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CREATED BY VANCOUVER MAGAZINE, IN PARTNERSHIP WITH SCENTUALS

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7 Easy Ways to Transition Your Skin Care Routine for Spring - Vancouver Magazine

Recommendation and review posted by Bethany Smith

The following discussion of our financial condition and results of operationsshould be read in conjunction with our audited consolidated financial statementsand related notes and other financial information included elsewhere in thisAnnual Report on Form 10-K. The discussion contains forward-looking statements,such as our plans, expectations and intentions (including those related toclinical trials and business and expense trends), that are based upon currentexpectations and that involve risks and uncertainties. Our actual results maydiffer significantly from management's expectations. The factors that couldaffect these forward-looking statements are in Item 1A of Part I of this report.This discussion should not be construed to imply that the results discussedherein will necessarily continue into the future, or that any expectationsexpressed herein will necessarily be indicative of actual operating results inthe future. Such discussion represents only the best present assessment by ourmanagement.

Business Overview

We have generated aggregate product revenues from our two commercial businessesof $7.2 million and $7.1 million for the years ended December 31, 2021 and 2020,respectively. We currently have no revenue generated from our principaloperations in therapeutic and clinical product development.

Our products are based on multi-decade experience with human cell culture and aproprietary type of pluripotent stem cells, human parthenogenetic stem cells("hpSCs"). Our hpSCs are comparable to human embryonic stem cells ("hESCs") inthat they have the potential to be differentiated into many different cells inthe human body. However, the derivation of hpSCs does not require the use offertilized eggs or the destruction of viable human embryos and also offers thepotential for the creation of immune-matched cells and tissues that are lesslikely to be rejected following transplantation. Our collection of hpSCs, knownas UniStemCell, currently consists of 15 stem cell lines. We have facilitiesand manufacturing protocols that comply with the requirements of GoodManufacturing Practice (GMP) standards as promulgated by the U.S. Code ofFederal Regulations and enforced by the United States Food and DrugAdministration ("FDA").

COVID-19 Pandemic

The impact of the COVID-19 pandemic has been and will likely continue to beextensive in many aspects of society, which has resulted in and will likelycontinue to result in significant disruptions to the global economy, as well asbusinesses and capital markets around the world. Impacts to our business haveincluded a reduction in sales volume primarily from media sales in ourbiomedical market segment and professional channel sales in our anti-agingmarket segment, temporary or reduced occupancy of portions of our manufacturingfacilities, and disruptions or restrictions on our employee's ability to travelto such manufacturing facilities which caused minor delays in manufacturing. Ourmanufacturing facilities continue to operate as they are deemed essentialsuppliers in accordance with laws applicable to California and Maryland. We havetaken precautionary measures to better ensure the health and safety of ourworkers, including staggering employees' shifts and isolating at-risk employees.

The scope and duration of these delays and disruptions, and the ultimate impactsof COVID-19 on our operations, are currently unknown. We are continuing toactively monitor the situation and may take further precautionary and preemptiveactions as may be required by federal, state or local authorities or that wedetermine are in the best interests of public health and safety. We cannotpredict the effects that such actions, or the impact of COVID-19 on globalbusiness operations and economic conditions, may continue to have on ourbusiness, strategy, collaborations, or financial and operating results.

Market Opportunity and Growth Strategy

Therapeutic Market - Clinical Applications of hpSCs for Disease Treatments

With respect to therapeutic research and product candidates, we focus onapplications where cell and tissue therapy is already proven but where there isan insufficient supply of safe and functional cells or tissue. We believe thatthe most promising potential clinical applications of our technology are: 1)Parkinson's disease ("PD"); and 2) traumatic brain injury ("TBI"). Using ourproprietary technologies and know-how, we are creating neural stem cells fromhpSCs as a potential treatment of PD, TBI and stroke.

Our most advanced project is the neural stem cell program for the treatment ofParkinson's disease. In 2013 we published in Nature Scientific Reports the basisfor our patent on a new method of manufacturing neural stem cells which is usedto produce the clinical-grade cells necessary for future clinical studies andcommercialization. In 2014 we completed the majority of the preclinical researchestablishing the safety profile of NSC in various animal species includingnon-human primates. In June 2016 we published the results of a 12-monthpre-clinical non-human primate study, which demonstrated the safety, efficacyand mechanism of action of the ISC- hpNSC. In 2017 we dosed four patients inour Phase I trial of ISC-hpNSC, human parthenogenetic stem cell-derived neuralstem

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cells for the treatment of Parkinson's disease. We reported 12-month resultsfrom the first cohort and 6-month interim results of the second cohort at theSociety for Neuroscience annual meeting (Neuroscience 2018) in November 2018. InApril 2019, we announced the completion of subject enrollment, with the 12thsubject receiving a transplantation of the highest dose of cells. There havebeen no safety signals or serious adverse effects seen to date as related to thetransplanted ISC-hpNSC cells.

In August 2014 we announced the launch of a stroke program, evaluating the useof ISC-hpNSC transplantation for the treatment of ischemic stroke using arodent model of the disease. The Company has a considerable amount of safetydata on ISC-hpNSC from the Parkinson's disease program and, as there isevidence that transplantation of ISC-hpNSC may improve patient outcomes as anadjunctive therapeutic strategy in stroke, having a second program that can usethis safety dataset is therefore a logical extension. In 2015 the Companytogether with Tulane University demonstrated that NSC can significantly reduceneurological dysfunction after a stroke in animal models.

In October 2016 we announced the results of the pre-clinical rodent study,evaluating the use of ISC-hpNSC transplantation for the treatment of TBI. Thestudy was conducted at the University of South Florida Morsani College ofMedicine. We demonstrated that animals receiving injections of ISC-hpNSCdisplayed the highest levels of improvements in cognitive performance and motorcoordination compared to vehicle control treated animals. In February 2019, wepublished the results of the pre-clinical study in Theranostics, a prestigiouspeer-reviewed medical journal. The publication titled, "Human parthenogeneticneural stem cell grafts promote multiple regenerative processes in a traumaticbrain injury model," demonstrated that the clinical-grade neural stem cells usedin our Parkinson's disease clinical trial, ISC-hpNSC, significantly improvedTBI-associated motor, neurological, and cognitive deficits without any safetyissues.

Anti-Aging Cosmetic Market - Skin Care Products

Our wholly-owned subsidiary LSC develops, manufactures and offers for saleanti-aging skin care products based on two core technologies: encapsulatedextract derived from hpSC and specially selected targeted small molecules. LSC'sproducts include:

LSC's products are regulated as cosmetics. LSC's products are sold domesticallythrough a branded website, Amazon, ecommerce partners and through theprofessional channel (including dermatologists, plastic surgeons, medical, dayand resort spas).

Biomedical Market - Primary Human Cell Research Products

Our wholly-owned subsidiary LCT develops, manufactures and commercializesapproximately 200 human cell culture products, including frozen human "primary"cells and the reagents (called "media") needed to grow, maintain anddifferentiate the cells. LCT's scientists have used a standardized, methodical,scientific approach to basal medium optimization to systematically produceoptimized products designed to culture specific human cell types and to elicitspecific cellular behaviors. These techniques can also be used to produceproducts that do not contain non-human animal proteins, a feature desirable tothe research and therapeutic markets. Each LCT cell product is quality testedfor the expression of specific markers (to assure the cells are the correcttype), proliferation rate, viability, morphology and absence of pathogens. Eachcell system also contains associated donor information and all informed consentrequirements are strictly followed. LCT's research products are marketed andsold by its internal sales force, OEM partners and LCT brand distributors inEurope and Asia.

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Results of Operations

Comparison of the Years Ended December 31, 2021 and 2020

Product sales revenue for the year ended December 31, 2021 was $7.2 million,compared to $7.1 million for the year ended December 31, 2020. The increase wasprimarily attributable to a $342 thousand increase in sales in our biomedicalmarket segment, largely offset by a $294 thousand decrease in sales in ouranti-aging market during 2021 compared to 2020.

Our biomedical product sales continue to recover from the impacts of COVID-19 aspurchasing activity from our largest original equipment manufacturer customersincreases.

Our professional skin care products, which are largely marketed to medicalprofessionals and spas that offer walk-up retail, experienced a significantdecline in customer demand due to COVID-19 and the related restrictions as thesebusinesses have continued with limited or reduced operations during the yearended December 31, 2021. The impact of these restrictions was mitigated in-partby expanding our offering of professional skin care products through ourecommerce channel. Anti-aging product sales through our ecommerce channelremained consistent year-over-year.

Cost of Sales

Cost of sales for the year ended December 31, 2021 was $2.9 million, compared to$2.8 million for the year ended December 31, 2020. The increase was primarilyattributable to an increase in costs as a result of an increase in productsales. Profit margins have deteriorated for the year ended December 31, 2021 ascompared to 2020, largely as a result of rising raw materials and labor relatedcosts, and a scarcity of certain materials, principally plastics. In response,we have increased our supply of raw materials on hand and have, where possible,sourced materials from alternative vendors.

Cost of sales consists primarily of salaries and benefits associated withemployee efforts expended directly on the production of the Company's products,as well as related direct materials, general laboratory supplies and anallocation of overhead. We aim to continue refining our manufacturing processesand supply chain management to improve the cost of sales as a percentage ofrevenue for both LCT and LSC.

General and Administrative Expenses

General and administrative expenses for the year ended December 31, 2021 was$4.1 million, compared to $4.4 million for the year ended December 31, 2020. Thedecrease was primarily attributable to a decrease in personnel-related costs andstock-based compensation of $522 thousand, a $87 thousand decrease in consultingand servicing fees, and a $26 thousand decrease in investor relations fees,partially offset by an increase in impairment of intangible assets of$184 thousand, a $57 thousand increase in director and officer liabilityinsurance premiums, a $24 thousand increase in human resource related expenses,a $16 thousand gain on foreign currency exchange rate conversion, and a$15 thousand increase in filing fees.

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Selling and Marketing Expenses

Selling and marketing expenses for the year ended December 31, 2021 was$1.4 million, compared to $1.8 million for the year ended December 31, 2020. Thedecrease was primarily attributable to a $178 thousand decrease inpersonnel-related costs, sales commissions and stock-based compensation,primarily as a result of headcount reductions and grants from 2018 which wereissued and fully vested in 2021, and a $211 thousand decrease in marketing,advertising, and building related expenses, partially offset by a $17 thousandincrease in consulting and creative service fees. The reduction in marketing,advertising, and building related expenses was largely attributable to travelrestrictions as a result of COVID-19.

Research and Development Expenses

Research and development expenses for the year ended December 31, 2021 was$0.7 million, compared to $1.0 million for the year ended December 31, 2020. Thedecrease was primarily attributable to a $247 thousand decrease inpersonnel-related costs and stock-based compensation primarily as a result ofheadcount reductions and grants from 2018 which were issued and fully vested in2021, a $89 thousand decrease in materials and supplies related to clinicaltrial expenses, a $46 thousand decrease in consulting services, partially offsetby a $45 thousand increase in building and utilities related expenses and a$44 thousand decrease in our research and development tax credit related toqualifiable expenditures from our research and development activities of ourAustralia subsidiary, Cyto Therapeutics, which reduced research and developmentexpenses for years ended December 31, 2021 and 2020.

Our research and development efforts are primarily focused on the development oftreatments for Parkinson's disease, traumatic brain injury, liver diseases,stroke, and the creation of new GMP grade human parthenogenetic stem cell lines.These projects are long-term investments that involve developing both new stemcell lines and new differentiation techniques that can provide higher puritypopulations of functional cells. Research and development expenses are expensedas incurred and are accounted for on a project-by-project basis. However, muchof our research has potential applicability to each of our projects. As wecompleted Phase 1 of our clinical in June 2021, we do not anticipate significantinvestment in research and development efforts related to therapeutic andclinical product development efforts for the foreseeable future, or until suchtime that we initiate a Phase 2 clinical trial.

Other Income, Net

Other income, net, for the year ended December 31, 2021 was $1.0 million,compared to other income, net, of $94 thousand for the year endedDecember 31, 2020. The increase was primarily attributable to forgiveness of ourFirst Draw Loan and Second Draw Loan from the PPP, collectively totaling$1.1 million, partially offset by a decrease of $207 thousand for the change inthe fair value of the warrant liability during the prior year period. Thewarrants expired unexercised in March 2021 and, as such, no further change inthe fair value of the warrant liability will be recognized.

Liquidity and Capital Resources

As of December 31, 2021, we had an accumulated deficit of approximately$110 million and have, on an annual basis, incurred net losses and negativeoperating cash flows since inception. Substantially all of our operating losseshave resulted from the funding of our research and development programs andgeneral and administrative expenses associated with our operations. We incurrednet losses of $0.9 million and $2.7 million for years ended December 31, 2021and 2020, respectively. As of December 31, 2021, we had cash of $171 thousand,compared to $689 thousand as of December 31, 2020.

In May 2020, we received a first draw loan of $654 thousand from the PPP ("FirstDraw Loan") which provided additional liquidity to support our currentoperations. In March 2021, we received a second draw loan of $474 thousand fromthe PPP ("Second Draw Loan"). In June 2021, we applied for and receivedforgiveness of unpaid principal and accrued interest from our First Draw Loan inthe amount of $661 thousand. In August 2021, we applied for and receivedforgiveness of unpaid principal and accrued interest from our Second Draw Loanin the amount of $476 thousand. As of December 31, 2021, we are not eligible toreceive any additional funding, or have any further obligations, related to thePPP.

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Cash Flows

Comparison of the Years Ended December 31, 2021 and 2020

The following table provides information regarding our cash flows for the yearsended December 31, 2021 and 2020 (in thousands):

Net cash used in operating activities $ (1,297 ) $ (341 )Net cash used in investing activities

Operating Cash Flows

For the year ended December 31, 2021, net cash used in operating activities was$1.3 million, resulting primarily from our net loss of $899 thousand and netchanges in operating assets and liabilities of $823 thousand, consistingprimarily of an increase in accounts receivable of $441 thousand, inventory,net, of $268 thousand, and decrease in operating lease liabilities of $342thousand, partially offset by net non-cash adjustments of $425 thousand. For theyear ended December 31, 2020, net cash used in operating activities was$341 thousand, resulting primarily from our net loss of $2.7 million and changein fair value of warrant liability of $207 thousand, offset by non-cashadjustments of stock-based compensation expense of $1.3 million, operating leaseexpense of $265 thousand and depreciation and amortization of $253 thousand,coupled with net changes in operating assets and liabilities of $623 thousand.

Investing Cash Flows

Net cash used in investing activities for the year ended December 31, 2021 was$45 thousand, compared to $108 thousand for the year ended December 31, 2020.The decrease was attributable to a decrease in payments for patent licenses of$58 thousand and purchases of property and equipment of $5 thousandyear-over-year.

Financing Cash Flows

Net cash provided by financing activities for year ended December 31, 2021 was$0.8 million, compared to $0.7 million for the year ended December 31, 2020. Forthe year ended December 31, 2021, net cash provided by financing activitiesconsisted of $474 thousand in proceeds from our second draw loan under thePaycheck Protection Program, coupled with proceeds from a note payable from arelated party of $350 thousand. For the year ended December 31, 2020, net cashprovided by financing activities consisted of $654 thousand in proceeds from ourfirst draw loan under the Paycheck Protection Program.

Liquidity and Going Concern

Management continues to evaluate various financing sources and options to raiseworking capital to help fund our current research and development programs andoperations. We will need to obtain significant additional capital from sourcesincluding exercise of outstanding warrants, equity and/or debt financings,license arrangements, grants and/or collaborative research arrangements tosustain our operations and develop products. Unless we obtain additionalfinancing, we do not have sufficient cash on hand to sustain our operations atleast through one year after the issuance date. The timing and degree of anyfuture capital requirements will depend on many factors, including:

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Our failure to raise capital or enter into applicable arrangements when neededwould have a negative impact on our financial condition. Additional debtfinancing may be expensive and require us to pledge all or a substantial portionof its assets. Further, if additional funds are obtained through arrangementswith collaborative partners, these arrangements may require us to relinquishrights to some of its technologies, product candidates or products that we wouldotherwise seek to develop and commercialize on its own. If sufficient capital isnot available, we may be required to delay, reduce the scope of or eliminate oneor more of its product initiatives.

We currently have no revenue generated from our principal operations intherapeutic and clinical product development through research and developmentefforts. In addition, as we completed Phase 1 of our clinical in June 2021, wedo not anticipate significant investment in research and development effortsrelated to therapeutic and clinical product development efforts for theforeseeable future, or until such time that we initiate a Phase 2 clinicaltrial. There can be no assurance that we will be successful in maintaining ournormal operating cash flow and obtaining additional funds and that the timing ofour capital raising or future financing will result in cash flow sufficient tosustain our operations at least through one year after the issuance date.

Based on the factors above, there is substantial doubt about our ability tocontinue as a going concern. The consolidated financial statements were preparedassuming that we will continue to operate as a going concern. The consolidatedfinancial statements do not include any adjustments to reflect the possiblefuture effects on the recoverability and classification of assets or the amountsand classification of liabilities that may result from the outcome of thisuncertainty. Management's plans in regard to these matters are focused onmanaging our cash flow, the proper timing of our capital expenditures, andraising additional capital or financing in the future.

Critical Accounting Policies and Estimates

Our discussion and analysis of our financial condition and results of operationsis based upon our consolidated financial statements, which have been prepared inaccordance with accounting principles generally accepted in the United States.The preparation of these financial statements requires us to make estimates andassumptions that affect the reported amounts of assets, liabilities, revenues,expenses and related disclosures. On an on-going basis, we evaluate ourestimates and assumptions and we base our estimates on historical experience andon various other assumptions that are believed to be reasonable under thecircumstances, the results of which form the basis for making judgments aboutthe carrying values of assets and liabilities that are not readily apparent fromother sources. Actual results may differ from these estimates under differentassumptions and conditions.

Our significant accounting policies are more fully described in Note 1 to ourconsolidated financial statements included elsewhere in this Annual Report onForm 10-K. Our most critical accounting estimates include current andnon-current inventory, intangible assets, and stock-based compensation. Wereview our estimates and assumptions periodically and reflect the effects ofrevisions in the period in which they are deemed to be necessary. We believethat the following accounting policies are critical to the judgments andestimates used in preparation of our consolidated financial statements.

Intangible Assets

Our intangible assets consist of acquired patent licenses and capitalized legalfees related to the acquisition, filing, maintenance, and defense of patents andtrademarks. Amortization begins once the patent is issued by the appropriateauthoritative bodies. In the period in which a patent application is rejected orefforts to pursue the patent are abandoned, all the related accumulated costsare expensed. Our patents and other intangible assets are amortized on astraight-line basis over the shorter of the useful life of the underlyingpatent, which is generally 15 years, or when the intangible asset is rejected orabandoned. All amortization expense and impairment charges related to intangibleassets are included in general and administrative expense in our consolidatedstatements of operations.

Allowance for Excess and Obsolete Inventory

Our inventory, particularly within our biomedical market, consists of certainproducts that have a long or, when frozen, indefinite shelf life. In addition,future demand for our products is uncertain. Accordingly, at each reportingperiod, we estimate a reserve for allowance for excess and obsolete inventory.This estimate is computed using historical sales data and inventory turnoverrates, which are subjective in nature and fluctuate between periods. Theestablishment of a reserve for excess and obsolete inventory establishes a

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new cost basis in the inventory with a corresponding adjustment to cost ofsales. If we are unable to sell such inventory, any related reserves are reducedin the period of sale.

Stock-Based Compensation

We are required to measure and recognize compensation expense for allstock-based payment awards made to employees and consultants based on estimatedfair value. We estimate the fair value of stock options granted using theBlack-Scholes option-pricing model.

The determination of fair value of stock-based awards using the Black-Scholesoption-pricing model requires the use of certain estimates and subjectiveassumptions that affect the amount of stock-based compensation expenserecognized in our consolidated statements of operations. These include estimatesof the expected volatility of our stock price, expected option life, expecteddividends and the risk-free interest rate. Estimated volatility is a measure ofthe amount by which our stock price is expected to fluctuate each year duringthe expected life of the award. The expected option life is calculated using themid-point method as prescribed by accounting guidance for stock-basedcompensation. We determined expected dividend yield to be 0% given that we havenever declared or paid any cash dividends on our common stock, and we currentlydo not anticipate paying such cash dividends. The risk-free interest rate isbased upon United States Treasury securities with remaining terms similar to theexpected term of the share-based awards. If any of the assumptions used in theBlack-Scholes model change significantly, stock-based compensation expense maydiffer materially from what we have recorded in the current period.

Recently Issued Accounting Pronouncements

A description of recently issued accounting pronouncements that may potentiallyimpact our financial position and results of operations is disclosed in Note 1to our consolidated financial statements included in this Annual Report on Form10-K.

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INTERNATIONAL STEM CELL CORP MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS (form 10-K) - Marketscreener.com

Recommendation and review posted by Bethany Smith

Cell therapy research is the process of developing and testing whole cells that can be delivered to a patient to treat or cure a disease. Bone marrow transplants are one of the earliest cell therapies developed where bone marrow cell from a healthy person are used to repopulate the bone marrow of patients with some cancers. Newer cell therapies are cells that may be harvested from altered to target a disease or may be cells that are produced in a laboratory. The cell therapy is infused or transplanted into the patient as a treatment for a disease.

Genes are responsible for creating the proteins that control cellular processes. In many diseases, genetic instructions to code for the protein are missing or defective. Gene therapy research is the process by which scientists develop and test a DNA or RNA molecule that could be delivered back to the patient to treat the disease. Scientists first identify the impacted gene and the nature of the defect in patient cells. Depending on the nature of the defect, a gene therapy can be developed that is a proper copy of the gene or suppresses the defective copy of the gene from being used by the cell. The gene therapy is then put inside of a vector or a nanoparticle that can target the therapy to the proper cells when it is administered to the patient. Once delivered to the patient, the gene therapy provides genetic instructions for that can be used by the cell to correct or minimize the impact of the defect.

Researchers work with contract research organizations, like Charles River, by using gene and cell therapy services to prove efficacy and safety through various study types prior to submitting to agency for approval for first-in-human clinical trials. During and after approval of human trials, researchers continue to work with contract research organizations for clinical trial support, extended nonclinical safety studies and manufacturing support. Additional steps and studies are taken following clinical trials to ensure safety in the manufacturing stages before market release.

Link:
Cell and Gene Therapy Services | Charles River

Recommendation and review posted by Bethany Smith

Professor Rachel Lennon, Professor of Nephrology at the University of Manchester said: Rare diseases have an enormous impact on an individuals life, and they do not attract the critical mass of researchers required to enable rapid improvements in treatment. This Kidney Research UK-Stoneygate award to establish a UK hub for Alport Research is fabulous news and it will transform our ability to increase awareness, improve understanding of the condition and to accelerate new treatment options for patients. By bringing together expertise in cell and matrix biology, genetics and clinical practice, we aim to improve genetic testing and to progress a range of therapy options to extend kidney survival in patients with Alport syndrome.

Sandra Currie, chief executive of Kidney Research UK said: Rare diseases offer a unique challenge to charities such as ours and the overall scientific community. Often, there are limited resources attributed to sourcing new treatments as well as an overall lack of understanding into the causes, impacts and ways to treat them. Embarking on this new collaborative way of working, we are tackling this issue head on by bringing together a wealth of facilities and expertise. With this new Hub, we have the potential to accelerate the discovery and testing of new treatments and possibly even a cure through gene therapy.

The diagnosis and treatment of Alport syndrome have improved in the last decade. However, more progress is urgently needed. With the additional resources and funding, researchers are optimistic that results and transformative treatments could begin to emerge from the hub within the next five years.

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Collaborative research approach set to accelerate treatment of rare kidney condition - The University of Manchester

Recommendation and review posted by Bethany Smith

This article was originally posted on genetherapylive.

Cell and gene therapies are currently being evaluated as possible avenues of treatment for multiple inherited retinal diseases in the field of ophthalmology. Here we highlight the most promising therapies currently in development.

GT005 is a gene therapy by Gyroscope Therapeutics currently being evaluated in the recruiting phase 1/2 FocuStrial (NCT03846193) for the treatment of dry AMD. The company announced positive interim data in February 2021.

Regenerative Patch Technologies is developing the cell therapy CPCB-RPE1 for the treatment of geographic atrophy (GA) in dry AMD. It is being evaluated in 16 participants in a phase 1/2 trial (NCT02590692), positive data from which were presented in June 2021.

The cell therapy ASP7317 (Astellas Pharma) is being developed for GA in dry AMD and is being evaluated in 18 participants in a phase 1 trial (NCT03178149).

AAVCAGsCD59/HMR1002, a gene therapy developed by Hemera Biosciences for the treatment of wet AMD, is being evaluated in 25 participants enrolled in an ongoing phase 1 clinical trial (NCT03585556).

Regenxbios gene therapy RGX-314 is being evaluated in the recruiting phase 2/3 ATMOSPHERE trial (NCT04704921) for the treatment of wet AMD. Regenxbio presented positive initial data at the Retina Society 54th Annual Scientific Meeting in October 2021.

Adverum Biotechnologies is developing the gene therapy ADVM-022 for wet AMD, which is being evaluated in the phase 1 OPTIC trial (NCT03748784). The company plans to initiate a phase 2 trial after observing positive results in the OPTIC trial presented at the Retina Society meeting. The therapy was also being evaluated in diabetic macular edema, but that development was halted after serious adverse events, including irreversible vision loss, were seen in participants in the phase 2 INFINITY trial (NCT04418427).

Editas Medicines gene editing therapy, EDIT-101, is being evaluated in LCA type 10 in the phase 1/2 BRILLIANCE trial (NCT03872479). Data presented at the XIXth International Symposium on Retinal Degeneration demonstrated efficacy in some patients as well as safety concerns such as retinal tears and hemorrhage.

The gene therapy rAAV2-CBSB-hRPE65 is being developed by University of Pennsylvania and National Eye Institute. The therapy is being evaluated in 15 participants ina phase 1 trial (NCT00481546).

Sepofarsen (QR-110) is an RNA antisense oligonucleotide in development by ProQR Therapeutics. The therapy is being evaluated in the phase 2/3 ILLUMINATE trial (NCT03913143), results from which were published in Nature Medicine in April 2021. The last patient completed their last, 12-month visit in January 2022.

MeiraGTX's gene therapy cevaretigene ritoparvovec/AAV RPE65 met the primary endpoints in the phase 1/2 OPTIRPE65 trial (NCT02781480) in 2019. Additional clinical progress is expected in 2022.

Gensight Biologics gene therapy GS010 has so far demonstrated positive data in 90 participants in the phase 3 REFLECT trial (NCT03293524), including statistically significant improvements in visual acuity.

4D-110 is 4D Molecular Therapeutics gene therapy currently being evaluated in a recruiting phase 1 study (NCT04483440). The therapy has been well-tolerated in the low-dose cohort but serious adverse events (AEs) were observed in the high-dose cohort.

The University of Alberta is studying the gene therapy RAAV2.REP1 in 6 participants in a phase 1/2 (NCT02077361). Positive 5-year results were presented at The Association for Research in Vision and Ophthalmology (ARVO) 2021 meeting.

The University of Oxford is studying a similar RAAV2.REP1 gene therapy in 30 participants in a phase 2 trial (NCT02407678).

STZ eyetrials gene therapy RAAV.hCNGA3 is being evaluated in the recruiting phase 1/2 Colourbridge trial (NCT02610582).

Applied Genetic Technologies is developing AGTC-401 (NCT02599922) and AGTC-402 (NCT02935517) for CNGB3 and CNGA3 achromatopsia, respectively. Both phase 1/2 trials have demonstrated tolerable safety profiles for the gene therapies.

MeiraGTX is also developing gene therapies for both CNGB3 and CNGA3 achromatopsia. AAV CNGB3 (NCT03001310) and AAV CNGA3 (NCT03758404) are both in phase 1/2 studies.

QR-1123 is another antisense oligonucleotide from ProQR Therapeutics being evaluated in the recruiting phase 1/2 AURORA trial (NCT04123626) for RHO-RP.

MeiraGTXs gene therapy AAV2-RPGR has demonstrated positive data in X-linked RP (XLRP) in a phase 1/2 study (NCT03252847). The therapy will be further evaluated in the phase 3 Lumeos trial (NCT04671433).

Positive efficacy data from Applied Genetics phase 1/2 trial (NCT03316560) of the gene therapy AGTC-501/rAAV2tYF-GRK1-RPGR was presentedat the American Academy of Ophthalmology (AAO) 2021 Annual Meeting. The therapy is being further evaluated in the phase 2/3 VISTA trial(NCT04850118) for XLRP.

4Ds gene therapy 4D-125 was well-tolerated for XLRP in a phase 1/2 trial(NCT04517149), according to data presented at the 2021 American Society of Retina Specialists (ASRS) 39th Annual Meeting.

The National Eye Institute is evaluating RS1 AAV in a phase 1/2 trial (NCT02317887) for XLRP.

Coave Therapeutics gene therapy CTx-PDE6b (AAV2/5-hPDE6B)is being evaluated for PDE6b-RP in a phase 1/2 trial (NCT03328130).

GenSights GS030-DP gene therapy in combination with the GS030-MD optogenetic device has shown positive efficacy and safety data in treating end-stage blindness in RP. These data, from a phase 1/2 trial (NCT03326336), were presented at the 2021 AAO meeting.

Novartis is evaluating their gene therapy CPK805 in RLBP1-RP in a first in-human phase 1/2 trial (NCT03374657) that is currently recruiting.

ProQRs ulteversen/QR-421a gene therapy showed efficacy in the phase 1/2 Stellar trial (NCT03780257) for Usher syndrome and RP in March 2021. ProQR plans to soon advance the program into finalstage clinical testing.

The gene therapy RST-001 is being evaluated in a phase 1/2 trial (NCT02556736). Updates have been sparse for the therapy, which was developed by RetroSense Therapeutics, which was acquired by Allergan in 2016, which was then acquired byAbbvie in 2020.

The first 4 patients treated in the phase 1/2 trial (NCT04278131) of BS01 are able to detect light and motion,Bionic Sight announced in March 2021. The trial continues to enroll participants.

Nanoscope Therapeutics vMCO-I/MCO-010is being evaluated in a phase 1/2 trial (NCT04919473). Positive data in participants with autosomal recessive RP were presented at the ASRS 2021 meeting.

STZ eyetrials gene therapy rAAV.hPDE6A is being evaluated for PDE6A-RP in the recruiting phase 1/2 Pigment trial (NCT04611503).

More here:
A Wrap-Up of Ophthalmologic Cell And Gene Therapies Currently in Development - MD Magazine

Recommendation and review posted by Bethany Smith

REDWOOD CITY, Calif. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, today announced an exclusive agreement with the Medical University of South Carolina (MUSC) Foundation for Research Development to license next generation complement-targeted gene therapies for the treatment of geographic atrophy and other ocular diseases.

Geographic atrophy, also known as atrophic age-related macular degeneration (AMD), is an advanced form of AMD, a progressive retinal disease affecting millions of adults worldwide. Patients with geographic atrophy experience irreversible loss of vision with significant impact on quality of life due to the chronic and progressive nature of the disease. There are currently no FDA-approved treatments available for geographic atrophy.

"This partnership highlights the broad potential of Kriyas technology and R&D platforms to drive innovation in diseases with established biology, and furthers our mission of developing transformative gene therapies for diseases with high unmet need," said Theresa Heah, M.D., M.B.A., President and Chief Medical Officer of Kriyas ophthalmology division. "We believe that complement hyperactivity is a clinically validated target implicated in the pathogenesis of retinal degeneration in geographic atrophy, and has the potential to address other ocular diseases. We are excited to develop gene therapies that precisely target this pathway, in collaboration with academic partners who have been at the forefront of characterizing the biology underlying these diseases.

The complement system plays a crucial role in the bodys innate immune system by enhancing its ability to clear pathogens and damaged cells, and regulating inflammatory immune responses through complement control proteins. Dysregulation and hyperactivity of the complement system is associated with the onset and progression of serious inflammatory diseases, including geographic atrophy and other ocular conditions. Through this agreement, Kriya is advancing gene therapies that are designed to durably express engineered molecules that selectively reduce complement hyperactivity at the site of pathology following one-time administration.

Geographic atrophy due to age-related macular degeneration is a devastating disease with a profound impact on patients, as there are no approved treatments for the irreversible loss of central vision that often occur, said Peter K. Kaiser, M.D., Professor of Ophthalmology, Cole Eye Institute, Cleveland Clinic. Recent advances in complement inhibitor therapies have provided important additional evidence that targeting complement holds great promise in treating geographic atrophy. I am excited by the potential of restoring balance to the complement system with a one-time gene therapy that can deliver a meaningful long-term solution and a major advancement in the field of retinal disease.

By targeting the inhibition of complement proteins directly involved in complement activation, our approach has the potential to deliver a selective and profound biological effect, said Dr. Brbel Rohrer, Ph.D., Professor of Ophthalmology at the Medical University of South Carolina, and co-inventor of the technology. After having confirmed the potential of this gene therapy strategy in mouse models of age-related macular degeneration, we are very excited to take the next step towards a clinical application for patients with geographic atrophy by partnering with Kriya, a leader in the development of novel gene therapies.

About Kriya

Kriya is a fully integrated company pioneering novel technologies and therapeutics in gene therapy. The company aims to revolutionize how gene therapies are designed, developed, and manufactured, improving speed to market, and delivering significant reductions in cost. Kriya is organized into four principal business units: Kriya Technologies, Kriya Therapeutics, Kriya R&D, and Kriya Manufacturing. The company is advancing a deep and diversified pipeline of innovative gene therapies in multiple therapeutic area divisions, with current pipeline programs in ophthalmology, oncology, rare disease, and chronic disease. Kriya was founded by pioneers in the biopharmaceutical industry and is backed by leading life sciences and technology investors. The company has core operations in Silicon Valley, California and Research Triangle Park, North Carolina. For more information, please visit http://www.kriyatx.com and follow on LinkedIn.

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Kriya Licenses Next Generation Complement-Targeted Gene Therapies for the Treatment of Geographic Atrophy and Other Ocular Diseases - Business Wire

Recommendation and review posted by Bethany Smith

Gene therapies have yielded promising results for individuals experiencing rare diseases. However, these groundbreaking therapies come with their own unique set of challenges regarding who will be able to access them, how much they will cost, and how the policymaking and scientific processes will conflict as more and more therapies undergo clinical trials.

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Last week, we convened a panel of experts to address these questions and discuss potential solutions in our latest 5 Slides Were Watching conversation, led by State of Reforms DJ Wilson. The panel featured Danny Seiden, president & CEO of the Arizona Chamber of Commerce and Industry, Dr. Jennifer Hodge, U.S. DMD Gene Therapy Lead at Pfizer, Dr. Rafael Fonseca, chief innovation officer at Mayo Clinic, and Dr. Sharon Hesterlee, chief research officer at the Muscular Dystrophy Association.

Hesterlee brought a slide showing the prevalence of rare diseases in Arizona, noting that 5,500 Arizonans were estimated to be living with rare genetic neuromuscular diseases that were potentially treatable with gene therapy. She highlighted that Charcot-Marie-Tooth disease and Myotonic dystrophy were the most prominent, and that both diseases currently have gene therapy treatments in preclinical development.

She emphasized that ethics need to be an important part of the conversation, and that it will be critical to educate patients and families about the treatments irreversible implications as more and more therapies begin to launch.

Its a permanent change to someone. What we see in particular with parents of a child who has a pediatric disease, they are put in a very difficult position because they have to make a decision without always understanding all of the science and all of the implications.

So I think there is a huge requirement for the physician [who does the informed consent] to be very clear, and then the parents have to decide if it doesnt work, my child cannot be redosed, my child may not be eligible for another trial I think thats been a big challenge and something that weve tried to help our community in the neuro-muscular disease space navigate.

Seiden brought a slide displaying the economic benefits that would come with the increased prevalence of gene therapies. He noted that outdated systems of payment would not be applicable to this kind of treatment, and that these therapies would allow for one-time costs as opposed to a lifetime of treatment for patients with rare diseases.

When you deal with rare diseases, you need to look at it on an annualized basis over the cost of a lifetime, because gene therapy has the potential to save money and a lot of heartache for the patients and the families involved with it Arizona is one of a handful of states that allows for value-based purchasing when it comes to Medicaid contracts With the [Arizona Health Care Cost Containment System (AHCCCS)], which is by far the largest provider, theyve recognized that you have to look at patient outcomes. Its not just about that initial upfront cost.

Hodge presented a slide illustrating the unmet needs of individuals with rare diseases and the potential impacts that gene therapies can have on these individuals. She emphasized the urgent need for innovative treatments for these diseases, as 95% of rare diseases worldwide have limited or no approved treatment options, and 80% of those rare diseases have a genetic cause. She said this makes patients with rare disease collectively one of the most underserved communities in medicine today.

She said educating every organization involved in the process of developing these therapies on the stories of real patients affected by these diseases will be critical as gene therapies move through both scientific and legislative processes.

Its really to address the underlying cause of rare diseases at the root, meaning the genetics, not the symptoms It cant be a line item in a bill, it cant be something on a piece of paper that you hear about, it has to be someone telling their story [and] thinking about the patient and what theyre going through.

You can learn so much by just sitting and talking and just hearing their story, and little things that you didnt even know affected them We need to bring that to more of the audience thats involved in making some of these decisions so they can see it as more than just a line on a piece of paper when theyre deciding something.

Fonseca showed a slide explaining some specific uses of gene therapy that could potentially provide individualized, life-saving treatment to people with red blood cell diseases, as well as preventive genetic interventions for diseases like cancer.

When you think about this approach in looking at the rare disorders, it turns out that by extrapolation, a lot of the diseases that we consider common also become more and more individualized, and therefore, theyre more and more unique. More and more, we see approaches that have to be very, very much [a] tailored design for patients

To have someone who is born with [a red-blood cell disorder] return to normal red blood cell function is just enormous. This is a worldwide problem, its a problem thats associated with pain, serious medical problems, a shorter lifespan, and great expenditures for the health system, and so [Im very excited about where were at with this].

Wilson highlighted that while few gene therapies have been officially launched in the market, many are currently in pre-clinical and clinical trials and are expected to provide promising health solutions for the future.

Continued here:
5 Slides We're Discussing: Gene therapy and the promise for rare disease - State of Reform - State of Reform

Recommendation and review posted by Bethany Smith

Cell lines

Human PC cell lines LNCaP and PC-3 obtained from JCRB Cell Bank (Osaka, Japan), and 22Rv1 purchased from The European Collection of Authenticated Cell Cultures were maintained in RPMI-1640 (FUJIFILM Wako Pure Chemical, Osaka, Japan) containing penicillin, streptomycin and 10% fetal bovine serum (Equitech-Bio, Kerrville, TX). The docetaxel-resistant 22Rv1 cell line, 22Rv1DR, was previously described18. All cell culture experiments were performed using cells within less than 20 passages except for PC-3PRF cells stably transfected with Tet-on tetracycline-inducible perforin expression vector and docetaxel-resistant 22Rv1DR cells.

Docetaxel was purchased from Selleckchem (Houston, TX, USA).

The perforin expression vector for Tet-On system (pT-Rex-DEST30-perforin) was purchased from Thermo Fisher Scientific (Waltham, MA, USA). The pcDNA6/TR regulatory vector (Thermo Fisher Scientific) and pT-Rex-DEST30-perforin vector were transfected to PC-3 cells using Lipofectamine 2000 (Thermo Fisher Scientific). Transfected cells were selected under 500g/ml G418 and 10g/ml Blasticidin (Thermo Fisher Scientific). Perforin was induced by 1g/ml of tetracycline. The human PSA promoter-driven perforin expression vector (pDRIVEperforin-psa-hpsa) was purchased from InvivoGen (San Diego, CA, USA).

Whole cell lysates were harvested and lysed in RIPA buffer containing the protease inhibitor cocktail (Sigma-Aldrich St. Louis, MO, USA). Western blot analysis was performed as described previously19. The anti-PSA and anti--actin antibodies were purchased from Cell Signaling Technology (Danvers, MA, USA). The immunoreactive proteins were detected using horseradish peroxidase-conjugated anti-rabbit antibody (Cell Signaling Technology) and ImmunoStar (FUJIFILM Wako Pure Chemical).

Perforin expression in conditioned medium, mouse serum and harvested xenograft tumors was measured by human perforin ELISA kit according to manufacturers instruction (Abcam, Cambridge, UK).

SS-cleavable and pH-activated lipid-like material (ssPalmM), 1,2-Dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE), cholesterol and 1,2-Dimyristoyl-rac-glycero-3-methoxypolyethylene glycol-2000 (DMG-PEG2000) were purchased from NOF Corporation (Tokyo, Japan). Encapsulation of plasmid vector in lipid nano-particles was conducted according to a previous report20. First, plasmid DNA and protamine solutions (0.3mg/mL and 0.144mg/mL) were prepared in 10mM HEPES buffer (pH5.3). Plasmid DNA/protamine core particle was prepared by the drop-wise addition of 1mL of the protamine solution into the 1mL of the DNA solution with vortexing. Liposome was composed of ssPalmM, DOPE, cholesterol and DMG-PEG2000 in a molar ratio of 3:4:3:0.5. Lipids (3.3mol of total lipids) were dissolved in 2mL of ethanol and the lipid solution (2mL) was rapidly diluted with an equal volume of the plasmid DNA/protamine core particle suspension with vortexing. The solution was further diluted with 36mL of 10mM HEPES (pH 5.3) to obtain 5% ethanol(v/v) concentration. The diluted solutions were concentrated to ten times by Amicon 8400 ultrafiltration stirred cell with a Biomax membrane (Merck Millipore, Allen, TX, USA) following further serial ultrafiltration with 100mM HEPES (pH 7.4) and 10mM HEPES (pH 7.4) using Amicon 8050 ultrafiltration stirred cell with a Biomax membrane. Finally, the liposome solution was filtrated by 0.45m of pore size Millex HV (Merck Millipore).

Liposome concentration was measured as total cholesterol concentration in the presence of sodium dodecyl sulfate using a Cholesterol E test Wako (FUJIFILM Wako Pure Chemical) and the total amount of fatty acids was calculated based of the molar ratio of each lipid. DNA concentration in liposomes was determined using Quant-iT Picogreen dsDNA Assay Kit (Thermo Fisher Scientific) in the presence of Triton X-100. Particle size and -potential were measured at 25C using Zetasizer Nano-S90 (Malvern Panalytical, Worcestershire, UK) after 50 times dilution of samples with distilled water.

This study was approved by the Medical Review Board of Gifu University, Graduate School of Medicine (No. 2018219). A written informed consent was obtained from participants and blood was collected from male volunteers without clinically detectable cancer. All methods were performed in accordance with the relevant guidelines and regulations in compliance with the Declaration of Helsinki. Human PBMCs were isolated by Ficoll-Paque density gradient centrifugation according to the manufacturers instructions (Amersham Biosciences, Piscataway, NJ).

Cells were seeded on 96-well plates. Twenty-four h after seeding, agents with or without PBMCs were added. Cell viability was determined using WST-1 assay kit (Roche Diagnostics, Mannheim, Germany). The mean value obtained from PBMCs alone was deducted from the values obtained from co-culture of prostate cancer cells and PBMCs.

All animal experiments were approved by the Gifu University Animal Experiment Approval Committee (No. 2019116) and carried out in accordance with the approved guidelines. This study is compliant with the ARRIVE guidelines. Six-week-old male athymic nude mice (BALB/cSlc-nu/nu) were purchased from Japan SLC, Inc. (Shizuoka, Japan). A suspension of 22Rv1DR cells (1107) cells in PBS was mixed with Matrigel (1:1) in a final volume of 0.2mL. The mixture was subcutaneously injected to generate tumors. Two weeks after the injection, tumor volume was measured and mice were randomly assigned to 2 groups (n=5). Agents were intravenously administrated via tale vein. The tumor volume and body weight were monitored and measured once a week. Four weeks after treatment, mice were sacrificed and the resected tumors were weighed.

Statistical analysis was performed using Graph Pad Prism 7 version 7.03 (Graph Pad Software, CA, USA). Comparison of 2 groups was made using t-test or MannWhitney U test. Comparison among 4 groups was made using one-way ANOVA with Tukeys post hoc for multiple comparisons. Differences were considered significant if p<0.05.

More here:
Gene therapy of prostate cancer using liposomes containing perforin expression vector driven by the promoter of prostate-specific antigen gene |...

Recommendation and review posted by Bethany Smith

SAN DIEGO--(BUSINESS WIRE)--Excellos, Incorporated, a cell therapy Contract Development and Manufacturing Organization (CDMO), announces its official corporate launch and closing of $15M in growth funding from Telegraph Hill Partners (THP).

Built on the foundation of the San Diego Blood Bank (SDBB), Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. The companys collection network consists of nine SDBB centers in the San Diego area that see an average of over 70,000 diverse donors annually, as well as exclusive access to select, consented material from SDBBs public cord blood bank. Excellos also has access to a nationwide collection network giving it one of the largest cellular material procurement portfolios. Uniquely connecting its broad collection network to state-of-the-art cGMP and R&D facilities in San Diego, Excellos provides a full suite of innovative end-to-end capabilities to facilitate the development and manufacture of autologous and allogeneic cell therapies. Excellos experience includes working with therapeutic companies developing chimeric antibody receptor-engineered T cells (CAR-T), tumor-infiltrating lymphocytes (TILs), and providing isolated immune cells that are integral to the advancement of immunotherapies.

Excellos will create highly characterized, standardized cell products and custom services that are essential for advanced therapeutics, said David Wellis Ph.D., CEO of Excellos. The SDBB had been incubating Excellos for a number of years, but fully capitalizing on the rapidly growing opportunity required significantly more resources than the SDBB was able to provide. We will now be able to focus exclusively on the needs of the expanding cell and gene therapy industry through the development of our data-driven platform to enable the characterization of cellular therapeutics starting at the donor level. The funding from THP will allow a significant facility expansion and overall growth in the capabilities of our organization.

The growth in the cell and gene therapy industry is driving a surge in demand for critical human cells, tissues, and services to support the development and commercialization of new products, said Paul Grossman, Ph.D., J.D., Partner at THP. We look forward to partnering with Excellos and their proven leadership team. Their exclusive access to one of the industrys largest donor bases, coupled with their technology-focused research and development activities, will help to accelerate the advancement of cell and gene therapies for those patients in need. Paul Grossman, Alex Herzick, and Deval Lashkari from THP have joined the Excellos Board concurrent with the financing.

Excellos founding leadership team brings a wealth of commercial and industry experience to bear. Chief Executive Officer David Wellis Ph.D., previously served as CEO of SDBB for nine years where he foresaw the need for products and services to serve the then nascent cell and gene therapy industry. His guidance allowed the organization to meet these needs, a path that ultimately led to the formation of Excellos. David has also held senior leadership roles at both Illumina and GenVault Corporation. Chief Commercial Officer George Eastwood, brings significant experience in the cell and gene therapy space, with a focus on cGMP materials and cell manufacturing. He served as VP, Global Sales and Business Development for HemaCare, and in his over five years there, he saw the company through a period of dramatic expansion that culminated in its acquisition by Charles River in 2020. Chief Scientific Officer Rob Tressler Ph.D., brings vast experience in R&D for advanced cell-based therapies. Most recently, as CSO of SDBB, he led the cell therapy, immunohematology, components manufacturing, and cord blood banking labs. Rob also has extensive drug development experience, both as Head of Preclinical Oncology at Geron Inc. and VP of R&D at Cellerant Therapeutics.

About Excellos: Built on the foundation of the San Diego Blood Bank, Excellos is focused on supplying cGMP cellular products and services, together with process development and manufacturing expertise to scientists and clinicians working with cell and gene therapies. Excellos is dedicated to improving human life by providing critical products and services to life science research and the next generation of cell and gene therapies. Learn more at http://www.excellos.com

About Telegraph Hill Partners: Telegraph Hill Partners, founded in 2001 and based in San Francisco, CA, invests in commercial stage life science, medical technology, and healthcare companies. For more information, please see http://www.telegraphhillpartners.com

Excerpt from:
Excellos Launches to Accelerate Innovation in the Cell and Gene Therapy Industry - Business Wire

Recommendation and review posted by Bethany Smith

MaxHggblom Distinguished Professor and ChairDepartment of Biochemistry and MicrobiologySchool of Environmental and Biological SciencesRutgers-New BrunswickHonored for distinguished contributions to understanding both the fundamental and application components of microbialbiotransformationsof pollutants, especially chlorinated aromaticcompoundsand metalloids.

MaxHggblomis a renowned research scientist and educator with a large body of microbial ecology and environmental biotechnology research that has expanded our understanding of how the biodegradation of environmental pollutants, such as dioxins and PCBs,impact our planet.

His research interests revolve around thebioexploration, cultivation and characterization of novel microbes.His research on bacteria has provided a foundation for applications that address the pollution problems facing impacted industrialized and urbanized environments.

Hggblomslab is also actively studying microorganisms that degrade pharmaceutical and personal care products in aquatic environments.

Over the past decadesthediverse chemicalsin pharmaceutical and personal care productshave emerged as a major group of environmental contaminants in numerous watersheds around the world; therefore, it is important to understand how microbes can degrade them.There is much to explore and learn,Hggblomadded.

Hggblomswork also touches climate change, particularly the roles and responses of microbes in rapidly changing environments, such as the Arctic.In his lab at Rutgers, students have the unique opportunity to exploreareas of research such asthe biodegradation and detoxification of anthropogenic pollutant chemicals, including certainpesticides;respiration of rare metalloids; or life in the frozen tundra soils.

For several years,my lab has worked on studying the microbial ecology of Arctic tundra soils to understand how the changing conditions impact microbial activity and turnover of soil organic matter, and consequently enhanced greenhouse gas flux,Hggblomsaid. This is an important area of research as the threat of microbial contribution to positive feedback of greenhouse gas flux is substantial.

His lab recently received funding from the National Science Foundation to studyhowdiverse microbial communitiesare established insoils.Hggblomwill work with an international research team of scientists from the U.S., China, South Africa and Finland to study soils from the three differentregionsacross Arctic, Tibetan Plateau and Antarctic habitats to expand our understanding of how soil ecosystems respond in critical polar regions.

Emily EversonLayden

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Twelve Rutgers Professors Named Fellows of the American Association for the Advancement of Science - Rutgers Today

Recommendation and review posted by Bethany Smith

The switch design works solely for certain subsets of cancers, specifically non-small-cell lung cancer cells with an EGFR gene mutation, where drugs that target mutated proteins in the cancer cell are already on the market.

We are taking a careful approach to design and testing, Pritchard said. We will look specifically for failures within the switch, and analyze what we find, sort of like when civil engineers analyze a building or bridge failure after the fact. Failures help us understand where our ideas about cancer therapy are incomplete, and what we can do to fix them and increase our knowledge.

After initial tests on cancer cell lines, the researchers will test the dual-switch gene drives on human organoids, provided by the University of Massachusetts Medical School, which are patient-derived cancer cells that more closely mimic real tumors.

Co-investigator Shelly Peyton, Armstrong Professional Development Professor at University of Massachusetts Amherst and expert in tissue engineering, will lead the design of microenvironments to determine how the gene therapy functions under different conditions. Peytons team will study how certain switches or parameters fail, or why they function well in some environments but not others.

The research here is trying to take the challenge of cancer treatment and flip it on its head, said Scott Leighow, fifth-year doctoral student in biomedical engineering, who gathered the preliminary data that were key to securing the grant. If we can do that, we'll have a therapy that can handle resistant forms of cancer a lot better than what's currently in our arsenal.

If the study is successful, the researchers will test their treatments on animal models to show proof-of-concept, Leighow said. Far in the future, the technology has the potential to offer a cellular gene therapy that might assist cancer patients who are not candidates for surgery.

The grant is part of a new consortia created by the NCI to promote collaborative approaches to synthetic biology for cancer applications.

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$2.5M grant awarded to flip the switch on lung cancer drug resistance - Penn State News

Recommendation and review posted by Bethany Smith

WASHINGTON, Jan. 25, 2022 (GLOBE NEWSWIRE) -- The GlobalHemophilia Market size is expected to reachUSD 14.2 Billionby 2028, exhibiting a CAGR of5.3%during the forecast period. Hemophilia is a bleeding condition, which leads to the prolonged bleeding after injury or a surgery due to a delay in the blood clotting, The Global Hemophilia Market is anticipated to grow at a substantial rate in coming years because of increasing cases of genetic abnormalities and prevalence of Hemophilia, states Vantage Market Research, in a report, titledHemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028).The market size valued atUSD 12.1 Billionin 2021.

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(Please note that the sample of this report has been updated to include the COVID-19 impact study prior to delivery.)

Market Overview:

Increasing Establishment of Hemophilia Treatment Centers to Drive the Market

The availability of limited treatment therapy options and growing burden on regulatory bodies towards the treatment is resulted to increase R&D efforts. The public as well as private healthcare bodies are heavily investing in development of specialized clinics that are established to meet the targeted needs of patients. In this regard, there is increasing number of Hemophilia treatment centres that also aims at proving treatment to underprivileged patients.It is recommended by authorities that people who are suffering from Hemophilia should visit a treatment center for optimal care and health education to stay healthy. The establishment of healthcare centres is anticipated to fuel theHemophilia Market growth.Additionally, efforts are been undertaken by regulatory bodies for spreading awareness regarding the disease and providing information about the effective treatment.

The COVID-19 outbreak has affected various industries worldwide. TheHemophilia Marketis no exception. Governments across the world took severe actions like border seals, lockdown, and implementing strict social distancing measures, in order to stop swift spread of COVID-19. These actions led to severe impact on the global economy impairing various industries. The impact of COVID-19 on the market demand is considered while estimating the current and forecast market size and growth trends of the market for all the regions and countries based on the following data-points:

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Benefits of Purchasing Hemophilia Market Reports:

The Report on Hemophilia Market Highlights:

Various Supportive Initiatives to Drive Market Growth in Asia Pacific

Asia Pacificis anticipated to witness fastest CAGR over the forecast period. The awareness campaigns and supportive initiatives taken by the government to commence the early screening of neonates, is about to boost the demand for diagnostic tools related to Hemophilia in the region. Some other factors such as advanced healthcare ecosystem and capacity of people to spend on such expensive medical services are defining the regional business growth. Additionally, easy medical reimbursement schemes are promoting the market growth.

List of Prominent Players in the Hemophilia Market:

Read Full Research Report @ https://www.vantagemarketresearch.com/industry-report/hemophilia-market-1216

Recent Developments:

December, 2021:The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for Hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal which could allow patients in Europe to access the therapy sooner,

December, 2021:Global biotherapeutics leader CSL Behring announced that the Committee for Medicinal Products for Human Use (CHMP), the chief scientific body of the European Medicines Agency (EMA) accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec (also known as EtranaDez), currently being studied in the Phase 3 HOPE-B clinical trial, is an investigational gene therapy for people living with hemophilia B, a life-threatening bleeding disorder.

December, 2021:Patients with severe Hemophilia can develop inhibitors against factor VIII or IX, preventing factor replacement therapy from working, said Dr. Guy Young (University of Southern California, CA, USA). A quarter of patients develop these inhibitors, leading to a worse prognosis. Novel agents are needed to protect these patients from bleeding events and arthropathy and improve their quality of life. In addition, the current IV therapies need to be administered multiple times per week, resulting in venous access issues and poor adherence.

December, 2020:Pfizer Inc. and Sangamo Therapeutics, Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe Hemophilia A.

Browse the Report Hemophilia Market by Type (Hemophilia A, Hemophilia B, Hemophilia C, Others), by Treatment (On-demand, Prophylaxis), by Therapy (Replacement therapy, ITI therapy, Gene therapy), by Region (North America, Latin America, Europe, Asia Pacific) - Global Industry Assessment (2016 - 2021) & Forecast (2022 - 2028) @ https://www.vantagemarketresearch.com/blog/hemophilia-225379

This market titled Hemophilia Market will cover exclusive information in terms of Regional Analysis, Forecast, and Quantitative Data Units, Key Market Trends, and various others as mentioned below:

Treatment: - On-demand, Prophylaxis

Therapy: - Replacement therapy, ITI therapy, Gene therapy

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Global Hemophilia Market to Reach $14.2 Billion by 2028 - - GlobeNewswire

Recommendation and review posted by Bethany Smith

Patient 1 with Sandhoff disease realized normalization of Hex A enzyme activity by Month 1, achieving 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3

Patient 2 with Tay-Sachs disease achieved Hex A enzyme activity 5-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 1

First-ever data supporting bicistronic vector approach in humans, TSHA-101 is designed to deliver both HEXA and HEXB genes in the endogenous ratio

Conference call and live webcast today at8:00 AM Eastern Time

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today reported positive initial serum -hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, which represent two forms of GM2 gangliosidosis. Todays data are the first ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.

TSHA-101 is the first bicistronic vector in clinical development, representing an important first for the field of gene therapy, noted RA Session II, President, Founder and CEO of Taysha. TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis. We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.

Based on natural history data, patients with asymptomatic GM2 gangliosidosis have Hex A enzyme levels that are at least 5% of normal activity. Key patient findings for Hex A enzyme activity following treatment with TSHA-101 include:

Patient 1 (Sandhoff disease)

Patient 2 (Tay-Sachs disease)

Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development at Taysha added, GM2 gangliosidosis is a progressive and life-limiting disease with no treatment options. Normalization of patient Hex A enzyme activity levels 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history supports TSHA-101s ability to potentially make a meaningful difference in the lives of patients with Sandhoff and Tay-Sachs diseases after a single intrathecal administration. We look forward to submitting a protocol amendment to expand patient enrollment in the ongoing Phase 1/2 trial and providing additional updates later this year.

Preliminary data suggest that TSHA-101 was well-tolerated with no significant drug-related events.

Patient 1 (Sandhoff) demonstrated signs of clinical improvement at Month 3 and was deemed stable to travel home. Upon returning home, Patient 1 (Sandhoff), who was unvaccinated, was exposed to a family member symptomatic for an upper respiratory infection, possibly Covid-19, and was hospitalized with pneumonia before contracting a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection. On January 14, 2022, the patient succumbed to pneumonia and pleural effusion with a concomitant hospital-acquired MRSA infection. The principal investigator has made the initial assessment that the death was unrelated to study drug. Final determination from the independent data safety monitoring board (DSMB) is anticipated in the near term.

TSHA-101 is an investigational gene therapy that delivers both the HEXA and HEXB genes that comprise the -hexosaminidase A enzyme. The two genes are driven by a single promoter within an AAV9 capsid ensuring that the two sub-units of Hex A are produced in the endogenous ratio within each cell, which is important to ensure efficient production of the enzyme. TSHA-101 is the first and only bicistronic vector currently in clinical development for GM2 gangliosidosis and has been granted Orphan Drug and Rare Pediatric Disease designations by the FDA and Orphan Drug Designation from the European Commission. TSHA-101 is administered intrathecally and is currently being evaluated in a single arm, open-label Phase 1/2 clinical trial for the treatment of infants with GM2 gangliosidosis sponsored by Queens University. Additional clinical safety and efficacy data are expected by the end of 2022.

GM2 gangliosidosis is a rare and devastating monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A (Hex A) enzyme. These genetic defects result in progressive dysfunction of the central nervous system. Residual Hex A enzyme activity determines the severity of the disease. The infantile form of the disease has an onset of symptoms usually before six months of age with residual Hex A enzyme activity of less than 0.1%. Juvenile onset occurs between 1.5 and five years of age with residual Hex A enzyme activity of approximately 0.5%. Early adult onset of the disease has residual Hex A enzyme activity of between 2% to 4%. There are no approved therapies for this disease, and current treatment is limited to supportive care.

Conference Call and Webcast Information

Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to provide an update on the GM2 gangliosidosis program. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13726741. The live webcast and replay may be accessed by visiting Tayshas website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.

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, plans, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, such as TSHA-101 and including our preclinical 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, 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, the potential market opportunity for these product candidates, and our corporate growth plans. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, both of which are 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/20220127005353/en/

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Recommendation and review posted by Bethany Smith

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced that its Investigational New Drug (IND) application for its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN), was cleared by the U.S. Food and Drug Administration (FDA).

SBT101 is the first AAV-based gene therapy in development specifically designed for people living with AMN, an adult-onset degenerative spinal cord disease caused by mutations in the ABCD1 gene. SwanBio plans to initiate a randomized, placebo-controlled Phase 1/2 clinical trial designed to assess the safety and explore the efficacy of SBT101 in patients with AMN in the second half of 2022.

Todays IND clearance is a formative milestone for SwanBio, enabling us to evolve from a preclinical company to a truly integrated research and development organization, underscoring the expertise of our team and potential of our technology platform, said Tom Anderson, chief executive officer and director of SwanBio Therapeutics. SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients.

The clinical program for SBT101 builds on SwanBios unique understanding of AMN, including new insights being gathered in an ongoing natural history study. SwanBio is deeply committed to the AMN community and has worked closely with patients, family members, and expert physicians including SwanBio Co-Founder Dr. Florian Eichler to ensure that its clinical programs are designed to meet their needs. SwanBio is supported by long-term investment partners Syncona Ltd. and Mass General Brigham Ventures, which both have proven track records in gene therapy, particularly in AAV-focused therapies.

About SBT101SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was shown to be well-tolerated in non-human primates at six months post-treatment.

About AdrenomyeloneuropathyAdrenomyeloneuropathy (AMN) is the adult-onset degenerative spinal cord disease that affects people living with adrenoleukodystrophy (ALD), a category of rare, genetic, and metabolic conditions. AMN is characterized by progressive loss of mobility, incontinence, and debilitating pain. It affects adults with mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Without a functioning version of this protein there is an accumulation of VLCFA to toxic levels that leads to progressive dysfunction of the central nervous system. Between 8,000-10,000 men in the U.S. and E.U. are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom control.

About SwanBio TherapeuticsSwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

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SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy -...

Recommendation and review posted by Bethany Smith

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that Leber Hereditary Optical Neuropathy (LHON) subjects treated with LUMEVOQ continued to experience significantly improved vision four years after a single injection of the gene therapy. The findings come from RESTORE (CLIN06), the long-term follow-up study to which participants in the RESCUE1 and REVERSE2 Phase III pivotal trials were invited.

When RESTORE subjects enrolled in the study, 2 years after the one-time injection, they had already experienced clinically meaningful improvements relative to the lowest point (the nadir) of their best-corrected visual acuity (BCVA): +18.8 ETDRS letters equivalent* in their LUMEVOQ-treated eyes and +17.3 letters equivalent in their sham-treated eyes. Four years after treatment, the bilateral improvement from nadir was sustained, with LUMEVOQ-treated eyes achieving a mean improvement against nadir of +22.5 letters equivalent and sham-treated eyes demonstrating a mean improvement of +20.5 letters equivalent.

The impact of such results on patients is demonstrated by increases in the self-reported quality of life (QoL) scores at Year 4 vs. baseline. Mean overall QoL increased by a clinically meaningful magnitude relative to baseline, driven by clinically meaningful increases in the sub-scores corresponding to mental health and the ability to carry out activities autonomously (e.g., role difficulties, dependency, near and far activities, general vision).

The 4-year RESTORE long-term extension study provides patients with Leber Hereditary Optic Neuropathy and their families as well as the neuro-ophthalmology community with highly informative data about both the efficacy and safety of intravitreal LUMEVOQ therapy, commented Dr. Robert Sergott, Director, Neuro-Ophthalmology Service, Wills Eye Hospital, and Founding Director and CEO, William H. Annesley EyeBrain Center, Thomas Jefferson University, Philadelphia, PA, USA. Compared to the natural history of LHON, the 4-year data extend and validate the 3-year observations by confirming that objective visual acuity improvement is sustained and is associated with improved functional visual quality of life without any long-term safety concern.

The RESTORE findings underline the therapeutic value of GenSights pioneering one-time treatment for LHON: durable and clinically significant improvement in visual function coupled with impressive safety, noted Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. The body of evidence we have now accumulated is without doubt good news for patients needing an urgent solution for their brutal blinding condition, and consequently we are continuing to work vigorously with the relevant authorities to bring regulatory review process to a successful conclusion.

RESTORE is one of the largest long-term follow-up studies for a rare disease treatment, with 62 subjects accepting the invitation to enroll. All subjects, who were affected by LHON caused by a mutated ND4 mitochondrial gene, were treated with an intravitreal injection of LUMEVOQ in one eye and with sham injection in the other.

Table 1. BCVA Mean Improvement Vs. Nadir* In LUMEVOQ Long-Term Follow-Up (RESTORE)

2 Years Post-Injection

3 Years Post-Injection3

4 Years Post-Injection

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LogMAR(Std Error)

LettersEquivalent**

LUMEVOQ-treated eyes

-0.375(0.306)

+18.8

-0.410(0.365)

+20.5

-0.453(0.440)

+22.5

Sham-treated eyes

-0.346(0.291)

+17.3

-0.387(0.369)

+19.4

-0.406(0.361)

+20.5

Note: The RESTORE sample consists of the RESCUE and REVERSE participants who accepted to be followed in the long-term follow-up study. Year 4 values were the LogMAR readings nearest to 1461 days post treatment recorded between 1461 +/- 273 days post- treatment. Missing values were imputed using the Last Observation Carried Forward (LOCF) method. *Nadir = worst best-corrected visual acuity recorded from baseline to Year 4. ** Assessments of best-corrected visual acuity (BCVA) were recorded in LogMAR. The change from nadir in LogMAR was converted to letters equivalent improvement by multiplying the LogMAR by -50 (ref. J.T. Holladay, J Refrac Surgery, 1997;13, 388-391).

Responder analyses at Year 4 indicate that improved BCVA was a benefit for a substantial proportion of the study participants. 71.0% of RESTORE subjects achieved Clinically Relevant Recovery (CRR)4 against nadir four years after treatment, and 80.7% of them had on-chart vision (BCVA 1.6 LogMAR) in one or both eyes.

Viewed against the trend in vision typically seen in untreated patients5, the findings represent a significant departure from the natural progression of LHON.

Safety findings at 4 years post-injection were consistent with previous readouts, which concluded that LUMEVOQ is well-tolerated: no serious adverse events were recorded among LUMEVOQ-treated eyes, and no discontinuations occurred due to ocular events. There were no systemic serious adverse events or discontinuations related to study treatment or study procedure.

The review of the European Marketing Authorisation Application for LUMEVOQ is ongoing, with the decision from the CHMP expected in Q4 2022.

References and notes:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,200-1,500 new subjects who lose their sight every year in the United States and the European Union.

About LUMEVOQ (GS010; lenadogene nolparvovec)

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About RESCUE, REVERSE, and RESTORE

RESCUE and REVERSE were two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measured the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, were used for statistical purposes. Both trials were adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between drug-treated and sham-treated eyes, adjusted to baseline.

The secondary endpoints involved the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was performed, including the proportion of patients who maintained vision (< ETDRS 15L loss), the proportion of patients who gained 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics included automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality-of-life scales, biodissemination and the time course of immune response. Readouts for these endpoints were at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were invited to participate in a long-term follow-up study, RESTORE, for three additional years.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767RESTORE: NCT03406104

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GenSight Biologics Reports Clinically Meaningful Vision Improvement is Maintained 4 Years After One-time Treatment with LUMEVOQ Gene Therapy -...

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AMSTERDAM--(BUSINESS WIRE)--VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of muscular and neurodegenerative disorders through vectorized antibodies, today announces a collaboration with Wageningen University to develop novel baculovirus-based technologies for the production of safe and affordable AAV gene therapies.

One of the most important challenges for the gene therapy industry is to develop robust and scalable manufacturing processes that yield safe therapies. VectorY, together with its partner Wageningen University, is making an important strategic investment in these therapies of the future, by developing a production platform capable of successfully manufacturing safe products at a significant lower COGS. The collaboration represents an important step to enable the medical and economical feasibility of gene therapy for diseases that affect larger patient populations.

Under the terms of the collaboration, the Bioprocess Engineering and Virology Groups at Wageningen University and VectorY Therapeutics will work together on two projects. One will utilize the molecular toolbox to generate innovative stable baculovirus genome seeds for AAV production at large scale. The second will focus on the design and evaluation of an intensified and scalable baculovirus production process in bioreactors using state of the art bioprocess technologies.

We are very pleased to be collaborating with VectorY to develop proprietary, next generation AAV expression systems and industry-leading bioprocessing capabilities, said Monique van Oers, Professor of Virology, Wageningen University.

We are honored to partner with Wageningen University, a significant pioneer in the Baculovirology and Bioprocessing field. This partnership will further strengthen VectorYs proprietary AAV production technologies for the development of Next Generation Gene Therapies, added Alexander Vos, CEO VectorY Therapeutics.

ENDS

Notes to Editors

About VectorY

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need. Founded in October 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform andantibody- based targeted degradation technologies, and proprietary manufacturing technology. VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

For more information, see http://www.vectorytx.com

Wageningen University & Research

The mission of Wageningen University and Research is To explore the potential of nature to improve the quality of life. Under the banner Wageningen University & Research, Wageningen University and the specialised research institutes of the Wageningen Research Foundation have joined forces in contributing to finding solutions to important questions in the domain of healthy food and living environment. With its roughly 30 branches, 6.800 employees and 12.900 students, Wageningen University & Research is one of the leading organisations in its domain. An integrated approach to problems and the cooperation between various disciplines are at the heart of Wageningens unique approach. As such, the Laboratory of Virology and The Bioprocess Engineering group work closely together in a number of biotechnological projects aimed at the further development of the baculovirus expression system for vaccine production and gene therapy applications. By combining our research efforts we can optimally profit from knowledge gained in fundamental virology and new technological developments in bioprocess engineering.

For more information, see https://www.wur.nl/en.htm

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BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, announced a research and development collaboration with Haihe Laboratory of Cell Ecosystem to develop hematopoietic stem cell regenerative therapies and platform technology by combining resources and expertise from both sides.

The Haihe Laboratory of Cell Ecosystem, run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is focused on conducting fundamental research, innovation, and translation in the cell ecosystem.

Under the agreement, both parties will jointly develop hematopoietic stem cell regenerative therapies, including the development of innovative genetically-modified hematopoietic stem cell therapies and the exploration of novel biomarkers to optimize quality control for stem cell production.

With top-notch resources and industry-university-research cooperation, well facilitate the development of cell-based medicine and therapies, said Professor Tao Cheng, Deputy Director of Haihe Laboratory of Cell Ecosystem and President of the Institute of Hematology and Blood Diseases Hospital at the Chinese Academy of Medical Sciences and Peking Union Medical College, a leading hematology researcher who has made a series of discoveries relating to the regulatory and regenerative mechanisms of hematopoietic stem cells. Hematopoietic stem cells (HSCs) have the potential for long-term self-renewal and can differentiate into various types of mature blood cells. These stem cells can be harnessed to provide treatment for a broad range of diseases such as hematological tumors, autoimmune diseases, and hereditary blood disorders. We believe that this collaboration with EdiGene will accelerate the innovation and translation in the field of HSCs, thus enabling healthier patients with new therapies."

Professor Cheng was awarded the second prize of the National Natural Science Award 2020 as the first author of work on basic and translational research that advanced the development of adult hematopoietic stem cells for therapeutic applications.

EdiGene is scaling up clinical translation and development of the first gene-editing hematopoietic stem cell therapy in China following the 2021 approval by the China National Medical Products Administration its IND for its investigational therapy ET-01. Our team has extensive experience in the development and translation of cutting-edge technologies including hematopoietic stem cell and gene editing, said Dong Wei, Ph.D., CEO of EdiGene. "This collaboration with Haihe Laboratory of Cell Ecosystem will further our exploration in the field of hematopoietic stem cells. The partnership with this leading academic institute and our translational know-how enable us to move forward in bringing more innovative treatment options to patients in China and around the world.

In 2021, EdiGene initiated a Phase I multicenter clinical trial of ET-01, its gene-editing hematopoietic stem cell therapy for transfusion-dependent -thalassemia. EdiGene has enrolled the first patient at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College. Currently, the clinical trial is being conducted in Tianjin and Guangdong-Hong Kong-Macao Greater Bay Area (Greater Bay Area). EdiGene also presented its latest research on new surface markers and migration of hematopoietic stem cells at the 63rd Annual Meeting of the American Society of Hematology (ASH) in 2021.

About Haihe Laboratory of Cell Ecosystem

The Haihe Laboratory of Cell Ecosystem ("the Laboratory"), run by the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, is one of the five registered Haihe Laboratories approved by Tianjin Municipal People's Government. With the goal of promoting population health with cell ecosystem, the Laboratory adheres to developing technological frontier, enhancing peoples health, and promoting research, innovation, and development of cell ecosystem in five key areas: cellular ecosystem, cellular ecology and immunity, cellular ecological imbalance and major diseases, cellular ecological reconstruction and frontier technology of cellular ecological research.

About Institute of Hematology and Blood Diseases Hospital (IH), Chinese Academy of Medical Sciences & Peking Union Medical College (CAMS/PUMC)

Founded in 1957, IH is a tertiary specialty hospital under the National Health Commission of China and is the supporting unit of the National Clinical Research Center of Hematologic Diseases and the State Key Laboratory of Experimental Hematology. It is also the main founding unit of Tianjin Base, the core base of the Chinese medical science and technology innovation system with the goal of becoming "the innovation hub of hematology in China." IH mainly engages in basic research, applied research, clinical diagnosis and treatment of hematological diseases, standard-setting, new technology research, new drug evaluation, and translation in hematology and related fields. IH is leading in the diagnosis and treatment of hematological diseases in China and a global scale and has made original achievements. Since 2010, IH has been awarded first place in the Hospital Specialty Reputation Ranking (Hematology) for 12 consecutive years. It has won first place in the Hematology Specialty Ranking for ten consecutive years since 2010 and ranked the first in hematology by the Scientific and Technological Evaluation Metrics (STEM) for Chinese hospitals for eight consecutive years since 2014.

About EdiGene, Inc

EdiGene is a global, clinical-stage biotechnology company focused on translating gene editing technologies into transformative therapies for patients with serious genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China and Cambridge, Massachusetts, USA. More information can be found at http://www.EdiGene.com.

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DUBLIN--(BUSINESS WIRE)--The "Protein Expression Global Market Report 2021: COVID-19 Growth and Change to 2030" report has been added to ResearchAndMarkets.com's offering.

The global protein expression market is expected to grow from $2.01 billion in 2020 to $2.13 billion in 2021 at a compound annual growth rate (CAGR) of 6%. The market is expected to reach $3.03 billion in 2025 at a CAGR of 9.2%.

Major players in the protein expression market are Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific Inc., New England Biolabs and Promega Corporation.

The protein expression market consists of sales of protein expression vectors, competent cells, reagents, equipment and related services. Protein expression is a process in which proteins are synthesized, modified, regulated and controlled in living organisms according to the host cell. Protein expression included yeast expression, insect expression, and bacterial expression, algal expression and mammalian cell expression.

The protein expression market covered in this report is segmented by protein expression into yeast expression, mammalian expression, algae expression, insect expression, bacterial expression, cell-free expression. It is also segmented by end use into pharmaceutical and biotechnological companies, academic research, contract research organizations; by product into reagents, competent cells, expression vectors, services, instruments and by application into therapeutic, industrial, research.

Government regulations related to protein therapeutics and production of biologics may hinder the protein expression market growth. Government regulations on biologics to undergo rigorous preclinical and clinical trials prior to regulatory approval, and time consuming process for approval of biologics with regards to health and the safety of any individual are restraining the market growth.

Marketing and distribution of biologics including insulin, hormones, therapeutic antibodies, and vaccines depends upon the successful completion of clinical trials, which is a long, expensive, and uncertain process. According to FDA, for an approval of new biologic, Under the regulations (21 CFR 314.81(b)(2)(vii) and 601.70, a clinical trial approval usually takes 10- 12 months where firms are required to submit a report annually on the status of clinical safety, clinical efficiency, clinical pharmacology, and nonclinical toxicology study.

Companies in the industry are increasingly adopting Microfluidics technology to enhance protein expression tests in order to reduce the time, cost, labor, and increase the accuracy and performance. The microfluidics technology effectively analyzes biological samples than the traditional (macroscale) instruments.

Microfluidics technology is used to measure the expression of proteins on cells and optimizes the output to generate results regarding protein expression. Therapeutics-on-a-chip (TOC) uses microfluidic platform and is able to synthesize proteins in a point of care setting to reduce cost associated with storage and transportation of therapeutic proteins.

For instance, companies such as MissionBio, NanoCellect Biomedical, RainDance Technologies and Sphere fluidics have implemented this technology in protein expression test.

Increase in demand for biologics to counter various genetic disorders and chronic diseases is one the major factors driving the research and sales of protein expression market. Biologics is a medicine produced from living organisms or contains components of living organisms such as protein, tissue, genes, allergens, cells, blood components, blood, and vaccines.

The increasing use of biologics (therapeutic protein and others) to cure chronic diseases such as cancer, cardiovascular conditions and genetic disorders, is increasing the demand for protein expression devices and equipment. According to the World Health Organization, chronic disease prevalence is expected to rise by 57% globally, by the year 2020.

Hence the rising demand for biologics is driving the protein expression market. For instance, according to an article published by Chemistry World, analysts expect the biologics market to hold a market share of more than a quarter of the entire pharmaceutical market by 2020. The global biologics market is expected to grow at 9.9% during 2018-2024.

The Protein Expression market in the U.S. is governed by Food and Drug Administration (FDA) that lays down a series of guidelines for the manufacturers and retailers of this industry. Within FDA, Center for Drug Evaluation and research (CDER) regulates biological products under FDA 101 which includes gene therapy products and vaccines. These regulations ensure quality, safety and efficacy of biological therapeutics products, and speed up innovations that make these products safer, and effective.

The US's FDA announced a fast-track initiative to review its drugs and biologics policy to speed the availability of therapies to patients with serious conditions, orphan drugs for rare disease, while preserving the safety and efficacy standards. FDA also removed a rule (Section 610.21 of the FDA code) which specified minimal potency limits for certain antibodies and antigens.

The European Medicines Agency has also introduced policies which include a provision to waive the scientific advice fee, which encourage more academic groups and small companies to propose candidates for biologics.

Key Topics Covered:

1. Executive Summary

2. Protein Expression Market Characteristics

3. Protein Expression Market Trends and Strategies

4. Impact Of COVID-19 On Protein Expression

5. Protein Expression Market Size and Growth

5.1. Global Protein Expression Historic Market, 2015-2020, $ Billion

5.1.1. Drivers Of the Market

5.1.2. Restraints On the Market

5.2. Global Protein Expression Forecast Market, 2020-2025F, 2030F, $ Billion

5.2.1. Drivers Of the Market

5.2.2. Restraints On the Market

6. Protein Expression Market Segmentation

6.1. Global Protein Expression Market, Segmentation by Protein Expression, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.2. Global Protein Expression Market, Segmentation by End Use, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.3. Global Protein Expression Market, Segmentation by Product, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

6.4. Global Protein Expression Market, Segmentation by Application, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7. Protein Expression Market Regional and Country Analysis

7.1. Global Protein Expression Market, Split by Region, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

7.2. Global Protein Expression Market, Split by Country, Historic and Forecast, 2015-2020, 2020-2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/iix75m

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Global Protein Expression Market Research Report 2021 Featuring Major Players - Agilent Technologies, Bio-Rad Laboratories, Thermo Fisher Scientific,...

Recommendation and review posted by Bethany Smith

The human heart, the size of a fist, located just behind and slightly left of the breastbone, tirelessly beats an average of 100,000 times a day. However, conditions that stop the heart from pumping blood efficiently can cause serious problems and ultimately require a heart transplantation.

In a study published in the journal 'Science Translational Medicine', researchers from Osaka University showed that a previously unknown mutation can lead to a condition called dilated cardiomyopathy, which is one of the main causes of heart failure.

Heart failure refers to an incurable condition where the heart is no longer able to meet the body's demands in terms of blood supply. It is one of the most common causes of death and it affects almost 40 million people worldwide, representing a huge public health problem. One of the main factors leading to heart failure is a disease called dilated cardiomyopathy (or DCM). DCM is characterized by dilation of the heart's chambers and a pumping disfunction. Previous research has shown that DCM is often inherited and has a genetic basis. However, for up to 80 per cent of the familial DCM cases, the genetic mutation causing the disease has still not been known.

ALSO READ: Can Omicron cause heart damage? Here's what experts say

The research team identified a gene called BAG5 as a novel causative gene for DCM. First, they studied patients from different families, highlighting a correlation between loss of function mutations in the BAG5 gene and DCM. The researchers found that this mutation has a complete penetrance, meaning that 100 per cent of the individuals presenting it will develop the disease. They then found in a mouse model of dilated cardiomyopathy that mice without BAG5 exhibited the same symptoms of human DCM, such as dilatation of the heart's chambers and irregular heart rhythm. This indicated that mutations that erase the function of BAG5 can cause cardiomyopathy.

"Here we showed that loss of BAG5 perturbs calcium handling in mouse cardiomyocytes," said Dr. Hideyuki Hakui, lead author of the study. BAG5 is important for calcium handling in the heart muscle cells, and calcium is essential for a regular rhythm and overall health of the cardiac muscle, explaining why a loss of BAG5 leads to cardiomyopathy.

"After demonstrating that BAG5 mutations led to loss of functional BAG5 protein," continued Dr. Yoshihiro Asano, senior author of the study, "we also showed that administration of an AAV9-BAG5 vector in a murine model could restore cardiac function. This finding suggests that gene therapy with adeno-associated viruses (AAV) should be further investigated as a possible treatment alternative to heart transplantation for patients who are BAG5 deficient." AAV gene therapy refers to an innovative form of therapy aimed at fixing mutated genes in diseases that have a genetic cause like DCM. Therefore, these findings have paved the way for a potential precision medicine treatment based on gene therapy.

More here:
Not just bones, calcium is vital for heart too: Study - Hindustan Times

Recommendation and review posted by Bethany Smith

New Jersey, United States,-The research study presented in this report offers a complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Gene Therapy Cell Culture Media Market. It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the Gene Therapy Cell Culture Media market. The authors of the report have segmented the Gene Therapy Cell Culture Media market as per product, application, and region. Segments of the Gene Therapy Cell Culture Media market are analyzed on the basis of market share, production, consumption, revenue, CAGR, market size, and more factors. The analysts have profiled leading players of the Gene Therapy Cell Culture Media market, keeping in view their recent developments, market share, sales, revenue, areas covered, product portfolios, and other aspects.

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The report includes company profiling of almost all important players of the Gene Therapy Cell Culture Media market. The company profiling section offers valuable analysis on strengths and weaknesses, business developments, recent advancements, mergers and acquisitions, expansion plans, global footprint, market presence, and product portfolios of leading market players. This information can be used by players and other market participants to maximize their profitability and streamline their business strategies. Our competitive analysis also includes key information to help new entrants to identify market entry barriers and measure the level of competitiveness in the Gene Therapy Cell Culture Media market.

Key Players Mentioned in the Gene Therapy Cell Culture Media Market Research Report:

Fujifilm Holdings Corporation, HiMedia Laboratories Pvt., Ltd, Lonza Group Ltd, Sartorius AG, Thermo Fisher Scientific Inc., Merck KGaA, Danaher Corporation, Takara Holdings Inc., Novartis International AG, Bio-Techne Corporation.

Gene Therapy Cell Culture MediaMarket Segmentation:

Gene Therapy Cell Culture Media Market, By Media Type

Serum-containing Media Serum-free Media Stem Cell Media Specialty Media Chemically Defined Media

Gene Therapy Cell Culture Media Market, By End User

Biotechnology & Pharmaceutical Industry Academic Institute Research Laboratory

The Gene Therapy Cell Culture Media market is segmented as per the type of product, application, and geography. All of the segments of the Gene Therapy Cell Culture Media market are carefully analyzed based on their market share, CAGR, value and volume growth, and other important factors. The report also provides accurate estimations about the CAGR, revenue, production, sales, and other calculations for the Gene Therapy Cell Culture Media market. Each regional market is extensively studied in the report to explain why some regions are progressing at a high rate while others at a low rate. We have also provided Porters Five Forces and PESTLE analysis for a deeper study on the Gene Therapy Cell Culture Media market

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Gene Therapy Cell Culture Media Market Report Scope

Geographic Segment Covered in the Report:

TheGene Therapy Cell Culture Mediareport provides information about the market area, which is further subdivided into sub-regions and countries/regions. In addition to the market share in each country and sub-region, this chapter of this report also contains information on profit opportunities. This chapter of the report mentions the market share and growth rate of each region, country and sub-region during the estimated period.

North America (USA and Canada) Europe (UK, Germany, France and the rest of Europe) Asia Pacific (China, Japan, India, and the rest of the Asia Pacific region) Latin America (Brazil, Mexico, and the rest of Latin America) Middle East and Africa (GCC and rest of the Middle East and Africa)

Key questions answered in the report:

1. Which are the five top players of the Gene Therapy Cell Culture Media market?

2. How will the Gene Therapy Cell Culture Media market change in the next five years?

3. Which product and application will take a lions share of the Gene Therapy Cell Culture Media market?

4. What are the drivers and restraints of the Gene Therapy Cell Culture Media market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Gene Therapy Cell Culture Media market throughout the forecast period?

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VMI provides a holistic overview and global competitive landscape with respect to Region, Country, and Segment, and Key players of your market. Present your Market Report & findings with an inbuilt presentation feature saving over 70% of your time and resources for Investor, Sales & Marketing, R&D, and Product Development pitches. VMI enables data delivery In Excel and Interactive PDF formats with over 15+ Key Market Indicators for your market.

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About Us: Verified Market Research

Verified Market Research is a leading Global Research and Consulting firm that has been providing advanced analytical research solutions, custom consulting and in-depth data analysis for 10+ years to individuals and companies alike that are looking for accurate, reliable and up to date research data and technical consulting. We offer insights into strategic and growth analyses, Data necessary to achieve corporate goals and help make critical revenue decisions.

Our research studies help our clients make superior data-driven decisions, understand market forecast, capitalize on future opportunities and optimize efficiency by working as their partner to deliver accurate and valuable information. The industries we cover span over a large spectrum including Technology, Chemicals, Manufacturing, Energy, Food and Beverages, Automotive, Robotics, Packaging, Construction, Mining & Gas. Etc.

We, at Verified Market Research, assist in understanding holistic market indicating factors and most current and future market trends. Our analysts, with their high expertise in data gathering and governance, utilize industry techniques to collate and examine data at all stages. They are trained to combine modern data collection techniques, superior research methodology, subject expertise and years of collective experience to produce informative and accurate research.

Having serviced over 5000+ clients, we have provided reliable market research services to more than 100 Global Fortune 500 companies such as Amazon, Dell, IBM, Shell, Exxon Mobil, General Electric, Siemens, Microsoft, Sony and Hitachi. We have co-consulted with some of the worlds leading consulting firms like McKinsey & Company, Boston Consulting Group, Bain and Company for custom research and consulting projects for businesses worldwide.

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Continue reading here:
Gene Therapy Cell Culture Media Market Size | Global Industry Trends, Segmentation, Business Opportunities And Forecast To 2029 The Oxford Spokesman...

Recommendation and review posted by Bethany Smith

RANDY HUTCHINSON| Better Business Bureau

The FDA says that stem cell products may offer the potential to treat many medical conditions and diseases for which few alternative treatments exist, but almost all of them have yet to be proven to be effective or safe. That hasnt stopped unscrupulous medical professionals and other promoters from touting them as miracle cures.

Stem cells are sometimes called the bodys master cells because they develop into blood, brain, bones and the bodys other organs. Stem cells that come from bone marrow or blood are routinely used to treat cancer and other disorders of the blood and immune system. But other uses, including treating COVID-19, have not been properly studied under an FDA Investigational New Drug Application, which includes providing sufficient data from human clinical testing to help evaluate a products potential risks.

The FDA cites these potential safety concerns for unproven treatments:

There are safety risks even with your own stem cells and contamination can occur if cells are manipulated after removal.

The FTC and Georgia Attorney General sued the co-founders of the Stem Cell Institute of America for allegedly targeting seniors with bogus claims that its therapy could treat arthritis, joint pain and other orthopedic ailments. The agencies said the company also claimed its stem cell therapy is comparable or superior to surgery, steroid injections and painkillers. A related company trained chiropractors and other healthcare practitioners in making similar claims. They also hosted free educational seminars for consumers at which they promoted injections that cost approximately $5,000 per joint, with many patients receiving multiple injections.

This wasnt the FTCs first enforcement action against deceptive stem cell therapy claims. In 2018, a California physician and his companies settled with the FTC over claims their amniotic stem cell therapy could treat Parkinsons disease, autism, macular degeneration, cerebral palsy, multiple sclerosis, heart attacks and a host of other serious ailments. The defendants earned at least $3.3 million offering injections that cost up to $15,000.

The only stem cell products approved by the FDA for use in the United States consist of blood-forming stem cells derived from umbilical cord blood. Theyre approved for limited use in patients with disorders that affect the production of blood.

In bringing the most recent action, the Acting Director of the FTCs Bureau of Consumer Protection said, At best, the use of unproven products or therapies can cost consumers thousands of dollars without affording them any results. At worst, it can be harmful to their health.

If youre considering a stem cell treatment, the FDA says to make sure its FDA-approved or being studied under an Investigational New Drug Application. The FTC and BBB offer these additional tips:

Randy Hutchinson is the president of the Better Business Bureau of the Mid-South. Reach the BBB at 800-222-8754.

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Beware of unproven stem cell therapies - The Jackson Sun

Recommendation and review posted by Bethany Smith

New Jersey, United States,-ThisStem Cell TherapyMarketreport provides a clear picture of key players growth as well as the qualitative aspects of business in each area. ThisStem Cell TherapyMarket Report provides a current report on revenue generation, recent trends, financial status, and costing, as well as business profiles and financial status. The competitive landscape and potential growth factors are presented in thisStem Cell TherapyMarket Report.This way market report buyers can get a clear picture of the significant growth and the resulting market strategy. Granular market data would help monitor potential profitability and make critical growth decisions.

Significant industry central participants are included in thisStem Cell Therapymarket report. Diverse market factors like sort, size, applications, and end-clients have been incorporated to examine organizations altogether in thisStem Cell Therapymarket report. Significant mainstays of the organizations, companies and industries that influence the high points and low points of organizations are moreover recollected for thisStem Cell Therapymarket report.The research, interviews with key market players, and questionnaire have been compiled based on the continuous changes in the market, challenges and restraints faced by organizations, and global autonomies to expand the field in the coming years.

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This unique Stem Cell Therapy market report also discusses COVID-19 impact and influence on the worldwide market. How might one work around it or come up with fresh techniques so that COVID-19 doesnt stifle market growth are also discussed this report. This fascinating Stem Cell Therapy market report has proven to be of great benefit to current market participants by providing them with a broad understanding of market dynamics. This Stem Cell Therapy market report is a reliable technique of the general industry scenario that indicates basic leadership if they will gain or lose money in this market.

Key Players Mentioned in the Stem Cell Therapy Market Research Report:

Osiris Therapeutics Medipost Co. Ltd., Anterogen Co. Ltd., Pharmicell Co. Ltd., HolostemTerapieAvanzateSrl, JCR Pharmaceuticals Co. Ltd., Nuvasive RTI Surgical Allosource

Stem Cell TherapyMarket Segmentation:

Stem Cell Therapy Market, By Cell Source

Adipose Tissue-Derived Mesenchymal Stem CellsBone Marrow-Derived Mesenchymal Stem CellsCord Blood/Embryonic Stem CellsOther Cell Sources

Stem Cell Therapy Market, By Therapeutic Application

Musculoskeletal DisordersWounds and InjuriesCardiovascular DiseasesSurgeriesGastrointestinal DiseasesOther Applications

Stem Cell Therapy Market, By Type

Allogeneic Stem Cell Therapy Market, By ApplicationAutologous Stem Cell Therapy Market, By Application

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Stem Cell Therapy Market Report Scope

Determining the pulse of the market becomes easy through this in-detail Stem Cell Therapy market analysis. Key players can find all competitive data and market size of major regions like North America, Europe, Latin America, Asia-Pacific and Middle East. As part of the competitive analysis, certain strategies are profiled which are pursued by key players such as mergers, collaborations, acquisitions and new product launches. These strategies will greatly help industry players to strengthen their market position and grow their business.

Key questions answered in the report:

1. Which are the five top players of the Stem Cell Therapy market?

2. How will the Stem Cell Therapy market change in the next five years?

3. Which product and application will take a lions share of the Stem Cell Therapy market?

4. What are the drivers and restraints of the Stem Cell Therapy market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Stem Cell Therapy market throughout the forecast period?

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Verified Market Intelligence is our BI-enabled platform for narrative storytelling of this market. VMI offers in-depth forecasted trends and accurate Insights on over 20,000+ emerging & niche markets, helping you make critical revenue-impacting decisions for a brilliant future.

VMI provides a holistic overview and global competitive landscape with respect to Region, Country, and Segment, and Key players of your market. Present your Market Report & findings with an inbuilt presentation feature saving over 70% of your time and resources for Investor, Sales & Marketing, R&D, and Product Development pitches. VMI enables data delivery In Excel and Interactive PDF formats with over 15+ Key Market Indicators for your market.

Visualize Stem Cell Therapy Market using VMI @ https://www.verifiedmarketresearch.com/vmintelligence/

About Us: Verified Market Research

Verified Market Research is a leading Global Research and Consulting firm that has been providing advanced analytical research solutions, custom consulting and in-depth data analysis for 10+ years to individuals and companies alike that are looking for accurate, reliable and up to date research data and technical consulting. We offer insights into strategic and growth analyses, Data necessary to achieve corporate goals and help make critical revenue decisions.

Our research studies help our clients make superior data-driven decisions, understand market forecast, capitalize on future opportunities and optimize efficiency by working as their partner to deliver accurate and valuable information. The industries we cover span over a large spectrum including Technology, Chemicals, Manufacturing, Energy, Food and Beverages, Automotive, Robotics, Packaging, Construction, Mining & Gas. Etc.

We, at Verified Market Research, assist in understanding holistic market indicating factors and most current and future market trends. Our analysts, with their high expertise in data gathering and governance, utilize industry techniques to collate and examine data at all stages. They are trained to combine modern data collection techniques, superior research methodology, subject expertise and years of collective experience to produce informative and accurate research.

Having serviced over 5000+ clients, we have provided reliable market research services to more than 100 Global Fortune 500 companies such as Amazon, Dell, IBM, Shell, Exxon Mobil, General Electric, Siemens, Microsoft, Sony and Hitachi. We have co-consulted with some of the worlds leading consulting firms like McKinsey & Company, Boston Consulting Group, Bain and Company for custom research and consulting projects for businesses worldwide.

Contact us:

Mr. Edwyne Fernandes

Verified Market Research

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Email: sales@verifiedmarketresearch.com

Website:- https://www.verifiedmarketresearch.com/

See the original post here:
Stem Cell Therapy Market Size, Business Opportunities and Forecast to 2029 The Oxford Spokesman - The Oxford Spokesman

Recommendation and review posted by Bethany Smith

DuBOIS Cayleigh Walkers hopes for a successful basketball season were almost benched when she tore her ACL playing summer league basketball at the YMCA in July 2021. However, the Clearfield High School sophomore resumed training just three weeks following her August surgery, fast tracking her return to the court with the Lady Bison varsity basketball team.

I was going up the court and felt it pop, Cayleigh recalled. I was in so much pain. I had to sit out the rest of the game while some of the parents tried to stretch it out. I was eager to have it examined because we were leaving for the beach two days later. Fortunately I was able to get an appointment to have it examined the next morning.

Cayleigh and her parents chose Penn Highlands Orthopedics and Sports Medicine for her care. At Penn Highlands, she was examined by Tyler Beers, PA-C, who tested her range of motion and felt around the injured area. He gave her a brace to wear and ordered an MRI which was scheduled the following week when the Walkers returned from their vacation.

The MRI revealed the torn ACL. The next day, Cayleigh met with Dr. Matthew A. Varacallo, medical director of Orthopedic Robotic Surgery at Penn Highlands Healthcare. The orthopedic surgeon specializes in sports medicine, total joint reconstruction, accelerated rehabilitation protocols and functional return to sports after surgeries and procedures.

Named to the Top 65 Total Knee Replacement Surgeons to Know by Beckers ACS Review, Dr. Varacallo pioneered the innovative Fertilized ACL technique along with Dr. Chad Lavender, an orthopedic surgeon at Marshall University in West Virginia. Currently, they are the only two surgeons in the US using the technique when performing ACL reconstruction surgery.

Typically, when an ACL tear occurs, one third of the athletes re-tear the same side or injure the other knee; but, the theory behind the Fertilized ACL technique is to improve and expedite bone tunnel healing rates, followed by graft revascularization and ligamentization (the process by which the tendon becomes a ligament) to improve graft function and incorporation into the knee joint, explained Dr. Varacallo.

The Fertilized ACL procedure begins when bone marrow is removed from the tibia the long bone on the inside of the lower leg which is rich in growth hormone and stem cells. A specialized perfusionist takes 60 milliliters of the bone marrow and spins it down to bone marrow aspiration concentrate (BMAC) which is used to initiate healing. The BMAC is mixed with bone graft. Small tunnels are then made in the femur and tibia to place the graft during the procedure. The bone graft mixture and stem cells/growth factors are then injected into the tunnels to help stimulate them to heal faster. Once the tunnels heal, the tendon can in theory possibly transform into a ligament faster.

This is truly a cutting edge procedure, explained Dr. Varacallo. In traditional ACL surgeries, the pressurized tunnels can be risk factors for re-injury because they can take up to six or seven months to heal. However, with the Fertilized ACL procedure, the tunnels heal faster because the graft starts to incorporate into the body faster. In fact, four weeks post surgery, you cannot even see the bone tunnels, the surgeon added.

Dr. Varacallo considers Cayleigh the poster child for successful ACL reconstruction because she is achieving milestones sooner than expected, and Samantha (Sam) Morgan, MS ATC PES, a certified athletic trainer with Penn Highlands Healthcare as well as with the Clearfield Area School District agrees.

Three days following surgery, Cayleigh began rehab, and at three and one half weeks post op she was running, and six to seven weeks following surgery she was punting a soccer ball. In contrast, with traditional ACL surgery, we would not see that type of progress until three or four months post op, Ms. Morgan detailed.

According to Dr. Varacallo, Cayleigh is several months ahead of where she should be in terms of her progress based on the functional testing ACL Report Card, The Report Card, which is used to measure how well a patient is meeting 15 different functional test milestones at two, four, six and eight months post op, was developed at the University of Kentucky where Dr. Varacallo completed his fellowship in Orthopedic Surgery and Sports Medicine.

The ACL Report Card is a great benchmark tool because it not only shows the medical team the patients progress, it gives the athlete something to shoot for in their rehab, said Dr. Varacallo.

I had four months of rehab five days a week, Cayleigh said, The first few weeks were challenging because I was in some pain, but Sam pushed me and helped me get through it.

At two months after surgery, Cayleigh was testing stronger on the surgery side than on the other non-injured side. One of the factors that contributed to her faster recovery was that prior to surgery she and the other patients who have Fertilized ACL reconstruction surgery have prehab.

We cannot operate on a stiff and weak knee, so prior to surgery, we have the patient undergo motion and strengthening exercises to improve outcomes following surgery, Dr. Varacallo explained. The stronger the knee heading into surgery, the faster the recovery.

In addition to enhancing flexibility, prehab teaches patients the exercises they will be doing during post-surgery rehab so that they are already familiar with what their physical therapy will entail.

Cayleigh had aggressive prehab, said Morgan. In addition to promoting flexibility of the knee, it is an educational tool that helps the patient understand the how and why of the injury and healing. Three days following surgery, Cayleigh started rehab, and because she had the prehab, she already knew how to perform her exercises.

The prehab, surgery and rehab are all interdependent on each other. In fact, Dr. Varacallo uses an analogy to tie the process together. I think of the reconstruction and healing process as a house the prehab is the foundation, surgery is the main living level and rehab is the attic.

According to Cayleighs mother, Tammy Walker, her daughters fast recovery is a combination of three factors, Dr. Varacallos innovative Fertilized ACL technique, the aggressive rehabilitation and equally important the determination by Cayleigh and Morgan to get her back to where she could play again.

We are very impressed with Dr. Varacallo, said Walker. He was very thorough and went through everything with us and even called to check on her progress following the surgery.

More here:
Penn Highlands Healthcare surgeon pioneers innovative ACL surgery offering faster recovery - The Courier-Express

Recommendation and review posted by Bethany Smith

Exclusive:

Football ace Ashley Cain and his girlfriend Safiyya Vorajee have been supporting parents of children battling cancer. The couple's baby girl Azaylia died last April

Image: Instagram)

Footballer Ashley Cain has been offering hope and solace to parents of a little girl battling the cancer that claimed the life of his own precious daughter.

Reality star Ashley and girlfriend Safiyya Vorajee have been supporting dozens of sick childrens families since the death of their baby girl.

Beloved Azaylia died last April after a courageous fight against the blood cancer acute myeloid leukaemia.

The ex-Coventry City winger and his partner have now struck up a special friendship with the family of five-year-old Esha Nadeswaran, who has spent eight months fighting the same disease at Londons Great Ormond Street Hospital.

Her dad Rishya, 45, of Gants Hill, North East London, said: Esha is our absolute hero and she is incredible. With our journey, it is like a marathon with no finish. We dont know when it is going to end and so we cant even pace ourselves.

There have been days where Ive gone to bed, crawled up and cried alone. But no matter how bad a day you are having and how much you hurt inside, Ashley reminded me you must stay positive and happy for your child.

He told me not to think too far ahead and do whatever I needed to do to support Esha. Its horrible to be told your child has cancer.

Listening to Ashley, and how vulnerable he felt, really struck a chord. Our chat has kept me going.

Former Ex On The Beach star Ashley, 31, and Safiyya, 34, were devastated to lose Azaylia when she was just eight months old.

She had been given stem cell treatment, which involves replacing damaged cells with healthy ones from a blood-match donor.

Ashley said Azaylia had the heart of a lion and he was given precious extra months with her after 100,000 people registered to be a donor.

The couple got involved in Eshas battle after Rishya and wife Kavitha, 41, appealed to members of the South Asian community to volunteer to be stem cell donors.

Esha has been in hospital since last May. She has had gruelling chemo and, in October, an emergency stem cell transplant. With her parents at her bedside, constant care includes regular platelet and red blood cell transfusions.

Rishya recalls the day they received the diagnosis. He says: She had been having tummy issues. Doctors ran tests and we were told wed receive the results in a fortnight. Two hours after returning home, they rang and we knew something was badly wrong. We rushed back and were told she had an acute form of leukaemia. It has been utterly heartbreaking.

Eshas Sri Lankan heritage meant it was hard to find a donor match and the family launched a social media campaign. Safiyya then reached out.

Rishya says: I arranged a Zoom chat with Safiyya and Ashley and I spoke father to father about what it feels like to be told your child has cancer. It was very emotional.

Ashley and Safiyya also recorded a video message on Eshas birthday.

Rishya, who works in banking, says: Esha really liked it. She is such a caring little girl and the way she has bounced back to defy doctors is something of a miracle.

She is always the one who goes from room to room cheering up the other children.

Rishya and his family now have 10,000 Instagram followers. But what they havent shared is their sacrifices to give her the best possible chance of survival.

Forming a Covid bubble, Rishya and his wife have had to live apart from elder daughter Ria, 10, who is with her grandparents.

They see her once a week at arms length and were apart at Christmas and on both girls birthdays. Its been really tough, says Rishya. My wife and I take it in turns to stay with Esha. The Young Lives vs Cancer charity has kindly been able to offer accommodation for the other to stay nearby.

We have been locked in this bubble for months now and its exhausting.

Ashley and Safiyya have helped dozens of parents. They set up a foundation raising money for treatment that may not be available on the NHS.

Image:

He says: No matter how painful it is, our mission is to really help and battle for these kids fighting cancer.

We speak to parents privately and try to guide them through the experience. We know first hand the stress and trauma parents have to go through.

Eshas dad and I talked about appreciating every single moment, making sure your child thinks theres nothing wrong. You need to stay positive and be the best you can be.

The pain of losing Azaylia is with us every minute. We go to her resting place every day and I want to hold this pain forever to use it effectively to help other children have a better chance.

Safiyya adds: So many children are suffering and we want to help.

The couple are planning a fundraising cycling event in June in conjunction with the Tour of Cambridgeshire.

Ashley says: Wed like everyone to wear orange and create a sea of orange so that Azalyia can look down from heaven and see what an amazing effect she has had on this world.

To join the cycle event, go to cycleforazaylia.com. Get updates on Esha at instagram.com/for_esha

Esha has the illness that cost Azaylia her life acute myeloid leukaemia, cancer of the white blood cells.

It starts in the bone marrow and spreads to the bloodstream and is very rare in young children.

The risk increases with age and it is most common in people aged over 75.

Acute leukaemia means it progresses quickly and chemotherapy is the main treatment, killing off as many unhealthy cells as possible and reducing the risk of the patient suffering a relapse.

Stem cell therapy involves removing a persons unhealthy cells and replacing them with healthy ones from a donor.

The new cells travel in the blood of the patient to the bone marrow, where they attach and grow.

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Continue reading here:
Ashley Cain supports parents of girl, 5, battling same cancer which killed his baby - The Mirror

Recommendation and review posted by Bethany Smith

This article was originally published here

J Oncol Pharm Pract. 2022 Jan 27:10781552221074269. doi: 10.1177/10781552221074269. Online ahead of print.

ABSTRACT

INTRODUCTION: Plasma cell leukemia (PCL) is a rare but aggressive variant of multiple myeloma (MM) with a poor prognosis. Due to the limited number of prospective clinical trials studying PCL, treatment options are often extrapolated from data available for the treatment of MM. Venetoclax has recently demonstrated antimyeloma activity in patients with relapsed/refractory MM carrying the t(11;14) translocation. However, few cases have reported the analogous efficacy of venetoclax in PCL.

CASE REPORT: A 64-year-old Caucasian male developed relapsed PCL despite treatment with hyperCD (hyperfractionated cyclophosphamide and dexamethasone) and Dara-KRd (daratumumab, carfilzomib, lenalidomide, dexamethasone). Due to the refractory nature of his disease and the presence of a t(11:14) translocation, the patient was subsequently initiated on venetoclax 400 mg daily and dexamethasone 4 mg once weekly.

MANAGEMENT AND OUTCOME: The patient achieved a complete response by International Myeloma Working Group criteria three months after initiating venetoclax-dexamethasone, including a repeat bone marrow biopsy that showed no abnormal plasma cells. He successfully underwent consolidation with melphalan-based autologous stem cell transplantation. He remains disease-free 9 months after venetoclax initiation.

DISCUSSION: Combination all-oral therapy with venetoclax and dexamethasone can induce deep hematologic responses in patients with relapsed/refractory PCL carrying the t(11;14) translocation.

PMID:35084252 | DOI:10.1177/10781552221074269

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Complete response following treatment of plasma cell leukemia with venetoclax and dexamethasone: A case report - DocWire News

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