

But the large pharma didn't have an anchor for its gene therapy ambitions. Marianne De Backer, Bayer's head of business strategy and development, had assembled a list of developers to pursue. AskBio was at the top.

"If you look at the [gene therapy] assets that are on the market today, like Zolgensma from Novartis, part of the technology is based on technology from AskBio," she said in an interview, referring to the Swiss company's spinal muscular atrophy treatment. A Duchenne muscular dystrophy treatment in late-stage testing at Pfizer also originated within AskBio, as did a Takeda program being studied in hemophilia.

De Backer faced two obstacles, though. Bayer, for one, didn't know the AskBio team, and couldn't meet them in person because of the travel restrictions that began during the pandemic.

"It was really almost a cold call," she said.

Bayer was also competing against the draw of a deep market for public stock offerings, which helped a record number of biotechs to IPO in 2020. De Backer said she needed to show AskBio that she could get the deal done quickly. So she and Mikhail spent six weeks hammering out terms, including an agreement the company could continue to operate independently an "arm's length" arrangement like one Bayer made with BlueRock.

Such promises are often made, and eventually broken, when a larger company acquires a smaller one. But Samulski's concerns that AskBio's work might be stifled within such a massive company were eased after speaking with BlueRock CEO Emile Nuwaysir.

Jude Samulski, co-founder and chief scientific officer at AskBio

Permission granted by AskBio

"[Nuwaysir] said, they have left me alone, they've encouraged me to do what I'm doing,' and I said, OK, that's what I needed to hear,'" Samulski said.

The acquisition allows the company to spend more time on science and less on raising money, he added.

"If I go back and write a grant today, it'll be three years before we can start the project," Samulski said. "In this setting, when we have our meeting ... the decision-makers are at the table and the science starts that afternoon."

For High, AskBio represents a return to a similar role as the one she had left: helping run an advanced gene therapy business newly acquired by big pharma. At AskBio, she's been named president of therapeutics.

The role, however, lines up with High's current career ambitions. AskBio has the manufacturing capabilities, breadth of clinical-stage programs and financial backing to take on diseases like Parkinson's and congestive heart failure the types of complex, common conditions gene therapy hasn't yet been proven in.

"There are great strengths in pharma, and there are great strengths in biotech, and the ideal situation is one that will let you employ the strengths of both types of organizations," she said.

High considered other options, such as working with a different and unproven drugmaking technology. But as someone who's spent much of her life living the story of gene therapy, she knows more than most the challenges of pioneering a new technology and convincing regulators of its worth.

Sometimes people "may underestimate the amount of time it takes to build all the tools that you need to enable regulators to say 'yes, this is safe,'" she said.

By sticking with gene therapy, much of the groundwork has been laid. She's just looking to take it a step further.

"I'm probably not going to work for another three decades," High said, with a laugh.

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How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division - BioPharma Dive

January 22, 2021 -In 2021, payers will expand upon lessons learned about telehealth and gene therapy in 2020 as they implement their strategies in the new year.

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Now that telehealth utilization has become more widespread, payers will work toward integrating virtual care and telehealth.

Over the course of the pandemic, payers had the opportunity to use telehealth and virtual care in a variety of contexts. As a result, the healthcare system has more data on what uses are best suited to telehealth.

For example, the data reinforced the fact that telehealth and virtual care are excellent modes of care for behavioral and mental health needs. One payer saw over 1 million telehealth claims between February and May 2020 and 50 percent of those telehealth visits were related to mental healthcare.

However, beyond behavioral and mental healthcare utilization, diagnostic work and some primary care services have also emerged as areas that can benefit from telehealth utilization. Virtual cares role in chronic disease management and specialty care is expanding, according to some payer experts.

Payers will also need to grapple with how to approach gene therapy coverage in 2021. Throughout the pandemic, the healthcare industry and the public at large have been clinging to the hope of a gene therapy that would cure the coronavirus.As a result, the pandemic underscored the demand and the impact of these types of treatments.

In 2021, payers will now want to return to creating innovative, new payment models for expensive gene therapy treatments. CVS Health and Aetna reported that care coordination is key to lowering costs around gene therapies.

Payers can also implement value-based care contracts in which pharmaceutical companies can be held accountable for drug therapies that do not meet certain quality metrics.

One of the biggest takeaways from 2020, however, is that the future is unpredictable and that at times historical data cannot prepare healthcare organizations for coming events.

As a result, experts are predicting that real-time data collection will become more essential to forecasting the future in healthcare.

A little less than 75 percent of health executives told PricewaterhouseCoopers that they planned to increase investments in predictive technology in 2021.

Working off of recent data can help organizations both on a macro scale and a micro scale as payers work toidentify widespread potential crises more quickly and to prevent health crises in individual members lives, PricewaterhouseCoopers pointed out.

Additionally, collaboration with other healthcare industry stakeholders can help inform payers efforts to observe trends and respond quickly.

Healthcare Strategies explored these and other trends in its recent special episode on 2021 expectations across the healthcare industry.

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2021 Payer Strategies Build on Telehealth, Gene Therapy Lessons - HealthPayerIntelligence.com