StemCell Therapy

Looking to the rheumatology space, UK-based digital health company Ampersand Healthis rolling out a new product called My Arthritis DTxto support patients with inflammatory arthropathies.

Patients with rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis can use the tool to self-managetheir condition. It provides users with educational resources and courses, and can connect to their clinical team if their UK National Trust uses the tool. Patients can asynchronously communicatewith their care teams.

Patients using the tool can tap into cognitive behavioral therapy, acceptance and commitment therapy, and mindfulness services. The tool also lets users track their arthritis over time, as well as monitortheir physical health, mental health and sleep.

HIMSS20 Digital

Currently, the product is in clinical trials. This marks Ampersand Health's second digital product. Its first product, My IBD Care, focuseson helping individuals with Crohn's disease or ulcerative colitis.

WHY IT MATTERS

Inflammatory arthropathies are very common. In fact, more than 54 million people in the U.S. have arthritis, according to the CDC.

The agency says that age increases the likelihood of the condition, as well as obesity. Patients can manage the condition through education programs, activities and losing weight, according to the agency.

THE LARGER TREND

Several digital health companies are working on products to support arthritis patients. In June, SidekickHealthpartnered with Pfizerto launch an appaimed at helping patients with diseases including rheumatoid arthritis manage their conditions from home. The deal is thought to be worth more than $8 million.

The interest in the space has long a history. In 2018,MyHealthTeams announced a deal to join forces with UCB to add a new spondyloarthritis social network. UCB has also worked with Garmin on a wellness program for rheumatoid arthritis patients.

Swedish startup Joint Academy raised $23 million in Series Bround of funding in September 2020 for its clinical evidence-based digital treatment for chronic joint pain, which connects patients with licensed physical therapists. Formerly calledArtho Therapeutics,its total funding raise comes to $34.2 million.

The rest is here:
UK-based Ampersand Health rolls out My Arthritis DTx - Mobihealth News

Introduction

Rheumatoid arthritis (RA) is a chronic systemic autoimmune disease characterized by persistent synovitis, systemic inflammation and the presence of autoantibodies. Uncontrolled active RA causes joint damage, disability, decreased quality of life, and cardiovascular and other comorbidities.1 Current recommendations state that therapy with disease-modifying antirheumatic drugs (DMARDs) should be started as soon as the diagnosis of RA is confirmed.2

Adherence can be defined as the process by which patients take their medications as prescribed. This process includes initiation of the drug, implementation of the prescribed regimen and discontinuation of the drug.3 Lack of adherence contributes to an inadequate response or failure to treatment, worsening or disease relapse, and unnecessary treatment changes.4 It has been stated that compliance declines over time.5 This is important because a lack of adherence to pharmacologic therapy is a prevalent issue in the treatment of chronic diseases such as RA.

Adherence has not been widely examined for most rheumatic conditions.6 The ability of physicians to recognize nonadherence is poor, and interventions to improve adherence have had mixed results.7 Currently, a gold standard for the measurement of adherence is not available.7,8 The use of patient questionnaires, an indirect method to measure adherence, is an inexpensive and useful method due to its simplicity.7 A compliance questionnaire in Rheumatology (CQR) was developed to measure compliance to treatment and to identify factors that contribute to suboptimal adherence in patients suffering from RA, polymyalgia rheumatica and gout.9 This 19-item measure has been proven to be useful to predict compliance and identify barriers that interfere with it.9,10 Recently, the Korean and the Spanish versions of CQR, sCQR, for patients with RA have been validated.11,12 They showed high reliability with good testretest results and a high predictive value suggesting that they could be used as screening instruments. In addition, the use of sCQR could also help to identify reasons for nonadherence.12

Increased knowledge of the impact of therapeutic adherence of patients with RA, and identification of possible predictors of adherence will allow to develop strategies to promote adherence. The main objective of this work was to describe the prevalence of treatment adherence in patients with RA in Spain using the sCQR. Secondary objectives were to detect possible differences in adherence in patients receiving biologic DMARDs (bDMARDs) compared to conventional DMARDS (cDMARDs) and/or glucocorticoids, in patients receiving intravenous therapies compared to other routes of administration and in patients treated in Rheumatology specific day hospitals versus polyvalent day hospitals. Another secondary objective was to identify potential predictors of adherence.

We performed an observational, cross-sectional, multicenter study in outpatient clinics of Rheumatology Departments from 41 centers in Spain.

Patients were invited to participate in the study during a routine visit to the rheumatology outpatient departments. In case of agreement, the sCQR was completed by the patient in the waiting room before seeing the rheumatologist and deposited in a box. In no case, the rheumatologist had access to the answers. If respondents did not understand any specific question or had trouble reading it, it could be read aloud to them verbatim, in any case, this would induce patients to respond in a certain way. Demographic and disease data were collected during the visit. No diagnostic or therapeutic interventions other than those required by the routine practice were performed. Available data at the moment of the visit were obtained to ensure reflecting real-world practice with no interference.

Adult patients that fulfilled the 1987 American College of Rheumatology (ACR) criteria for RA or RA diagnosis given by a rheumatologist, attending routine visits, were recruited on a consecutive basis. They had to be treated with glucocorticoids, cDMARDs (methotrexate, leflunomide, sulfasalazine, antimalarials) and/or bDMARDs (infliximab, adalimumab, etanercept, certolizumab, golimumab, tocilizumab, rituximab, abatacept) for at least 3 months and give their informed consent in order to participate in the study. Patients had to have the ability to complete the questionnaire or have someone who could help them in its completion.

To calculate sample size, results obtained in the preliminary study of the transcultural adaptation of sCQR were considered, where a 78% adherence to treatment was established.12 Estimating a similar proportion of therapeutic adherence and having in mind that the prevalence of RA in Spanish population is 0.5%13 and Spanish population is 46,439,864 inhabitants, for an alpha error of 5% and an accuracy of 3% it was estimated that the necessary sample would be 730 patients. Assuming a loss percentage of 15%, a total population of 859 patients was determined to be necessary.

To achieve a representative sample, centers were selected across the Spanish geography according to population density and public health spending. Approximately 40 centers were estimated to be necessary. At least one region was randomly selected from each of the four groups, created according to stratification criteria, depending on population and health expenditure (supplementary Figure 1).

An effort was made to include as many regions and centers in the study. In each region, second- and third-level centers participated so that the sample was as representative as possible. The geographic variability of the recruited centers also contributed to guarantee this. The established minimum number of centers being selected in each group depended on the population of each of the groups.

The compliance questionnaire in rheumatology (CQR) is a 19-item questionnaire that encompasses various aspects of adherence (Box 1). Spanish validated version was used in this study. Items indicating greater adherence (questions 13, 57, 10 and 1318) were scored from 3 to 0 (3, strongly agree; 2, agree; 1, little agreement; 0, completely disagree). Items indicating poorer adherence (questions 4, 8, 9, 11, 12 and 19) were scored from 3 to 0 (0, strongly agree; 1, agree; 2, little agreement; 3, completely disagree). To adjust its weight, a coefficient was applied to each item, to generate a Z score (ZK): ZK = a + W1X1K + W2X2K + + W19X19K, where ZK is the discriminant Z score for patient K, a is a constant, Wi is the discriminant weight for item i and patient K. A cutoff point allowed classifying patients into those with satisfactory compliance or unsatisfactory compliance.12

Box 1 Compliance Questionnaire in Rheumatology

The following data were collected: demographic data (age, sex, study level, civil status and cohabitation), data related to RA (disease duration, number of drugs used to treat RA, auxiliary drugs were not considered, eg folic acid or vitamin D, glucocorticoids use and route of administration, cDMARD use and route of administration, bDMARD use and route of administration), hospital infrastructure (specific day hospital versus polyvalent).

The study was approved by Santiago-Lugo Ethics Committee (Registry Code: 2017/296 dated 29/05/2018). All the procedures were performed in accordance with the requirements for studies involving human participants and followed the principles stated in the Declaration of Helsinki. Both informed and written consent were sought from each participant using a consent form before enrollment in the study. Survey confidentiality and anonymity were assured to all enrolled participants.

A descriptive analysis of all of the variables included in the study was performed. To identify if there were differences depending on the treatment received (bDMARDs versus cDMARDs), its route of administration or the area of origin, Chi2 was used. Likewise, in order to identify adherence predictive factors, a univariate and multivariate linear regression study adjusted for significant variables and for age and sex variables was performed. Values of statistical significance were considered at p < 0.05. In the regression analysis, the categorical variables included were the number of medications the patient was taking, steroid use, use of cDMARDs, routes of administration of cDMARDs, use of bDMARDs, routes of administration of bDMARDs, health area, sex, level education, marital status and cohabitation. Continuous variables included in the regression analysis were the age and disease duration. For the statistical analysis, Stata version14.0 (Stata/MP14.0 for Windows; StataCorpLP, College Station, TX) was used.

A total of 859 patients contributed by 41 centers were selected and included in the study. All patients answered the questionnaire, 729 patients completed the 19 items and 130 skipped at least one of them. For analysis purposes, we considered patients who completed the 19 items. Baseline patient characteristics are shown in Table 1. A total of 418 patients (48.7%) were being treated with bDMARDs and 682 (79.3%) were receiving cDMARDs. Five hundred and sixty-five patients (65.8%) were receiving more than one drug for the treatment of RA (glucocorticoids, cDMARDs and/or bDMARDs) at the time they filled the questionnaire.

Table 1 Baseline Characteristics of Patients

An adherence rate of 79.01% was established. As for the secondary objectives, no differences were determined in adherence among patients related to the type of drugs they were receiving, bDMARDs versus cDMARDs (p = 0.1442), among patients receiving intravenous therapies versus other routes of administration (p = 0.7453) and among patients treated in specific day hospitals versus polyvalent day hospitals (p = 2.6815). The use of bDMARD combined with cDMARD also showed no difference in adherence compared to bDMARD monotherapy administration (p=0.314).

The univariate analysis detected the number of drugs and cohabitation as predictors of adherence to treatment (Table 2). When performing the multivariate analysis adjusted for sex and age, the same two variables remained as predictors of adherence, determining that both, number of drugs and cohabitation, are independent predictors of adherence.

Table 2 Association of Study Variables with Treatment Adherence

To test for sensitivity, the same statistical analysis was performed including all of the 859 patients, without ruling out the patients who skipped questionnaire items, considering these patients as non-adherents. The univariate analysis detected the same variables, number of drugs and cohabitation, as predictors of adherence to treatment (supplementary table 1). Civil status and age were also significant. Multivariate analysis adjusted for sex and age confirmed the number of drugs as an independent predictor of adherence.

Finally, ANOVA analysis was performed to test for sex and age influence on adherence and no statistical significance was detected.

This study conducted in Spanish population shows that adherence to treatment occurred in 79% of patients with RA. Multivariate analysis, adjusted for age and sex, revealed that a number of drugs and cohabitation were independently associated with adherence in this population.

There are currently different methods to assess medication adherence. A lack of consensus exists when determining which is the best instrument.14 This study was conducted using a highly reliable and specific tool, being the only rheumatology-specific adherence measure,8,9 that has been recently validated for its use in Spanish RA population.12 It must also be stressed the special effort that was made when selecting centers to participate in the study, having into account geographical distribution and other variables, such as population density and public health expenditure for each region, in order to obtain a representative sample of Spain. Results obtained in this study can be extrapolated to the total Spanish population.

The adherence rate of 79% detected in this study is similar to rates observed in previous studies performed in Spanish population where an adherence of 79% was detected for patients being treated with oral antirheumatic drugs15 and 85% in the case of SC bDMARDs.16 Reported adherence rates in literature are highly variable, ranging from 30% to 80%.8 A systematic review of the literature estimated a 66% adherence to medication in patients with RA.17 Different definitions, methods, treatments and populations are behind this variability making it difficult to determine the magnitude of the problem. Optimal adherence depends on the type of drug and it remains to be determined in RA, if we understand optimal adherence as the relation between adherence level and disease flare. Independently of the definition of optimal adherence, we must be ambitious and try to ensure that patients follow treatment instructions rigorously. Nonetheless, adherence to treatment in RA patients is still suboptimal.8

The type of treatment did not determine the adherence rate in our study. No differences were detected in adherence among patients receiving bDMARDs versus cDMARDS and/or glucocorticoids. To our knowledge, this is the first study that studies the relationship between the type of therapy used to treat RA and adherence.

Our findings suggest that route of administration does not have an impact on adherence. This is in line with what has been shown in previous studies, where adherence and persistence rates appeared broadly similar for the different routes of drug administration in RA.18

The multivariate analyses determined that the number of drugs was an independent predictor of adherence. This fact is especially relevant, considering that treatment strategies in patients with RA rely heavily on the combined use of steroids, cDMARDs and bDMARDs. Contradictory results related to the impact of the number of medications on adherence have been reported.1922 Regimen complexity (multiple medications, multiple doses, specific dietary or time requirements) has been related to poorer adherence in chronic diseases.23 Less frequent dosing has been related to better compliance.5,16 Simpler, more convenient dosing regimens resulted in better compliance.24 Since using complex regimens requires a good communication between doctor and patient, patients have to understand the consequences of not following the instructions correctly. Prescription needs to be a shared decision process, in order to achieve a consensus. Patient empowerment may have a positive effect on adherence; however, highly empowered patients might believe that they can make treatment decisions. Intelligent non-adherence is becoming a common term. Patients have to be properly informed.

The second independent predictor of adherence in this study was the cohabitation status. Living alone has been previously associated with poor adherence.25,26 Social support seems to be an important factor contributing to proper compliance. Family support has been related to an improvement in adherence to bDMARD in RA.27

Duration of disease, civil status, education level, sex and age were not identified as predictors of adherence. These findings are in line with previous reports in which no evidence for any association with adherence was determined.14

The study has several limitations. The study design, being cross-sectional, has to be considered. Adherence is not stable over time, having a dynamic nature.8 To address this issue, the inclusion of patients was not determined by disease duration so that patients in different stages of disease were included (Table 2). Patients had to be treated for at least 3 months but no upper limit on treatment duration was established. Disease activity was not measured and this variable may have an impact on adherence. Study population was not selected based on disease activity, participants reflected real-life RA population treated in routine clinical practice. Another disadvantage is the use of a self-reported questionnaire to test for adherence, even though it is a highly specific method with a highly predictive value,12 this method is relatively insensitive, since patients may claim to be adherent to avoid caregiver disapproval.8 Despite this, the use of indirect methods as the one used in this study is a more simple and feasible way to evaluate adherence and due to its highly predictive value, it can be used as a screening instrument.12

Increased knowledge of the impact of therapeutic compliance on patients with RA, and the identification of possible predictors of adherence to treatment allows to develop strategies to favor adherence to treatments and avoid problems arising from lack thereof. In any case, prescription needs to be a shared decision process where clinicians and patients can discuss their concerns and expectations, in order to achieve a consensus that will favor adherence to treatment.

Alegre Sancho JJ, Almodovar Gonzalez R, Barbazan Alvarez C, Bernad Pineda M, Blanco Alonso R, Blanco Madrigal JM, Caliz Caliz R, Calvo Alen J, Calvo Catala J, Carrasco Cubero C, Castao Sanchez M, Chamizo Carmona E, De Toro Santos FJ, Delgado Beltran C, Eges Dubuc A, Escudero Contreras A, Fernandez Nebro A, Gamero Ruiz F, Garcia Aparicio A, Hernandez Cruz B, Guerra Vazquez JL, Hernandez Miguel MV, Lopez Lasanta M, Marenco de la Fuente JL, Muoz Fernandez Santiago, Navarro Blasco FJ, Nolla Sole JM, Ornilla Laraundogoitia E, Ortiz Garcia A, Pablos Alvarez JL, Perez Esteban S, Perez Garcia C, Roman Ivorra J, Romero Yuste S, Rosello Pardo R, Salvador Alarcon G, Tornero Molina J, Urruticoechea Arana A, Vela Casasempere P.

This study was financed by Roche Farma S.A. Spain.

Dr Manuel Pombo-Suarez reports grants from Roche Farma S.A. Spain, during the conduct of the study. The authors report no other conflicts of interest in this work.

1. Scott DL, Wolfe FHT. Rheumatoid Arthritis. Lancet. 2010;376:10941108.

2. Smolen JS, Landew R, Bijlsma J, et al. EULAR recommendations for the management of rheumatoid arthritis with synthetic and biological disease-modifying antirheumatic drugs: 2016 update. Ann Rheum Dis. 2017;76:960977. doi:10.1136/annrheumdis-2016-210715

3. Vrijens B, De Geest S, Hughes DA, et al. A new taxonomy for describing and defining adherence to medications. Br J Clin Pharmacol. 2012;73:691705. doi:10.1111/j.1365-2125.2012.04167.x

4. Shafrin J, Bognar K, Everson K, Brauer M, Lakdawalla DN, Forma FM. Does knowledge of patient non-compliance change prescribing behavior in the real world? A claims-based analysis of patients with serious mental illness. Clin Outcomes Res. 2018;Volume 10:573585. doi:10.2147/CEOR.S175877

5. De Klerk E, Van der Heijde D, Landew R, Van der Tempel H, Urquhart J, Van der Linden S. Patient compliance in rheumatoid arthritis, polymyalgia rheumatica, and gout. J Rheumatol. 2003.

6. Harrold LR, Andrade SE. Medication adherence of patients with selected rheumatic conditions: a systematic review of the literature. Semin Arthritis Rheum. 2009;38:396402. doi:10.1016/j.semarthrit.2008.01.011

7. Osterberg L, Blaschke T. Drug therapy: adherence to medication. N Engl J Med. 2005;353:487497. doi:10.1056/NEJMra050100

8. Van Den Bemt BJF, Zwikker HE, Van Den Ende CHM. Medication adherence in patients with rheumatoid arthritis: A critical appraisal of the existing literature. Expert Rev Clin Immunol. 2012;8:337351. doi:10.1586/eci.12.23

9. De Klerk E, Van Der Heijde D, Van Der Tempel H, Van Der Linden S. Development of a questionnaire to investigate patient compliance with antirheumatic drug therapy. J Rheumatol. 1999.

10. De Klerk E, Van Der Heijde D, Landew R, Van Der Tempel H, Van Der Linden S. The compliance-questionnaire-rheumatology compared with electronic medication event monitoring: a validation study. J Rheumatol. 2003.

11. Lee JY, Lee SY, Hahn HJ, Son IJ, Hahn SG, Lee EB. Cultural adaptation of a compliance questionnaire for patients with rheumatoid arthritis to a korean version. Korean J Intern Med. 2011;26:28. doi:10.3904/kjim.2011.26.1.28

12. Salgado E, Fernndez JRM, Vilas AS, Gmez-Reino JJ. Spanish transcultural adaptation and validation of the English version of the compliance questionnaire in rheumatology. Rheumatol Int. 2018. doi:10.1007/s00296-018-3930-7

13. Carmona L. The prevalence of rheumatoid arthritis in the general population of Spain. Rheumatology. 2002;41:8895. doi:10.1093/rheumatology/41.1.88

14. Pasma A, Vant Spijker A, Hazes JMW, Busschbach JJV, Luime JJ. Factors associated with adherence to pharmaceutical treatment for rheumatoid arthritis patients: A systematic review. Semin Arthritis Rheum. 2013;43:1828. doi:10.1016/j.semarthrit.2012.12.001

15. Marras C, Monteagudo I, Salvador G, et al. Identification of patients at risk of non-adherence to oral antirheumatic drugs in rheumatoid arthritis using the Compliance Questionnaire in Rheumatology: an ARCO sub-study. Rheumatol Int. 2017;37:11951202. doi:10.1007/s00296-017-3737-y

16. Calvo-Aln J, Monteagudo I, Salvador G, et al. Non-adherence to subcutaneous biological medication in patients with rheumatoid arthritis: A multicentre, non-interventional study. Clin Exp Rheumatol. 2017.

17. Scheiman-Elazary A, Duan L, Shourt C, et al. The rate of adherence to antiarthritis medications and associated factors among patients with rheumatoid arthritis: A systematic literature review and metaanalysis. J Rheumatol. 2016;43:512523. doi:10.3899/jrheum.141371

18. Fautrel B, Balsa A, Van Riel P, et al. Influence of route of administration/drug formulation and other factors on adherence to treatment in rheumatoid arthritis (pain related) and dyslipidemia (non-pain related). Curr Med Res Opin. 2017;33:12311246. doi:10.1080/03007995.2017.1313209

19. Treharne GJ, Lyons AC, Kitas GD. Medication adherence in rheumatoid arthritis: effects of psychosocial factors. Psychol Heal Med. 2004;9:337349. doi:10.1080/13548500410001721909

20. Van Den Bemt BJF, Van Den Hoogen FHJ, Benraad B, Hekster YA, Van Riel PLCM, Van Lankveld W. Adherence rates and associations with nonadherence in patients with rheumatoid arthritis using disease modifying antirheumatic drugs. J Rheumatol. 2009;36:21642170. doi:10.3899/jrheum.081204

21. Park DC, Hertzog C, Leventhal H, et al. Medication adherence in rheumatoid arthritis patients: older is wiser. J Am Geriatr Soc. 1999;47:172183. doi:10.1111/j.1532-5415.1999.tb04575.x

22. Kristensen LE, Saxne T, Nilsson J, Geborek P. Impact of concomitant DMARD therapy on adherence to treatment with etanercept and infliximab in rheumatoid arthritis. Results from a six-year observational study in southern Sweden. Arthritis Res Ther. 2006;54:600606. doi:10.1186/ar2084

23. Ingersoll KS, Cohen J. The impact of medication regimen factors on adherence to chronic treatment: A review of literature. J Behav Med. 2008;31:213224. doi:10.1007/s10865-007-9147-y

24. Claxton AJ, Cramer J, Pierce C. A systematic review of the associations between dose regimens and medication compliance. Clin Ther. 2001;23:12961310. doi:10.1016/S0149-2918(01)80109-0

25. Lorish CD, Richards B, Brown S. Missed medication doses in rheumatic arthritis patients: intentional and unintentional reasons. Arthritis Care Res. 1989;2:39. doi:10.1002/anr.1790020103

26. De Cuyper E, De Gucht V, Maes S, Van Camp Y, De Clerck LS. Determinants of methotrexate adherence in rheumatoid arthritis patients. Clin Rheumatol. 2016;35:13351339. doi:10.1007/s10067-016-3182-4

27. Morgan C, McBeth J, Cordingley L, et al. The influence of behavioural and psychological factors on medication adherence over time in rheumatoid arthritis patients: A study in the biologics era. Rheumatol. 2015. doi:10.1093/rheumatology/kev105

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[Full text] Adherence to Treatment in Patients with Rheumatoid Arthritis from Spai | PPA - Dove Medical Press

Introduction:Region-specific health-related quality of life (HRQoL) scores or utility values are representative and pivotal for economic evaluations as they are influenced by the value judgment of the local population. This study systematically reviewed and pooled EuroQoL-5 Dimension (EQ-5D) utility scores of rheumatoid arthritis (RA) across primary studies from Asia.

Methods:Studies reporting EQ-5D utility scores among adult RA patients from Asian countries were systematically searched in PubMed-Medline, Scopus and Embase since inception through February 2020. Selected studies were systematically reviewed and study quality assessment was performed. Meta-analysis was performed using a random-effect model with subgroup and meta-regression analysis to explore heterogeneity.

Results:Among 1391 searched articles, 37 studies with 31 983 participants were systematically reviewed and meta-analysis was conducted among 31 studies. The pooled EQ-5D scores and EQ-5D visual analog score were 0.66 (95% CI 0.63-0.69, I2= 99.65%) and 61.21 (50.73-71.69, I2= 99.56%) respectively with high heterogeneity. For RA patients with no, low, moderate and high disease activity based on Disease Activity Score (DAS)-28, the pooled EQ-5D scores were 0.78 (0.65-0.90), 0.73 (0.65-0.80), 0.53 (0.32- 0.74), and 0.47 (0.32-0.62), respectively. On meta-regression, age of patients (P < .05) was positively associated and use of glucocorticoids (P < .05) was inversely associated with utility values.

Conclusion:Lower EQ-5D scores were associated with severe disease activity, increasing age and female gender among RA patients. The study provides pooled EQ-5D scores for RA patients that are useful inputs for cost-utility studies in Asia.

Keywords:EQ-5D-3L health utility; EQ-5D-5L; rheumatoid arthritis.

Link:
Health-related quality of life in rheumatoid arthritis: Systematic review and meta-analysis of EuroQoL (EQ-5D) utility scores from Asia - DocWire News

Among patients with newly diagnosed rheumatoid arthritis (RA), Disease Activity Score 28-joint count (DAS28) characterized using C-reactive protein (CRP) values are lower than the corresponding score using erythrocyte sedimentation rate (ESR) values, both at baseline high disease activity and post-treatment, according to study results published in ACR Open Rheumatology.

Disease activity scores are used to guide treatment and determine the efficacy of therapeutic strategies. Previous studies have shown that DAS28-CRP values are lower than DAS28-CRP values, but current guidelines do not provide specific cutoffs for high disease activity for each of the scores.

Existing studies that compared DAS28-CRP and DAS28-ESR used data from patients who received immunosuppressive therapy. The objective of the current study was to compare the scores from immunosuppressive treatment-nave patients.

The retrospective electronic chart review included 171 immunosuppressive treatment-nave patients with newly diagnosed RA. DAS28-CRP and DAS28-ESR were compared according to the cutoff value for baseline high disease activity (>5.1). A receiver operator characteristic curve (ROC) and Youden index were used to calculate the DAS28-CRP high disease activity optimal cut-point value corresponding to DAS28-ESR >5.1.

At baseline, the mean DAS28-ESR was higher than the mean DAS-28 CRP (5.1 1.2 vs. 4.1 1.0; P <.001) and more patients met high disease activity criteria for DAS28-ESR than for DAS28-CRP (48.5% vs. 14.6%, respectively). ROC curve and Youden index analysis showed that the cutoff point estimation of high disease activity using DAS28-ESR >5.1 corresponded to a DAS28-CRP score <4.06 (area under the ROC curve = 0.93, P =.000).

Data on both DAS28-ESR and DAS28-CRP score following treatment were available for 151 patients. On average, DAS28-CRP values were 0.66 points higher than the corresponding DAS28-ESR. DAS28-CRP values were significantly lower compared with DAS28-ESR in all subgroups classified by gender, age, and disease severity.

In patients in remission (values <2.6), mean DAS28-CRP values were 0.36 points lower than the corresponding DAS28-ESR value (1.45 vs. 1.81, respectively).

The study had several limitations, including the racially homogeneous cohort (91.8% white), single center study, as well as lack of data on body mass index or comorbidities which may have a significant impact on the difference between DAS28-ESR and DAS28-CRP.

There is a difference between DAS28-ESR and DAS28-CRP, even when calculated for immunosuppressive treatmentnave patients. DAS28-CRP is significantly lower than DAS28-ESR, wrote the researchers.

Greenmyer JR, Stacy JM, Sahmoun AE, Beal JR, Diri E. DAS28-CRP cutoffs for high disease activity and remission are lower than DAS28-ESR in rheumatoid arthritis. ACR Open Rheumatol. 2020;2(9):507-511. doi:10.1002/acr2.11171

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Comparing DAS28-ESR and DAS28-CRP in Patients with Rheumatoid Arthritis - Rheumatology Advisor

Gilead Sciences rheumatoid arthritis (RA) treatment Jyseleca has been given a recommendation from the UKs National Institute of Health and Care Excellence (NICE).

Jyseleca (filgotinib) is an oral JAK inhibitor that can be administered as a monotherapy or used alongside another another common RA medicine called methotrexate.

Patients with moderate-to-severe RA will now be able to access the drug on the NHS in England, if they have responded inadequately to previous intensive therapy with two or more disease-modifying anti-rheumatic drugs (DMARDs).

This is a landmark decision from NICE and represents a pivotal moment for the treatment of RA, said James Galloway, consultant rheumatologist at Kings College London Hospital.

While no single medicine works for everyone, the addition of Jyseleca is an important step forward that we believe will help more patients achieve remission, even when their disease is at a less advanced stage, he added.

In phase 3 trials, Jyseleca was shown to be effective in reducing the symptoms of RA, including joint tenderness and swelling.

In one of these studies FINCH 1 the JAK inhibitor was compared to AbbVies TNF antibody Humira(adalimumab) or placebo given on top of methotrexate in patients with moderate-to-severe RA who werent responding to methotrexate alone.

Daily oral dosing with Jyseleca was significantly better than placebo in achieving a 20% improvement in symptoms (an ACR 20 response), the primary endpoint in the study, and matched the efficacy of Humira which is given by injection.

FINCH 3 compared Jyseleca alone or in combination with methotrexate as a front-line therapy for patients with moderate-to-severe RA. In this study, the combination was significantly better than methotrexate alone at helping patients achieve an ACR20 response.

In addition, Jyseleca monotherapy was as effective as methotrexate on the ACR20 measure, but was significantly better on the 50% improvement (ACR50) and 70% improvement (ACR70) scales.

In August 2020, the US Food and Drug Administration (FDA) rejected Gileads Jyseleca, handing the company a complete response letter (CRL).

In this CRL, the FDA asked for data from two ongoing clinical trials MANTA and MANTA-Ray investigating the effect of the 200mg dose of Jyseleca on sperm concentrations.

The FDA has expressed concerns regarding the overall benefit/risk profile of the filgotinib 200 mg dose, Gilead added in a statement.

Topline results from the MANTA and MANTA-Ray studies are expected in the first half of 2021, at which point Gilead is likely to refile Jyseleca with the FDA.

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Gilead's Jyseleca is given a NICE recommendation for rheumatoid arthritis - PMLiVE

Background:Although targeted biological treatments have transformed the outlook for patients with rheumatoid arthritis, 40% of patients show poor clinical response, which is mechanistically still unexplained. Because more than 50% of patients with rheumatoid arthritis have low or absent CD20 B cells-the target for rituximab-in the main disease tissue (joint synovium), we hypothesised that, in these patients, the IL-6 receptor inhibitor tocilizumab would be more effective. The aim of this trial was to compare the effect of tocilizumab with rituximab in patients with rheumatoid arthritis who had an inadequate response to anti-tumour necrosis factor (TNF) stratified for synovial B-cell status.

Methods:This study was a 48-week, biopsy-driven, multicentre, open-label, phase 4 randomised controlled trial (rituximab vs tocilizumab in anti-TNF inadequate responder patients with rheumatoid arthritis; R4RA) done in 19 centres across five European countries (the UK, Belgium, Italy, Portugal, and Spain). Patients aged 18 years or older who fulfilled the 2010 American College of Rheumatology and European League Against Rheumatism classification criteria for rheumatoid arthritis and were eligible for treatment with rituximab therapy according to UK National Institute for Health and Care Excellence guidelines were eligible for inclusion in the trial. To inform balanced stratification, following a baseline synovial biopsy, patients were classified histologically as B-cell poor or rich. Patients were then randomly assigned (1:1) centrally in block sizes of six and four to receive two 1000 mg rituximab infusions at an interval of 2 weeks (rituximab group) or 8 mg/kg tocilizumab infusions at 4-week intervals (tocilizumab group). To enhance the accuracy of the stratification of B-cell poor and B-cell rich patients, baseline synovial biopsies from all participants were subjected to RNA sequencing and reclassified by B-cell molecular signature. The study was powered to test the superiority of tocilizumab over rituximab in the B-cell poor population at 16 weeks. The primary endpoint was defined as a 50% improvement in Clinical Disease Activity Index (CDAI50%) from baseline. The trial is registered on the ISRCTN database, ISRCTN97443826, and EudraCT, 2012-002535-28.

Findings:Between Feb 28, 2013, and Jan 17, 2019, 164 patients were classified histologically and were randomly assigned to the rituximab group (83 [51%]) or the tocilizumab group (81 [49%]). In patients histologically classified as B-cell poor, there was no statistically significant difference in CDAI50% between the rituximab group (17 [45%] of 38 patients) and the tocilizumab group (23 [56%] of 41 patients; difference 11% [95% CI -11 to 33], p=031). However, in the synovial biopsies classified as B-cell poor with RNA sequencing the tocilizumab group had a significantly higher response rate compared with the rituximab group for CDAI50% (rituximab group 12 [36%] of 33 patients vs tocilizumab group 20 [63%] of 32 patients; difference 26% [2 to 50], p=0035). Occurrence of adverse events (rituximab group 76 [70%] of 108 patients vs tocilizumab group 94 [80%] of 117 patients; difference 10% [-1 to 21) and serious adverse events (rituximab group 8 [7%] of 108 vs tocilizumab group 12 [10%] of 117; difference 3% [-5 to 10]) were not significantly different between treatment groups.

Interpretation:The results suggest that RNA sequencing-based stratification of rheumatoid arthritis synovial tissue showed stronger associations with clinical responses compared with histopathological classification. Additionally, for patients with low or absent B-cell lineage expression signature in synovial tissue tocilizumab is more effective than rituximab. Replication of the results and validation of the RNA sequencing-based classification in independent cohorts is required before making treatment recommendations for clinical practice.

Funding:Efficacy and Mechanism Evaluation programme from the UK National Institute for Health Research.

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Rituximab versus tocilizumab in anti-TNF inadequate responder patients with rheumatoid arthritis (R4RA): 16-week outcomes of a stratified,...

PIPis designed to aid people with extra costs associated with long-term health conditions, or disabilities. The sum is set at a weekly rate, and people will be able to obtain varying amounts dependent on how their condition affects them. PIP is tax-free, though, and Britons can receive the sum whether they are in work or out of work.

PIP as a payment from the Department for Work and Pensions (DWP), could therefore provide assistance to those with arthritis or a range of other conditions.

Arthritis is common within the UK, and is a condition which causes pain and inflammation in the joints.

The NHS estimates more than 10 million people currently have arthritis or other similar joint conditions, across the country.

Of these, then, many could stand to benefit from a PIP payments to assist them with their day-to-day lives.

READ MORE:Cold Weather Payment: DWP triggers new postcodes - 25 payout due

Those who are over state pension age who wish their PIP to continue, are urged to renew their claim when their current award draws to a close.

A PIP payment, though, is not dependent on the condition a person has, rather how it affects them on a day-to-day basis.

The sum is comprised of two parts - the daily living part and the mobility part.

Whether a person receives one or both of these payments and how much they will ultimately receive will depend on the severity of their condition.

However, to receive PIP there is a claims process one will have to go through with the DWP.

People who think they may be eligible for a payment are encouraged to reach out to the DWP via phone.

Alternatively, individuals may be able to get a PIP claim form sent to them by post, where they will be required to fill out details about their condition and how it affects them.

Individuals will need certain information to hand, including:

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PIP: Britons could claim up to 151 a week for arthritis or other conditions - Express

Sure, the cat never really moved around all that much in the first place. But now kitty stays in one spot for long periods of time and looks stiff when she does eventually move.

The signs point to arthritis, says the German Association for Animal Health. Changes in behaviour can be a first hint.

If you think your feline friend has arthritis, its time for a trip to the vet, who can prescribe a suitable pain medication if it is indeed the case; unfortunately, there is no cure.

Owners can do a bit to make life for their kitty a bit more comfortable: A bigger litter box with a low entrance can help, as can making the path to its favourite spots more accessible.

If possible, the cat should be encouraged to carefully keep active, and owners need to keep an eye on their kittys weight: Fewer kilos mean less stress on the joints. Theres also the option of supporting joint metabolism with a special diet. dpa

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Cats can get arthritis and here are the first signs to look out for - The Star Online

Photo: Contributed

The Spotlight series is a series of articles looking at common, and preventable, diseases.

I explain the science behind the condition, how to spot early signs and what you can do to prevent it.

The Science

Arthritis refers to a multitude of conditions that cause inflammation and pain in the joints. Arthritis can be split into osteoarthritis and inflammatory arthritis, such as rheumatoid arthritis.

One in five Canadians live with arthritis; it is common in older age, although young people can also suffer from it.

Osteoarthritis is the most common form of arthritis, and also the most preventable. It is caused by joint damage that occurs over time with aging, or due to injury.

Osteoarthritis causes a loss of cartilage, which is the material that covers and protects the bones.

Without cartilage, the bones grind against one another, causing pain, swelling and stiffness. The joints most commonly affected by osteoarthritis are the knees, hips, hands and spine.

Inflammatory arthritis is a collective term for all the other types of arthritis.

Common examples are:

Inflammatory arthritis not only affects the joints, but also other systems in the body. They are caused by autoimmune disorders, where the bodys immune system attacks the tissue in and around the joint.

This causes pain, stiffness and swelling, as well as systemic symptoms like fevers, weight loss and fatigue.

Signs and Symptoms

All types of arthritis cause pain, stiffness and swelling of one or more joints. The symptoms can change during the day, and also with exercise and rest. Typically, cold weather also worsens symptoms.

Eventually, this can lead to reduced mobility.

How to Prevent Arthritis

Many factors affecting your risk of arthritis cannot be controlled, such as your genetics and gender. However, some factors can be prevented.

If you have a strong family history of arthritis, its worth going to your family doctor to discuss your risk. Be mindful of the signs and symptoms, as getting treatment early can make a big difference.

In terms of preventing arthritis, the most important factor is maintaining a healthy weight. Being overweight puts excess force through your joints with each step you take, increasing the wear and tear on the joint and ultimately causing long term damage.

Maintaining a healthy weight for your height is crucial in preventing arthritis.

As well as eating healthily to maintain a good weight, getting regular exercise is ideal. The best form of exercise for preventing arthritis is a mix of cardiovascular and strength training; for instance, try alternating swimming or cycling with weight training.

This strengthens your body without putting too much stress on any one joint.

As well as the injury from excess weight, other injuries to your joints can increase your risk of arthritis.

Be careful when exercising and playing sports, and remember to always warm up and cool down to reduce your risk of an injury. If appropriate, consider joint supports if you do have an existing injury.

You can also reduce your risk of injury by being careful lifting heavy objects, sitting in an ergonomic position if you work at a desk and using a backpack to carry heavy items, rather than carrying items on one arm.

If you are concerned about an existing injury or the possibility of one, speak to your family doctor or physiotherapist.

Take Home Message

Although some factors are out of your control, there is plenty that you can do to reduce your risk of arthritis.

It can be a debilitating disease, so getting plenty of exercise, eating well and maintaining a healthy weight are ways that you can significantly reduce your risk of the disease.

Be mindful of any existing injuries, and look after your joints to prevent any new ones.

Link:
Spotlight on: Arthritis - Health and Happiness - Castanet.net

Press Release

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

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

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

25 January 2021

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

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

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

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

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

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

-Ends-

Contacts

About 4BIO Capital

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

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

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

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

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

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

Guidance Issuing Office

Center for Biologics Evaluation and Research

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

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

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

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

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

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

01/19/2021

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

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

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

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

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

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

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

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

AboutNANOSCOPE THERAPEUTICS Inc.

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

Contact:

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

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SOURCE Nanoscope Therapeutics

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

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

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

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

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

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

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

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

Katherine High, president of therapeutics at AskBio

Permission granted by AskBio

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Permission granted by AskBio

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

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

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

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

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

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

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

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

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

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

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

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

Listen to the full podcast to hear more details. And dont forget to subscribe on iTunes, Spotify, or Google Podcasts.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

This will address an accelerating gene therapy market that is expected to grow globally by 16.6 percent from 2020-2027.

"The Gene Therapy Ready network demonstrates our commitment to empowering sites and supporting our industry partners as they pursue advanced genetic engineering to find cures for the world's most pressing health conditions," said Scott Uebele, President and Chief Research Services Officer at Advarra. "Our commitment to efficient study activation is unwavering, and this is another example of how Advarra bringslife sciences companies,CROs, research sites, investigators,andacademiatogether at the intersection of safety,compliance,technology, and collaboration."

All Gene Therapy Ready sites stand ready to help industry sponsors conduct clinical trials that advance cures, develop vaccines, and find treatments for rare disease. By placing clinical trials with a Gene Therapy Ready site, research sponsors can save significant time during study startup.

"This innovative network is truly the first of its kind. We constantly look for ways to support our sponsors in rapidly starting trials in a safe, compliant, and quality manner. With the Gene Therapy Ready network, we can improve study startup times by a month or more, potentially placing cures in the hands of patients faster," said James Riddle, Vice President of Research Services and Strategic Consulting at Advarra. "The Gene Therapy Ready site network charts a course to success by providing our sponsor clients with a clear choice for IBC review services."

About Advarra

Advarra advances the way clinical research is conducted: bringing life sciences companies, CROs, research sites, investigators, and academia together at the intersection of safety, technology, and collaboration. With trusted IRB and IBC review solutions, innovative technologies, experienced consultants, and deep-seated connections across the industry, Advarra provides integrated solutions that safeguard trial participants, empower clinical sites, ensure compliance, and optimize research performance. Advarra is advancing clinical trials to make them safer, smarter, and faster. For more information, visit advarra.com.

SOURCE Advarra

https://www.advarra.com

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Advarra Announces New Gene Therapy Ready Site Network - PRNewswire

However, the partners were not willing to disclose, as of today, which diseases exactly are being targeted under this alliance.

The collaborative project combines Neurogenes manufacturing and drug development capabilities with the University of Edinburghs novel platform and neurodevelopmental disease expertise.

Under the terms of the collaboration, the US company will provide financial support for Dr Stuart Cobbs laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms.Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

Dr Cobbs lab uses a broad range of technologies to develop novel treatments for neurodevelopmental disorders based on a deep understanding of the molecular pathology.

In addition to Dr Cobbs position at the university, where he is a Simons fellow and reader in neuroscience, he is also Neurogenes chief scientific officer (CSO).

Neurogenes lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Its product pipeline of gene therapy candidates addresses distinct monogenic neurological diseases.

Neurogene is trying to find treatments for, among others, Batten disease - a group of rare, inherited diseases of the nervous system also called neuronal ceroid lipofuscinoses (NCLs).The company is focusing on CLN5 and CLN7, two rare, late infantile and rapidly progressive subtypes of Batten disease.Children with CLN5 or CLN7 typically develop signs and symptoms of the diseases at a young age, including seizures, progressive deterioration in intellectual and motor capabilities, and loss of vision.CLN5 is caused by a variant in the CLN5 gene, which leads to disruption of normal CLN5 protein function. The CLN7 subtype of Batten disease is caused by a variant in the CLN7 gene, also called the MFSD8 gene, which leads to disruption of normal CLN7 protein function.

Another disorder Neurogene is targeting is Charcot-Marie-Tooth disease (CMT) a group of inherited diseases that affect the peripheral nervous system (PNS). CMTs are the most common inherited motor and sensory nerve disorders - neuropathies.

It is also working to determine and address the root cause of diseases such as aspartylglucosaminuria (AGU) a rare, neurodegenerative lysosomal storage disorder (LSD).

In December 2020, Neurogene announced the completion of a US$115m Series B financing, which was led by EcoR1 Capital, with participation from existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an undisclosed leading healthcare investment fund.

New investors included funds and accounts managed by BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, and Alexandria Venture Investments.

The company said proceeds from the financing would be used to advance Neurogenes portfolio of multiple gene therapy programs into the clinic, as well as accelerate investment in novel gene therapy product designs and Neurogenes technology platform addressing key limitations in conventional gene therapy, while building out its AAV vector GMP manufacturing capabilities.

Oleg Nodelman, portfolio manager, EcoR1 Capital, said then: Neurogene is establishing itself as a leader in the gene therapy arena for neurological diseases. We are impressed by the companys innovation and accomplishments to date and are pleased to provide our support to Neurogene to advance medical research in this rapidly evolving area.

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Neurogene in tie up with university to advance gene therapy technologies - BioPharma-Reporter.com

LabCorps contract research organization business Covance has promoted Maryland Franklin, Ph.D., to vice president and head of its cell and gene therapy unit.

She moves up from being Covances site lead and executive director of scientific development at the Ann Arbor, Michigan, facility, which focuses on preclinical oncology.

Now, she steps up to run its cell and gene therapy business, a major element in any CRO's portfolio these days as more and more biopharmas look to tap the therapies for potentially curative treatments for a range of diseases.

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It remains a tricky proposition to pull off, but cell and gene therapy are very much the current course for R&D across the life sciences as well as a major part of Covances business. Under her new role, Franklin will oversee these offerings.

These solutions aim to help sponsors reduce risk, transition programs within and between phases of development faster and create a more patient-centric experience, Covance said in a statement, as Franklin will also be tapped to further extend Covance by Labcorps industry leading position.

RELATED: Covance to 'transform' into a decentralized CRO

We are thrilled to welcome Dr. Franklin to Covance by Labcorp. Her experience and expertise will bring perspective and insight to cell and gene therapy at Covance, said Bill Hanlon, Ph.D., president of clinical, therapeutic and regulatory sciences for Covance.

Dr. Franklin joins us at a critical juncture in our ability to support sponsors needs throughout the drug development process. She will guide our highly experienced scientists across functional disciplines to seamlessly develop and commercialize a cell or gene therapy. With Dr. Franklins expertise, we hope to further grow and advance our cell and gene therapy programs.

Cell and gene therapy approaches continue to show great promise in treating a variety of diseases that range from extremely debilitating rare diseases to applications in oncology, added Franklin. With several approved advanced therapies to date and many, many more in development, Im excited to join Covance by Labcorp to and help sponsors in their mission to improve the lives of patients.

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Covance boosts Franklin to lead its cell and gene therapy unit - FierceBiotech

New York, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Cancer Gene Therapy Market 2021-2025" - https://www.reportlinker.com/p05060878/?utm_source=GNW Our report on cancer gene therapy market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the side effects of traditional cancer treatments, benefits associated with gene therapy for cancer treatment and the rising prevalence rate of cancer boosting the demand for cancer therapeutics. In addition, the side effects of traditional cancer treatments is anticipated to boost the growth of the market as well. The cancer gene therapy market analysis includes application segments and geographical landscapes.

The cancer gene therapy market is segmented as below: By Application Oncolytic virotherapy Gene transfer Gene-induced immunotherapy

By Geographical Landscapes North America Europe Asia ROW

This study identifies the rising partnerships and collaborations as one of the prime reasons driving the cancer gene therapy market growth during the next few years. Also, favorable government regulations for gene therapy programs and rapid growth potential in developing economies will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our report on cancer gene therapy market covers the following areas: Cancer gene therapy market sizing Cancer gene therapy market forecast Cancer gene therapy market industry analysis

Read the full report: https://www.reportlinker.com/p05060878/?utm_source=GNW

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

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The Global Cancer Gene Therapy Market is expected to grow by $ 2.96 bn during 2021-2025 progressing at a CAGR of 20% during the forecast period -...

NEW YORK--(BUSINESS WIRE)--Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, and University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases, today announced a research collaboration to advance development of multiple platform approaches to enable next generation gene therapies.

The collaboration provides comprehensive research capabilities to Neurogene, enabling the company and the University to expedite a multiple platform approach to improve upon existing gene therapy technologies. Under the terms of the collaboration, Neurogene will provide financial support for Dr. Stuart Cobbs laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms. Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

This partnership provides Neurogene with preeminent neurological research expertise and capabilities. Dr. Cobbs lab has contributed significant scientific expertise to improve the quality of our current rare disease pipeline and generated promising early data to allow us to tackle complex neurological diseases not addressable with conventional gene therapy, said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. With this collaboration, I look forward to advancing our mission to provide safe and effective genetic therapies to patients and families as quickly as possible.

Neurogene is a science-driven company committed to investing in innovation and is the right partner for us to build upon the early successes in gene therapy technology, Stuart Cobb, Ph.D., Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, University of Edinburgh, stated. We are excited to collaborate with Neurogene on the critically-important endeavor of improving upon current gene therapy technologies. Gene therapy is a very promising yet complex development area, and we are privileged to help address the unmet needs that exist within rare neurological diseases.

In addition to Dr. Cobbs position at the University of Edinburgh, he serves as Chief Scientific Officer of Neurogene.

This Collaboration has been supported by Edinburgh Innovations, the University of Edinburghs commercialization service.

About Neurogene Inc.

Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options currently exist. Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address. For more information, visit http://www.neurogene.com.

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Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies - Business Wire

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease, said RA Session II, President, Founder and CEO of Taysha. We are encouraged by the early evidence of TSHA-105s disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available to patients as expeditiously as possible.

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. This rare form of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life.

We are pleased that the FDA recognizes TSHA-105s potential as an innovative therapeutic option for SLC13A5 deficiency, said Rachel Bailey, Ph.D., Assistant Professor in Pediatric Neurology at UT Southwestern. This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life.

As a mother of two children with SLC13A5 deficiency, I have witnessed firsthand the devastating impact that numerous seizures and comorbidities accompanying the disease has on those affected by this disease, said Kim Nye, Founder of TESS Research Foundation. Tayshas commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

About Taysha Gene Therapies

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-105, 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 benefits of rare pediatric disease designation and orphan drug designation to 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. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5...