Category Archives: Gene therapy

Emily Kaiser [EK]: Hello, welcome. I'm Emily Kaiser, editor of Modern Retina. I'm sitting down with Dr. Rahul Khurana to discuss the Aaviate study. Dr. Khurana, can you tell us a little bit about the Aaviate data presented at the Angiogenesis meeting?

Rahul Khurana, MD [RK]: For sure, Emily.

Aaviate is a really exciting clinical trial that I've been involved with and at Angiogenesis this year, we presented the updated data on the first two cohorts. And so to give everyone kind of a background, obviously, gene therapy is a very exciting area of interest right now.

We know there's a lot of unmet needs in our treatments of macular degeneration. You know, these treatments often are very effective, but they require a lot of treatments. And there's a high burden with our current set of treatments out there, and gene therapy offers the potential for a one-time treatment to give us long-term, anti-VEGF suppression and really a long-term answer to this kind of chronic disease.

And so Aaviate is along with many gene therapy programs, or studies, that are looking to kind of tackle this. And the thing which is interesting about Aaviate is that most of gene therapy has really looked at it the traditional approaches. Either theyve gone intravitreal, which is something we're very used to because we do these injections, or done a subretinal gene therapy delivery, which requires surgery, which is much more invasive. And Aaviate utilizes a suprachoroidal approach.

And the benefit of this is that we get good drug delivery of inhibiting anti-VEGF gene therapy, but by delivering it in the office. And one of the benefits of suprachoroidal delivery over intravitreal delivery is that in intravitreal, there is a potential for a lot of exposure of the medicine to not just the back of the eye, but also the front of the eye. And we've seen in some early gene therapy programs, a lot of complications involving inflammation and hypotony.

In a suprachoroidal approach, you can get a very high concentration to the retina with very low concentration to the anterior segment.

EK: That's really interesting. Has anything developed since the presentation?

RK: the study has been ongoing. So what I presented at Angiogenesis was the first two cohorts in the sense that those patients had been fully enrolled, and we had up to date up to six months. So that was really exciting. And we'll kind of delve into some of the details there. But there's still cohorts three, four, and five, which are now fully enrolled. So since that time, we've now fully enrolled those patients. And we're basically waiting to hear back on updates, or we were waiting to hear back those results. And we need to once they're fully enrolled, we need to have the subsequent time to see how these patients do.

EK: And what are the next steps?

RK: Part of the next steps for gene therapy is really to finish up the clinical study. The patients are all enrolled, which is wonderful. Now we want to see how they did in these higher enrolling cohorts. So one thing that we haven't talked about is what were the results that we actually found from the first two cohorts? And so as I mentioned before, Aaviate takes patients who have been previously treated so these patients who were in the study were basically patients who needed to get multiple injections.

On average, they average nearly nine injections in the previous year, which is about an injection every five weeks. And we took those patients who basically needed regular anti-VEGF therapy, and we basically offered them a super coronal injection of RGX-314, which involves a novel Aaviate vector, which encodes for an anti VEGF monoclonal antibody fragment, which is transduced, or basically transvexed, the patient's own retinal cells to produce anti-VEGF protein to effectively give you long term suppression.

And the data showed that in the first two cohorts where this was done, not only was the treatment quite safe, there was a very low rate of inflammation and no serious adverse events. But more impressively, that the number of treatments had gone down dramatically. The patients in the study were able to maintain their visual acuity, which was wonderful to see. But more importantly, the number of injections went down significantly.

As I told you, before, most of these patients needed about an injection every five weeks, and in the study, the number of injections went down nearly 70 to 79% than they had before receiving the gene therapy, they were able to maintain the visual acuity, maintain the retinal anatomy, and the number of patients who didn't even need injections was nearly 30% in the first cohort, and nearly 40% in the second cohort. And that was quite exciting because this truly was kind of delivering on the promise of a once-and-done therapy. But as I said before, we really need the long-term data to kind of see how this translates and also we need to see how higher doses if we can get better efficacy and also maintain a very good safety profile.

EK: Wow. So what does this mean for clinicians and for patients?

RK: I think it offers a really exciting hope for both our patients and physicians. As we mentioned before, we have a lot of treatment options for anti-VEGF therapy and they do work very well. The problem is that they require a lot of treatments and there's a high treatment burden, and this is challenging for patients because not all patients can come back in there's a high rate of lost to followup, non-compliance, and non-adherence to the treatment regimens. And we've seen in Phase 3 clinical studies, especially in follow up in real world practice that when patients are not getting regular treatments, they lose vision. And that's why we've it's been hard to replicate the excellent results we've seen in the Phase 3 studies in real world practice. And the hope is that if one of these gene therapy treatments can work, we can offer a really one-and-done or a much more sustainable treatment therapy for our patients, which ultimately lead to better compliance and better visual outcomes.

EK: Fantastic. Well, thank you so much for the update.

RK: My pleasure. Thanks for having me.

Note: This transcript has been lightly edited for clarity.

Original post:
AAVIATE: Gene therapy via suprachoroidal drug delivery may lower treatment burden for patients with AMD - Modern Retina

FLT180a generated protective FIX levels with no bleeding or need forFIX replacement

The treatmentwaswelltolerated witha good safety profile

LONDON, July 10, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced the presentation of safety and initial efficacy data from the first cohort of the Phase 1/2 dose-confirmation B-LIEVEtrial for FLT180a, the companys AAVS3-based gene therapy candidate for people with hemophiliaB, at the International Society on Thrombosis and Haemostasis (ISTH) Congress being held in London, July 9-13, 2022.

As of the data cut-off of May 23, 2022, a one-time FLT180a dose of 7.7e11 vg/kg generated a rapid increase of coagulation factor IX (FIX), reaching levels in the normal range (93, 92 and 80 IU/dL) for the three patients in cohort 1 through days 77, 56 and 36, respectively. Patients stopped FIX prophylaxis and did not require FIX replacement or experience bleeding following treatment with FLT180a.

The treatment and the prophylactic immune management regimen were well tolerated. No serious adverse events or infusion reactions were observed, and there has been no evidence of FIX inhibitors. All adverse events (AEs) were mild, and most were transient. AEs related to immune management were consistent with the known profiles of corticosteroids and tacrolimus.

Dosing of cohort two was completed in June, with early results showing a similar initial response to FLT180a. Based on the data from cohort one and consistent with the advice of the independent Data Monitoring Committee of the B-LIEVE trial, patients in cohort two received the same dose of FLT180a and prophylactic immune management regimen that were used in the first cohort.

As the data continue to evolve since the data cut-off for cohort one, two patients have experienced a decrease in FIX expression together with a mild and transient increase in liver enzymes. All patients continue to have expression levels above baseline, and no patient has experienced a bleed or required FIX supplementation.

The initial data show that FLT180a provides rapid and consistent elevations in FIX to normal levels, which can prevent bleeding and the need for regular FIX replacement in people with hemophilia B, said Pamela Foulds, MD, Chief Medical Officer of Freeline. Emerging data suggest that while FIX expression was maintained at protective levels, a further refined immune management regimen may be required to avoid mild and transient transaminitis and to sustain FIX levels in the normal range. Potential adjustments in cohort two and forthcoming results from that cohort will help us interpret this further.

Our strategy is to advance gene therapy programs that have the potential to deliver best-in-class or first-in-class treatments, said Michael Parini, Chief Executive Officer of Freeline. While we continue to believe FLT180a has the potential to deliver a best-in-class gene therapy for people with hemophilia B, the availability of other treatment options and the need to prioritize our valuable resources dictate that we evaluate strategic options for FLT180a. These include, but are not limited to, seeking a partner that would enable the continuation of FLT180a through Phase 3 development.

With two other promising programs in Fabry disease and Gaucher disease in the clinic, each with the potential to be first-in-class gene therapy treatments for these debilitating diseases, we will continue to focus our attention and resources on those programs that offer the highest value for patients and Freelines shareholders, said Parini.

The poster #PB0213 entitled Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients with Hemophilia B by Guy Young et al. will be presented from 6:30pm-7:30pm BST today and will be made available on the Investors section of Freelines website.

About the B-LIEVE Dose-Confirmation Trial

B-LIEVE is a Phase 1/2 dose confirmation trial of FLT180a using a short course of prophylactic immune management with the goal of normalizing FIX levels in patients with severe and moderately severe hemophilia B. The starting dose of 7.7e11 vg/kg was selected based on the results of the Phase 1/2 B-AMAZE dose-finding trial and modeling. The goal of the B-LIEVE trial is to finalize a dose for a pivotal Phase 3 trial that enables predictable and sustained factor IX (FIX) expression at protective levels.

About FLT180a for People with Hemophilia B

Freelines FLT180a candidate uses a potent AAVS3 capsid rationally designed for effective targeting and transduction of liver cells and containing an expression cassette encoding a gain of function Padua variant of human factor IX (FIX). FLT180a has been studied in B-AMAZE, a Phase 1/2 dose-finding trial with the goal of normalizing FIX activity in patients with moderately severe and severe hemophilia B. Patients treated in B-AMAZE are being followed in a long-term follow-up study. A Phase 1/2 dose-confirmation trial of FLT180a called B-LIEVE to finalize a dose for a Phase 3 pivotal trial is in progress.

About Hemophilia B

Hemophilia B is a rare, debilitating, hereditary bleeding disorder caused by a defect in the gene encoding coagulation factor IX (FIX). Hemophilia B is linked to the X chromosome and mainly affects boys and men; however, women who carry an affected copy of the coagulation factor gene may also experience symptoms. Hemophilia B is classified as mild, moderate or severe, depending on the level of FIX in the blood, and is diagnosed through blood tests. The 2020 Annual Global Survey by the World Federation of Hemophilia estimates that there are approximately 15,000 patients with hemophilia B in the United States, Europe and Japan. A meta-analysis using national registries in Australia, Canada, France, Italy, New Zealand and the UK estimated a prevalence in males of 3.8 in 100,000 or approximately 1 in 30,000.1

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The company is dedicated to improving patient lives through innovative, one-time treatments that may provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patients bloodstream. The companys integrated gene therapy platform includes in-house capabilities in research, clinical development and commercialization. The company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the U.S.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the opinions, expectations, beliefs, plans, objectives, assumptions or projections of Freeline Therapeutics Holdings plc (the Company) regarding future events or future results, in contrast with statements that reflect historical facts. Examples include, among other topics, statements regarding the timing, progress and results of the Companys Phase 1/2 B-LIEVE dose confirmation clinical trial of FLT180a and data readouts from that trial, whether a further refined immune management regimen may be required, the potential of FLT180a to deliver a best-in-class gene therapy for people with hemophilia B, whether the Companys evaluation of strategic options for FLT180a will result in any particular course of action and the potential of the Companys programs in Fabry disease and Gaucher disease to be first-in-class gene therapy treatments for such diseases and offer the highest value for patients and the Companys shareholders. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward-looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the United States, United Kingdom or European Union, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience, which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2021 and in subsequent reports on Form6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on theSECwebsite at http://www.sec.gov.

Media Contact:

Arne Naeveke, PhD Vice President, Head of Corporate Communications arne.naeveke@freeline.life +1 617 312 2521

IR Contact:

investor@freeline.life

References 1 Iorio A et al. Annals of Internal Medicine 2019;171(8):540-7

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WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced in a presentation by Armen Asatryan, MD, Chief Medical Officer, at the National Tay Sachs and Allied Diseases Annual Family Conference held in Denver, Colorado, that the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD) has shown encouraging early efficacy data and a favorable safety profile in the first three patients treated through their six month visit post-treatment in the Companys open-label Phase 1/2 First-in-Human (FIH) clinical trial at Dayton Childrens Hospital (Dayton, Ohio). Myrtelles FIH trial utilizes the Companys proprietary rAAV vector to directly target oligodendrocytes, the affected brain cells in CD responsible for producing myelin the insulating material that enables proper neuronal function. In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA). The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain, and thereby supporting myelination. Five patients have been treated in the ongoing Phase 1/2 study with favorable safety and tolerability results to date and no study drug-related adverse events.

Analyses of the first three patients at 6-months post-treatment by Magnetic Resonance Imaging (MRI) demonstrated increases in white matter and myelin in the brain. Improvements were also exhibited in scores on the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL), validated functional scales. Observed improvements contrast the continuous clinical deterioration expected with the natural progression of the disease.

The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients reflexes, eye tracking, vocalizations to communicate, and overall awareness said Robert Lober, MD, PhD, FAANS, Co-Principal Investigator and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine and Attending Neurosurgeon at Dayton Childrens Hospital in Dayton, OH. These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.

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Myrtelles current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease, commented Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle. We intend to build on these encouraging initial findings and complete our current Phase 1/2 clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as may, believe, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding encouraging early data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children diagnosed with Canavan disease, the improvements suggesting that the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease, the potential of Myrtelles current investigational gene therapy targeting oligodendrocytes, to usher in new treatment options for children with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

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Wilmington, Delaware, United States, Transparency Market Research Inc.: Patients suffering from idiopathic hyperhidrosis experience extreme sweating of the axilla, soles, palms, or face. Elevated environmental temperatures or emotional stress worsens sweat secretion; however, this extra sweating can be caused without any clear triggers. Typically, these symptoms develop in adolescence, get worse after puberty, and reduce during old age.

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Individuals may express inconvenience associated with humid footwear or trouble handling paper, tools, musical instruments, or sports equipment. More importantly, patients are embarrassed while shaking or holding hands, meeting new people, and intimacy, consequently leading to social withdrawal.

The global market for hyperhidrosis treatment reached a valuation of ~US$ 1.2 Bn in 2018, and is projected to grow at a CAGR of ~4% between the years 2019 to 2027.

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Rise in Demand for Minimally-invasive and Non-invasive Therapeutic Procedures to Boost Market

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During the fireside chat, moderated by Caroline Peachey, Editor of the European Pharmaceutical Review, Kobel will focus on TC BioPharm's journey as a CGT startup, the challenges the Company has faced, and the impact of quality control on its development. Kobel will also offer advice to other cell and gene therapy manufacturers and discuss the industry landscape.

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This press release may contain statements of a forward-looking nature relating to future events. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions. These statements reflect our current beliefs, and a number of important factors could cause actual results to differ materially from those expressed in this press release. We undertake no obligation to revise or update any forward-looking statements, whether as a result of new information, future events or otherwise. The reference to the website of TC BioPharm has been provided as a convenience, and the information contained on such website is not incorporated by reference into this press release.

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TC BioPharm CEO, Bryan Kobel to Speak at the "Innovating Cell and Gene Therapy Quality Control Conference 2022" - PR Newswire

Key Companies Covered in the Advanced Therapy Medicinal Products Market Research are Spark Therapeutics, Inc., Bluebird Bio, Inc., Novartis AG, uniQure N.V., Celgene Corporation, Gilead Lifesciences, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., MEDIPOST, Vericel Corporation and other key market players.

Global Advanced Therapy Medicinal Products Market is valued approximately at USD 7.9 Million in 2020 and is anticipated to grow with a healthy growth rate of more than 13.2% over the forecast period 2021-2027.

Advanced Therapy Medicinal Products have enabled the pharmaceutical industry and healthcare industry to open new modes for the treatment of incurable diseases and cancer types. The global Advanced Therapy Medicinal Products market is being driven by growing prevalence of diseases, an expanding healthcare industry and increasing research and development.

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For instance, in 2020, WuXi Advanced Therapies launched new closed process CAR-T Cell Therapy Platform to accelerate the timeframe for cell and gene therapy expansion, which is expected to boost the market growth. In 2020, EdiGene and Immunochina collaborated on Research & Development to develop allogeneic CAR-T therapy for cancer. Furthermore, the strategic development between hospitals and companies along with the government regulations and approvals will provide new opportunities for the global Advanced Therapy Medicinal Products industry. However, high manufacturing costs and low investments by the governments of less developed countries in the healthcare may impede market growth over the forecast period of 2021-2027.

In the regional analysis of the global Advanced Therapy Medicinal Products Market, North America is dominating the market while Asia-Pacific is expected to have the fastest growth rate among the key regions such as Asia Pacific, North America, Europe, Latin America, and Rest of the World. North America is the leading region across the world in terms of market share due to growing demand for therapies in ambulatory centers and hospitals, technological progressions coupled with the well-established healthcare infrastructure which has stimulated product demand in the region. Whereas, high growth rate of Asia-Pacific can be attributed to the increasing number of cancer patients and people getting more aware due to government and private investments in improving healthcare infrastructure in the region.

The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players.

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The detailed segments and sub-segment of the market are explained below:

By Type:CAR-T TherapyGene TherapyCell TherapyTissue Engineered Product

By Region:North AmericaU.S.CanadaEuropeUKGermanyFranceSpainItalyROE

Asia PacificChinaIndiaJapanAustraliaSouth KoreaRoAPACLatin AmericaBrazilMexicoRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2018, 2019Base year 2020Forecast period 2021 to 2027.

Target Audience of the Global Advanced Therapy Medicinal Products Market in Market Study:

Key Consulting Companies & AdvisorsLarge, medium-sized, and small enterprisesVenture capitalistsValue-Added Resellers (VARs)Third-party knowledge providersInvestment bankersInvestors

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Table of content

What is the goal of the report?

The market report presents the estimated size of the Market at the end of the forecast period. The report also examines historical and current market sizes. During the forecast period, the report analysis the growth rate, market size, and market valuation. The report presents current trends in the industry and the future potential of the North America, Asia Pacific, Europe, Latin America, and the Middle East and Africa markets. The report offers a comprehensive view of the market based on geographic scope, market segmentation, and key player financial performance.

What is the key information extracted from the report?

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Advanced Therapy Medicinal Products Market [REVENUE SOURCE] 2022 Gross Margins and Forecast Research Report till 2030 - Taiwan News

What happened

The downtrodden biotech space has kicked off the second half of 2022 with a boom. Hard-hit gene-editing and gene therapy companies in particular have started the back half of the year on the right foot. Underscoring this point, Bluebird Bio (BLUE 2.59%) stock has already risen by 17% over the holiday-shortened week as of Thursday's closing bell, according to data provided by S&P Global Market Intelligence.

What's more, shares of CRISPR Therapeutics (CRSP -1.92%) have gained 22.6% over the same period, and fellow gene editor Editas Medicine (EDIT -1.32%) also saw its equity rise in price by a healthy 20.7% this week. By contrast, Bluebird and Editas shares both fell by over 50% over the first six months of 2022, while CRISPR's stock price stumbled by a noteworthy 20% during the first half of the year.

Image Source: Getty Images.

What's behind this sudden trend reversal? The most likely explanation is simply short-sellers covering their positions (buying back their borrowed shares). In keeping with this theme, Bluebird, Editas, and CRISPR all saw a sharp rise in their short interest during the first six months of 2022. Short-sellers piled into these three names earlier this year due to the fact that they are all cash flow negative, which is a tough spot to be in during a persistent bear market and an era of rising interest rates. Bluebird, in fact, is staring down a serious cash crunch at the moment.

Short-sellers, for their part, are probably backing away at this stage for no other reason than to play it safe in the event that big pharma starts to go bargain shopping.

Why might big pharma target beaten-down gene-editing and gene therapy companies in the second half of the year? The key reason is that these high-value fields are starting to move beyond the research stage of their life cycle and into the realm of commercially available therapies.

Speaking to this point, Bluebird's gene therapies for beta thalassemia and cerebral adrenoleukodystrophy appear to be on their way toward a formal approval from the Food and Drug Administration (FDA) following a pair of positive advisory committee votes last month. What's more, CRISPR is also expected to file for regulatory approval for its Vertex Pharmaceuticalspartnered blood disorder candidate, exa-cel, later this year.

Are any of these three biotech stocks still worth buying? CRISPR is arguably the most attractive bargain among the three. The company's ex-vivo gene-editing platform has posted stellar trial results so far, and Vertex could very well decide to buy its partner as a result.

Bluebird, on the other hand, is a tough call. The company ought to have a compelling buyout case if the FDA does grant it a pair of approvals soon. The bad news is that the biotech's balance sheet may force a sale at a heavily discounted price (relative to the commercial potential of its lead assets).

Finally, Editas might simply get lost in the mix when everything is said and done. There are several gene-editing companies vying for the spot of top dog, and Editas' clinical pipeline lags in several key areas at the moment. Time will tell.

George Budwell has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics, Editas Medicine, and Vertex Pharmaceuticals. The Motley Fool recommends Bluebird Bio. The Motley Fool has a disclosure policy.

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Why Shares of Bluebird Bio, CRISPR Therapeutics, and Editas Medicine Soared This Week - The Motley Fool

In 2019, when Immuneel Therapeutics Ltd, founded by Pulitzer winning author and US oncologist Dr Siddhartha Mukherjee and leading biotech entrepreneur Kiran Mazumdar Shaw, planned to set up a breakthrough cell therapy facility for cancer treatment in India, its top team never imagined the world would be hit by Covid-19. It faced the brunt of the pandemic, but the team adapted fast from vacating its one-room office in Bengaluru to locating the construction crew near the hospital and finding Indian vendors instead of outsourcing the project to a global firm through the last two years to get the facility up and running.

No amount of preparation would have helped us plan for something like this. There were several challenges: one, you are building something in a hospital, two, its a cancer facility, three, there were restrictions on the number of people you could bring in at a time, four, the entire supply chain was disrupted, and you couldnt get anything shipped from any of the usual suppliers. And we had to ensure high quality GMP (good manufacturing practices) production of cell therapies in a country like India where this had never been done before, said Dr Arun Anand, Chief Operating Officer, Immuneel Therapeutics.

A similar therapy has also been indigenously developed by Mumbais Tata Memorial hospital and IIT Bombay. It is at an early Phase I clinical trial stage.

Not only did Immuneel have to design the centre with GMP (good manufacturing practices), but the team conducting the trial also had to ensure adequate training for the staff, with many posted on Covid-19 duty. The centre is located on the eighth floor of Mazumdar Shaw Medical Center at Narayana Hrudayalaya Health City in Bengaluru.

The centre would conduct Phase II trial of a Spanish CAR-T therapy for treating patients with leukaemia and lymphoma. It has a strategic collaboration with Spain-based Hospital Clinic de Barcelona (HCB) and it has successfully conducted remote tech transfer with HCB on manufacturing protocols. The therapy modifies the patients T-cells using an inactivated HIV virus to weaponise them to fight the cancer cells. In CAR-T cell therapy, we extract t cells from a cancer patients body and use a virus a modified version of HIV virus that has no risk for HIV to deliver genes to the cells that weaponise the T-cells and can attack the cancer cells, said Dr Mukherjee.

We wanted to be in a hospital ecosystem for the team to feel connected to the mission of serving patients. A feeling of empathy is necessary to do something creative; to come up with solutions. We are on the eighth floor in the hospital, and the bone marrow transplant centre is on the seventh floor. Every day when patients go up and down the elevators, we are also travelling, and we see the patients and physicians. This is unique for a company; most companies prefer to set up in lovely ecosystems, glass-faade buildings, outside of the city, far from where the real work happens, said Dr Anand.

Explaining the process, Dr Mukherjee said, We remove the T-cells from the body through apheresis, they go into an extremely sterile laboratory where everyone is working with hoods and masks, no one is allowed to enter except for the people manipulating them, these T-cells are then weaponised using this gene therapy, and then they are grown in the lab in incubators until they increase to a large number, then they are frozen. This process takes about ten to fourteen days.

Then you have to do very careful quality control, which involves making sure that the product is sterile, that the gene therapy has actually worked, that the product has actually expanded and expanded in the right way. And, then it is released. In the hospital, it is transfused back into patients, he said.

With trials and innovations in India, the team hopes to bring down the cost of the therapy from the current US $350,000 to $400,000. The clinical trial has already started, and we have dosed several patients, said Dr Mukherjee who was in India recently for the project.

We were a small team then just 20 people and we occupied a small room on the seventh floor of the hospital. And one evening I was informed by the hospital administrator that they needed the entire floor because they wanted to convert that into a Covid-19 ward. They did it in the next two days. So, overnight we did not even have a room to sit in. What that meant is we implemented an early work-from-home plan, said Dr Anand.

When our construction started in early 2020, we arranged for the contractor to house the entire crew very close to the hospital. The entire crew was in 3-4 houses taken on long-term lease just a kilometre away from the hospital. The crew did not need to travel by bus or public transport, they could just walk. And all of them were tested on a periodic basis (for Covid-19), he said.We did a session with the workmen and the contractor, telling them what we are trying to do. When they realised their work would save the lives of children, they were motivated. I am a firm believer if the purpose is noble, there will be support from the ecosystem to make it happen, he said.

The construction period was stretched to accommodate for breaks and shorter shifts in view of protective equipment being worn by the workers. Instead of nine months, the project was completed in 12. The crew was also trained on wearing masks, and were regularly tested, but still ended up getting the infection, said Anand.

Dr Sharat Damodar, one of the principal investigators of the clinical trial, said, Training the staff took some time till recently Covid-19 was the priority, so most of our nurses and doctors were posted on Covid-19 duty so to pull them out in the middle of it for training for such a new therapy was obviously difficult. Most of the people were drawn from a pool of nurses and doctors who were not doing Covid-19 duty, who were helping in bone marrow transplant and high-end chemotherapy.

He said it was good the clinical trial started as soon as the Covid-19 cases dropped because patients were happy to come in for the new therapy, which promised a better outcome than existing therapies. Emphasising the need to bring the technology to India, he said prior to the pandemic, many Indians travelled to China for treatment, at a lower cost.

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Cell therapy cancer centre takes shape in Bengaluru, trials are on - The Indian Express

HOUSTON--(BUSINESS WIRE)--Kiromic BioPharma, Inc. (NASDAQ: KRBP) (Kiromic or the Company), a clinical-stage fully integrated biotherapeutics company using its proprietary DIAMOND artificial intelligence and data mining platform to develop cell and gene therapies with a focus on immuno-oncology, announces the timely completion of construction on its expanded current good manufacturing practice (cGMP) manufacturing facility in Houston. This significant milestone was accomplished within the timeline established by the Company, specifically June 30, 2022.

The expanded facility located at Kiromics headquarters is one of the conditions required for the Company to begin the activation of its cell therapy clinical trial for the Deltacel product candidate by the end of this year. The completion also addresses a key component in the clinical hold communication the Company received from the U.S. Food and Drug Administration (FDA) in June 2021.

The on-time completion of our cGMP manufacturing facility is one of the conditions necessary to begin the activation of the Deltacel clinical trial by the end of this year, stated Pietro Bersani, Kiromic BioPharmas Chief Executive Officer. The facility supports an expanding product pipeline of cell therapies designed to target solid tumors, furthering our commitment to delivering lifesaving treatments to patients with cancer who have limited therapeutic options. We believe our allogeneic, off-the-shelf manufacturing process will result in shorter lead times and lower costs, thereby increasing the availability of these promising cellular therapies for oncology patients.

The expanded 34,000-square-foot facility includes flexible cellular therapy and viral vector suites, a dedicated cGMP microbiology lab, a dedicated cGMP quality control (QC) lab, a research and development laboratory, and an FDA Code of Federal Regulations (CFR-9) compliant vivarium.

About Kiromic BioPharma

Kiromic BioPharma, Inc. is a clinical-stage, fully integrated biotherapeutics company using its proprietary DIAMOND artificial intelligence (AI) 2.0 platform to discover and develop cell and gene therapies with a therapeutic focus on immuno-oncology and other diseases. Kiromic is in the process of developing a multi-indication allogeneic cell therapy platform that exploits the natural potency of Gamma Delta T-cells to target solid cancers. From its heritage as a cancer vaccine development company, Kiromic is focused on discovering, developing, and commercializing novel immuno-oncology applications through its robust product pipeline. The pipeline development is leveraged through the Companys proprietary target discovery engine called "DIAMOND." Kiromic's DIAMOND is where data science meets target identification to dramatically compress the years and hundreds of millions of dollars required to develop a live drug. The Company maintains offices in Houston, Texas. To learn more, visit http://www.kiromic.com and connect with us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Kiromic makes such forward-looking statements pursuant to the safe harbor provisions of the United States Private Securities Litigation Reform Act, Section 21E of the Securities Exchange Act of 1934, as amended, and other federal securities laws. All statements other than statements of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as: will, potential, could, can, believe, intends, continue, plans, expects, anticipates, estimates, may, or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements regarding: Kiromics current and anticipated IND applications including statements regarding the scope of and timing for submission of an IND application; the Deltacel product platform; the sponsored research agreement and the data that will be generated as a result of such collaboration; the timing for submitting and activating Kiromics IND applications; the benefits of utilizing non-genetically engineered Gamma Delta T cells as our first in-human study; Kiromics ability to achieve its objectives; and the timing for the initiation and successful completion of Kiromics clinical trials of its product candidates. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to, the risks and uncertainties discussed in our Annual Report on Form 10-K for the year ended December 31, 2021, and as detailed from time to time in our SEC filings. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance, or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements. Such forward-looking statements relate only to events as of the date of this press release. We undertake no obligation to update any forward-looking statements except to the extent required by law.

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Kiromic BioPharma Achieves Milestone with Timely Completion of Expanded cGMP Manufacturing Facility to Support Cell Therapy Oncology Pipeline -...

If a new Planet of the Apes or Jurassic Park film comes out, Im going to go see it. The latest, Jurassic World: Dominion, didnt disappoint.A plague of locusts

The science is mostly accurate, the bioethics message obvious, and the plot adheres to Isaac Asimovs change one thing rule for science fiction. In the world of Jurassic Park, that lone variable is time. We just have to accept that a titanosaur like Argentinosaurus somehow grew and developed from a lab-nurtured baby to a 130-foot-tall and 110-ton adult in a few years.

The official summary from IMDb for the new film is vague and continues the impossibly-rapid-growth theme:

Four years after the destruction of Isla Nublar, dinosaurs now liveand huntalongside humans all over the world. This fragile balance will reshape the future and determine, once and for all, whether human beings are to remain the apex predators on a planet they now share with historys most fearsome creatures in a new Era.

Four years? Animals radiating around the world? Even rats or rabbits couldnt do that. And the film is actually more about insects.

Evil company Synbio has let loose hordes of genetically modified locusts, each the size of a shoebox. They eat only crops that havent been grown with the companys insecticide. (Old story, the first GMO corn was grown in 1996.) The rapidly-reproducing, gargantuan locusts are expected to decimate the nations crops by summers end.

The upper floors at Synbio appear innocent: a clearinghouse to rehab the poor reptiles roaming the planet after they burst free in the last movie. The beasts are then sent to a Wildlife Relocation Center in Pennsylvania or to a facility in the Dolomite Mountains of Italy for some R&R. Synbio bought the company behind Jurassic Park back in the 90s, and it is now focusing on saving 20 key species.

But there are other parts to the company.

The Habitat and Development Laboratory contains untouched genomes of rare species. Underground lies Synbios top secret project, hexapod. Six legs.

The company is conveniently near amber deposits that provided the initial dino DNA back in the 1990s in preserved mosquitoes, and this time around, the locust genetic material. The amber mine looks a lot like Howe Caverns, near where I live in upstate New York.

Of course other folks have dinos too. Sawridge Cattle Company in western Nevada, for example, is now an illegal breeding facility. Cute baby triceratopses are stuffed into cages like the ones at county fairs that house pigs.

The locusts and dinosaurs are the backdrop to the human drama that centers around 14-year-old Maisie Lockwood (Isabella Sermon), granddaughter of Sir Benjamin Lockwood. He was the business partner of John Hammond, both responsible for the modern-day dinos in the first place.

Maisie, it turns out, is a clone. Her mother, Charlotte (Elva Trill), wanted to have a kid, so why not just clone herself? The 23 human chromosome pairs on her computer screen announce that she is a geneticist, although Im a geneticist too and I never feel the need to remind myself of the number, which isnt useful for anything.

Young Maisie is precocious and gorgeous and for unexplained reasons, has an English accent. Owen Grady (Chris Pratt) and Claire Dearing (Bryce Dallas Howard), from the last movie, adopt her and they live in the woods to protect her from kidnappers.

A few dinosaurs live near their house, and Maisie befriends an adorable mom and her baby, Blue and Beta. They arose from dino DNA mixed with monitor lizard DNA, like the frog DNA patched into the genomes of the original Jurassic Park dinos. We need the little raptor to understand you. They are genetically identical, like you and Charlotte. Charlotte, like Blue, was able to have a child all by herself, sneers a kidnapper, for Maisie, too, arose without the contribution of a sperm.

But why would anyone want to kidnap Maisie? Why is she the most valuable IP on the planet?

When Maisie was a toddler, her mother Charlotte, much to her surprise despite the chromosomes on her screen, suddenly sickened from an unnamed, undiagnosed genetic disease, even though young-adult-onset of a genetic disease is rare. Oops! She cloned a child with a mystery mutation!

Not to worry. Charlotte whipped up some viral DNA to deliver working genes, something thats been done in gene therapy since the first experiments, in 1990, coincidentally when Michael Crichton published Jurassic Park. Back then, we could just add genes in gene therapy. But now, thanks to gene editing techniques like CRISPR, we can also remove the bad genes.

Presumably such a gene swap is what Charlotte did to beget Maisie. Whatever she did, we know that the important part is that she made the change in every single cell of her toddlers body, which is difficult to envision. Several characters say every single cell so we know it is true, although with current technology, that is not possible. Gene therapy on a person targets specific cell types that are involved in the disease.

However Charlotte jettisoned the bad genes out of her kid, Maisie is here, and she holds the secret to how pathologist Henry Wu (BD Wong from Law and Order) can rid the world of the locusts: apply the same technology that Charlotte used to knit a genetic change into all the trillions of her daughters cells.

She changed every cell in your body, Wu reminds Maisie. If I can figure out how, I can change the entire swarm before its too late. Your DNA could change the world! If I could, I could fix a terrible mistake that I made, he laments, referring to the bioengineering of the giant locusts.

A large-scale change that eradicates a species, even if locally, is indeed possible with a technology called a gene drive. I explain it here. Its not a very good idea.

The problem of the locusts is so profound that the old characters from the original Jurassic Park, circa 1993, come aboard. They re-enact some of the old bantering and flirting, nearly verbatim.

Laura Derns Ellie Sattler gee-whizzes and eye-bulges. Shes the paleobotanist who stuck her hands in dino doo in the inaugural film. She helpfully identifies the locusts as a species that died out during the Cretaceous period (145 to 66 million years ago).

Grumpy paleontologist Alan Grant (Sam Neill) is still grumpy, while pontificating mathematician Ian Malcolm (Jeff Goldblum, also of The Fly fame) still is plagued by clichs: ethics of genetic power, we must trust in humanity, unforeseen consequences, and the nonsensical we must transform human consciousness. He works for the evil company or so it seems. Synbios work, he spouts, will cure autoimmune disease, cancer, and Alzheimers.

Ian invites Ellie and Alan to visit Synbio. Upon arrival, Ian slips her a key to the locust lab.

Theyve all aged remarkably well, trim and with great hair and skin. This contradicts the change one thing mantra of rapidly-developing dinosaurs; non-aging humans.

Several boring chase scenes ensue, the one of barbecuing locusts the most intriguing. The main characters spend about 45 minutes running around the Synbio facility to steal a sample of DNA. It is unclear exactly why theyre doing this, because they can easily get DNA from Maisie, and theyre surrounded by the insects, presumably pooping DNA all over the place.

As the movie drags on, more chase scenes happen, in response to various asset containment breaches. The most exciting is a duel between two ferocious dinos: two apex predators in one place! Ellie chants. Oh my!

In yet another scene, as a horrified Claire looks on in a forest, a ginormous dino slurps up deer like my husband eats Trader Joes potato chips.

Our friends, after many near-death experiences, save the day. The surviving humans flee as dinos burn. At an unnamed later time, Dr. Wu waves a magic wand and the locust problem is solved.

The plot holds together loosely. What kept me awake, once Id gotten over the novelty of the dinosaurs (yes, they have feathers), were the scenes borrowed from other films and TV shows.

Owen spies a pterodactyl on the wing of a plane that resembles Star Wars Millennium_Falcon, echoing the Nightmare at 20,000 Feet Twilight Zone episode in which William Shatner (aka Captain Kirk) spies a monster on the wing of a plane.

Why did it have to be snakes? uttered Indiana Jones after plunging into a serpent-filled chasm. Nobody said thered be bugs! is the new version.

Claire bails out of the Millennium Falcon and evokes the winged monkeys of the Wizard of Oz. Later on, shes spies small dinos pop up from the shrubbery like the munchkins along the yellowbrick road.

Claire and Owen sneak into the locust lab wearing white suits that look EXACTLY like the deployed sperm in Woody Allens Everything You Always Wanted to Know About Sex.

For no clear reason, theres a re-enactment of the cantina scene from Star Wars, complete with locusts turning on a barbecue spit, gambling, and drinking.

Despite the absurdity of populating a planet with full-grown, bellowing dinosaurs in a fraction of time, Ill go back for the next installment of Jurassic World. Meanwhile, Id like to know how Ellie, Alan, and Ian aged so well.

Ricki Lewis has a PhD in genetics and is a science writer and author of several human genetics books.She is an adjunct professor for the Alden March Bioethics Institute at Albany Medical College.Follow her at herwebsiteor Twitter@rickilewis

A version of this article was originally posted atPLOSand has been reposted here with permission. PLOS can be found on Twitter@PLOS

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DNA and gene editing are the real stars of the new Jurassic Park movie - Genetic Literacy Project