Category Archives: Cell Therapy

The potential importance of cell and gene therapy (CGT) to healthcare and the biopharma industry seems clear. CGT accounts for just 1 percent of launched products in major markets, with treatment of the vast majority of diseases still using small-molecule drugs. Yet those productswhich include cell therapies, such as chimeric antigen receptor (CAR) T-cell therapy for aggressive B-cell lymphomas, and gene therapiesto treat a range of monogenic rare diseaseshave proved transformative for patients. And there are many more in development. As of February 2020, CGT products account for 12 percent of the industrys clinical pipeline and at least 16 percent of the preclinical pipeline, but as most manufacturers do not disclose their preclinical assets, the true figure may be considerably higher (Exhibit 1).

Exhibit 1

New CGT products will surely emerge from this pipeline upon the continuing discovery of indications that CGT can address and the growing industry understanding of the genetic drivers and determinants of more complex, multifactorial diseases. Indeed, the pace of CGT-asset development is similar to that of monoclonal-antibody (mAb) assets in that modalitys early years, and mAb therapy went on to transform the biopharma market (see sidebar, Cell and gene therapy: Mirroring monoclonal-antibody therapy).

Exciting clinical results are helping to propel this pace. Success rates for CGT products are higher than those for small-molecule products, probably because CGT tends to target specific disease drivers rather than the broad targets (with potential for off-target effects) of small-molecule therapy. The sample size of launched CGTs is small, so comparisons may change as the market evolves. Nevertheless, there is a marked difference thus far. Between 2008 and 2018, the R&D success rate from Phase I to launch for small-molecule products was 8.2 percent; for CGT products, it was 11 percent.

Recognizing CGTs potential, 16 of the worlds largest (by revenue) 20 biopharmacos now have CGT assets in their product portfolios. Yet most companies are moving cautiouslyonly two of the top 20 have CGT assets making up more than 20 percent of their pipelines. They are still considering whether, when, and how to reposition their portfolios. In the meantime, biotech companies remain leaders in CGT innovation.

As of February 2020, only a small percentage of launched CGT assets either originated from or are owned by a top 20 biopharmacoin both cases, only 15 percent of launched assetsindicating how much opportunity there is for such companies to increase their exposure to CGT assets (Exhibit 2).

Exhibit 2

The figures are not altogether surprising, given that biopharmacos expertise often lies in disease areas, not in the development of the technology platforms that generate CGT products. More often than not, the original research behind new platforms is conducted by academics (who go on to set up their own biotech companies) and investors (whose models include company origination because of the potential financial gains and the concentrated technical risk that platform investments carry). Venture-capital firms are more comfortable than established biopharmacos with such risks.

Nevertheless, given the growth potential of CGT and the promise it holds for patients, most large biopharmacos are considering increasing their presence in the market. This article is intended to help guide their decisions, describing the key considerations when assessing investment opportunities and the various entry strategiesas well as the trade-offs to be made when choosing among them.

There are many technology platforms in development that seek to address different challengesassociated with CGT. In cell therapy, work is afoot to improve the manufacture of autologous therapies to reduce the cost of goods sold or vein-to-vein time, enable breakthrough efficacy in solid tumors, and improve the patient or customer experience. In gene therapy, there are investment opportunities in platforms that aim to overcome the limitations of current vectors (such as the size of the transgene, suboptimal tropism, or the triggering of an immune response) that enable nonviral delivery methods, reduce manufacturing costs, and expand manufacturing capacity.

The decision, therefore, is about not only whether to increase investment in CGT but also which technology platforms or assets to back. Companies should thus assess each investment opportunity by both strategic fit and technology attractiveness. Strategic considerations on a CGT platform or asset include whether it complements a companys disease areas of focus, the internal pipeline would benefit from diversification with new modalities, and the company has the required capabilities, capital, and conviction.

A host of questions need to be asked to gauge the attractiveness of the technology. Has it demonstrated proof of concept? What risks remain? Does the company have enough understanding of the underlying mechanisms? Does the technology enable first-mover advantage? What are the intellectual-property considerations? Is the platform differentiated from competing platforms? And given the rapid pace of innovation in CGT, what is the risk that the technology platform quickly becomes obsolete?

CAR T-cell therapy, whereby a patients T cells are genetically engineered to express a chimeric antigen receptor that targets a specific tumor antigen, illustrates the potential risk. In a relatively short time, the field has progressed from an initial set of constructs to a second generation that has given rise to two FDA-approved products, YESCARTA and KYMRIAH, even as third- and fourth-generation products are in development.

Investment opportunities that have a strong strategic fit and high-potential technologythose that fall into the top-right quadrant shown in Exhibit 3will be attractive. For example, a CAR T-cell or T-cell-receptor platform would fall in the top right for many oncology-focused companies. In the absence of such opportunities, those in the top-left or bottom-right quadrants may still be worthwhile as a means of gaining exposure to CGT, perhaps through an early-stage investment. For example, next-generation, unproven gene-editing technologies may fall in the bottom-right quadrant for companies focused on rare diseases with known genetic drivers. Companies would have to be prepared to tolerate the associated risks, however, and not all will conclude that now is the time to make a move.

Exhibit 3

Once a manufacturer has decided that it makes strategic sense to invest in CGT and has identified an attractive technology, it must choose an entry strategy. There are three main options: build a proprietary platform, buy an existing platform or one or more of its assets, or form a partnership to gain access to assets on platforms developed by others (Exhibit 4). The three options have different profiles in the capital required, changes to the operational model needed, and risk (as measured by the degree of diversification offered across different technologies).

Exhibit 4

Companies that build a platform or platforms from scratch enjoy full control over development efforts and retain all the financial rewards of successful assets. They also get the chance to build their own CGT capabilitiesscientific, clinical, and commercialand have the freedom to adapt as the technology evolves. In return, they have to commit significant resources to internal R&D and will, in effect, be placing big, early bets on a single or very limited number of platforms. Additionally, they may need to make significant changes to operating models designed for traditional modalities.

Buying a developed platform or late-stage asset carries less technical risk (assuming robust early data), though invariably a price premium too. This means that few, if any, companies will be able to acquire a large number of them, so companies continue to bet on a single or limited number of platforms.

The third optionforming a partnership to gain access to assets on platforms that others have developedlies between these two extremes in investment cost and risk. Because partnerships in the still-nascent CGT sector are relatively cheap, biopharmacos can afford to spread their bets on where future success might lie through establishing several partnerships.

Accordingly, most biopharmacos to date have followed the partnership route when placing a stake in CGT. Between 2010 and 2014, there were a total of 16 M&A deals in the CGT space. That rose to more than 60 between 2015 and 2019. However, even in 2019, when M&A activity was strongest, partnerships accounted for more than 80 percent of total transaction activity (Exhibit 5).

Exhibit 5

Nearly all of the top 20 biopharmacos have formed at least one partnership, while ten have made an acquisition. Just one has built its own platform. Exhibit 6 details this, along with the impact that the deals have had on the composition of company pipelines.

Exhibit 6

Partnerships come in three main varieties: those that give a biopharmaco access to a single asset, those that give it access to all assets in selected therapeutic areas that might emerge from a platform, and those that give it access to all platform assets, regardless of the therapeutic area or indication.

Partnerships structured to give a biopharmaco access to a single asset are the simplest way to enter the CGT market and are often chosen by companies that have a strong focus on certain indications and believe that their competitive advantage lies in owning multiple therapies across modalities in that space. A single-asset partnership also minimizes the investment required. However, this kind of partnership may leave a biopharmaco having to introduce a new operating model for a single asset.

Partnerships structured to give a biopharmaco access to all assets from a platform in certain therapeutic areas can help companies with a strong strategic focus on a given therapeutic area strengthen their portfolios and build more expertise in that area. In addition, more assets in a new modality means more opportunity to build the relevant development and commercial expertise.

The third option, partnering to win access to all the assets in a particular modality generated by a platform, tends to be the partnership of choice for biopharmacos that believe future competitive advantage lies in access to the best technology, no matter what may be the associated indication or therapeutic area. Through such a partnership, a company can follow the science, developing the technology for the indications in which it can provide the most clinical benefit. Such a strategy requires more investment than other forms of partnership, however, and so carries more concentrated technology risk. Companies may also find themselves developing products for therapeutic areas in which they have no expertise and thus are at a competitive disadvantage.

In addition to these three kinds of partnerships with biotech companies, some biopharmacos are considering more innovative ways to allocate their limited resources across multiple CGT technologies in a manner that also boosts their chances of keeping pace with rapid innovation. By partnering with venture-capital firms or biotech originators to launch new assets, new platforms, or even new companies or by collaborating with large academic institutions to license multiple new technologies, they are making much earlier-stage bets on where future success might lie.

The CGT era is an exciting one for healthcare, and all biopharmacos will want to reassess their portfolio strategies to decide whether and to what extent to diversify their pipelines. Most big biopharmacos have chosen partnerships to explore CGT initially, though the likelihood is that many will use a combination of strategies to increase their exposure and access to several technologies as the market evolves. Yet whether a company is still testing the water or is ready to commit, it will need to think carefully about how it builds its exposure to the CGT market and be fully aware of how to assess each investment opportunity, the range of possible entry strategies, and the different advantages and risks that each carries.

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Cell and gene therapy: Biopharma portfolio strategy | McKinsey

Dublin, Oct. 05, 2021 (GLOBE NEWSWIRE) -- The "Global Gene and Cell Therapy (GCT) Market - Analysis By Vector, Application, By Region, By Country (2021 Edition): Market Insights, Pipeline, Forecast with Impact of COVID-19 (2021-2026)" report has been added to ResearchAndMarkets.com's offering.

The Global Gene and Cell Therapy (GCT) Market is estimated at USD 2504.2 Million in the year 2020

Growth in the historic period in the cell and gene therapy market resulted from increase in investments in cell and gene therapies, growth in research and development, advances in cancer drug discovery, rise in public-private partnerships, strong economic growth in emerging markets, increased healthcare expenditure, and rising in pharmaceutical R&D expenditure.

Companies in the gene and cell therapy for oncology market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises.

While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

Further, the market was restrained by inadequate reimbursements, challenges due to regulatory changes, low healthcare access, and limited number of treatment centers.

Going forward increasing prevalence of cancer and chronic diseases, rising geriatric population, rising geriatric population, rising focus on cell and gene therapy, and rise in healthcare expenditure will drive the growth in the gene and cell therapy market.

Factors that could hinder the growth of the market in the future include high costs of therapy, stringent regulations, reimbursement challenges, and coronavirus pandemic.

The report tracks competitive developments, strategies, mergers and acquisitions and new product development. The companies analysed in the report include F. Hoffman-La Roche Ltd., Novartis, Sanofi, Alnylam Pharmaceuticals Inc., Pfizer, BlueBird Inc., Sarepta Therapeutics, Voyager Therapeutics, Orchard Therapeutics Plc, AnGes Inc.

Story continues

Key Topics Covered:

1. Report Scope and Methodology1.Report scope & Methodology1.1 Scope of the Report1.2 Research Methodology1.3 Executive Summary

2. Strategic Recommendations

3. Gene and Cell Therapy Market: Product Outlook

4. Global Gene and Cell Therapy Market: Sizing and Forecast4.1 Global Gene and Cell Therapy Market Size, By Value, Year 2016-2026

5. Global Gene and Cell Therapy Market Segmentation - By Vector, By Application5.1 Competitive Scenario of Global Gene and Cell Therapy Market: By Vector5.1.1 Lentivirus - Market Size and Forecast (2016-2026)5.1.2 AAV - Market Size and Forecast (2016-2026)5.1.3 Retrovirus & Gammaretrovirus - Market Size and Forecast (2016-2026)5.1.4 Others - Market Size and Forecast (2016-2026)5.2 Competitive Scenario of Global Gene and Cell Therapy Market: By Application5.2.1 Oncology - Market Size and Forecast (2016-2026)5.2.2 Neurological Disorders - Market Size and Forecast (2016-2026)5.2.3 cardiovascular disorders - Market Size and Forecast (2016-2026)5.2.4 Others - Market Size and Forecast (2016-2026)

6. Global Gene and Cell Therapy Market: Regional Analysis6.1 Competitive Scenario of Global Gene and Cell Therapy Market: By Region

7. North America Gene and Cell Therapy Market: An Analysis (2016-2026)7.1 North America Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value7.2 North America Gene and Cell Therapy Market - Prominent Companies7.3 Market Segmentation By Vector (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others)7.4 Market Segmentation By Application (Oncology, Neurological Disorders, cardiovascular disorders and Others)7.5 North America Gene and Cell Therapy Market: Country Analysis7.6 Market Opportunity Chart of North America Gene and Cell Therapy Market - By Country, By Value, 20267.7 Competitive Scenario of North America Gene and Cell Therapy Market: By Country7.8 United States Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value7.9 United States Gene and Cell Therapy Market Segmentation - By Vector, By Application (2016-2026)7.10 Canada Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value7.11 Canada Gene and Cell Therapy Market Segmentation - By Vector, By Application (2016-2026)

8.Europe Gene and Cell Therapy Market: Segmentation: An Analysis (2016-2026)

9.Asia Pacific Gene and Cell Therapy Market:: An Analysis (2016-2026)

10.Global Gene and Cell Therapy Market Dynamics10.1 Drivers10.2 Restraints10.3 Trends

11.Market Attractiveness11.2 Market Attractiveness Chart of Global Gene and Cell Therapy Market - By Vector, 202611.3 Market Attractiveness Chart of Global Gene and Cell Therapy Market - Application, 202611.4 Market Attractiveness Chart of Global Gene and Cell Therapy Market - By Region, 2026

12.Competitive Landscape12.1 Major Technological Innovations, Mergers & Acquisitions and Role of Manufacturers12.2 Product Pipeline of Leading Gene Therapy Companies12.3 Market Share Analysis

13.Company Analysis13.1 F. Hoffman-La Roche Ltd.13.2 Novartis13.3 Sanofi13.4 Alnylam Pharmaceuticals Inc.13.5 Pfizer13.6 BlueBird Inc.13.7 Sarepta Therapeutics13.8 Voyager Therapeutics13.9 Orchard Therapeutics Plc13.10 AnGes Inc.

For more information about this report visit https://www.researchandmarkets.com/r/7ixaxq

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Global Gene and Cell Therapy (GCT) Markets, 2016-2020 ...

Chimeric antigen receptor (CAR) T-cell therapy is a type of immunotherapy that uses a patients own genetically modified T cells to find and kill cancer. UPMC Hillman Cancer Center currently offers two types of FDA-approved CAR T-cell therapy.

UPMC Hillman Cancer Center was one of the first in the United States certified to provide ABECMA (idecabtagene vicleucel) for adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an antidCD38 monoclonal antibody.

Hillman was the first of 10 centers in the United States to offer BREYANZI (lisocabtagene maraleucel), an FDA-approved CAR T-cell therapy for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including:

UPMC Hillman Cancer Center is the first in western Pennsylvania to provide TECARTUSTM (brexucabtagene autoleucel), an FDA-approved CAR T-cell therapy for patients with relapsed or refractory mantle cell lymphoma.

UPMC Hillman Cancer Center is part of the network of certified treatment centers providing KYMRIAHTM(tisagenlecleucel), an FDA-approved CAR T-cell therapy for:

UPMC Hillman Cancer Center is the first center in western Pennsylvania providing YESCARTATM (axicabtagene ciloleucel), the first FDA-approved CAR T-cell therapy for adult patients with certain types of B-cell lymphoma.

The FDA has approved this treatment for patients with the following conditions that have either not responded to or have relapsed following two or more lines of systemic therapy:

Patients will undergo an extensive evaluation to determine their eligibility for this highly specialized treatment. To learn more, please call 1-833-UPMC-CART.

Patients who are approved for CAR T-cell therapy will undergo the following treatment process:

To refer a patient for evaluation for one of these clinical trials, please call 1-833-876-2227 (1-833-UPMC-CART).

If you think you might be a candidate for one of these clinical trials, please call 1-833-876-2227 (1-833-UPMC-CART).

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FDA-approved CAR T-cell Therapies | UPMC Hillman

Duchenne muscular dystrophy (DMD) is the most common and serious form of muscular dystrophy. One out of every 3500 boys is born with the disorder, and it is invariably fatal. Until recently, there was little hope that the widespread muscle degeneration that accompanies this disease could be combated.

However, stem cell therapy now offers that hope. Like other degenerative disorders, DMD is the result of loss of cells that are needed for correct functioning of the body. In the case of DMD, a vital muscle protein is mutated, and its absence leads to progressive degeneration of essentially all the muscles in the body.

To begin to approach a therapy for this condition, we must provide a new supply of stem cells that carry the missing protein that is lacking in DMD. These cells must be delivered to the body in such a way that they will engraft in the muscles and produce new, healthy muscle tissue on an ongoing basis.

We now possess methods whereby we can generate stem cells that can become muscle cells out of adult cells from skin or fat by a process known as reprogramming. Reprogramming is the addition of genes to a cell that can dial the cell back to becoming a stem cell. By reprogramming adult cells, together with addition to them of a correct copy of the gene that is missing in DMD, we can potentially create stem cells that have the ability to create new, healthy muscle cells in the body of a DMD patient. This is essentially the strategy that we are developing in this proposal.

We start with mice that have a mutation in the same gene that is affected in DMD, so they have a disease similar to DMD. We reprogram some of their adult cells, add the correct gene, and grow the cells in incubators in a manner that will produce muscle stem cells. The muscle stem cells can be identified and purified by using an instrument that detects characteristic proteins that muscles make.

The corrected muscle stem cells are transplanted into mice with DMD, and the ability of the cells to generate healthy new muscle tissue is evaluated. Using the mouse results as a guide, a similar strategy will then be pursued with human cells, utilizing cells from patients with DMD. The cells will be reprogrammed, the correct gene added, and the cells grown into muscle stem cells. The ability of these cells to make healthy muscle will be tested by injection into mice with DMD that are immune-deficient, so they will accept a graft of human cells.

In order to make this process into something that could be used in the clinic, we will develop standard procedures for making and testing the cells, to ensure that they are effective and safe. In this way, this project could lead to a new stem cell therapy that could improve the clinical condition of DMD patients. If we have success with DMD, similar methods could be used to treat other degenerative disorders, and perhaps even some of the degeneration that occurs during normal aging

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Stem Cell Therapy for Duchenne Muscular Dystrophy ...

DUBLIN, October 05, 2021--(BUSINESS WIRE)--The "Global Gene and Cell Therapy (GCT) Market - Analysis By Vector, Application, By Region, By Country (2021 Edition): Market Insights, Pipeline, Forecast with Impact of COVID-19 (2021-2026)" report has been added to ResearchAndMarkets.com's offering.

The Global Gene and Cell Therapy (GCT) Market is estimated at USD 2504.2 Million in the year 2020.

Growth in the historic period in the cell and gene therapy market resulted from increase in investments in cell and gene therapies, growth in research and development, advances in cancer drug discovery, rise in public-private partnerships, strong economic growth in emerging markets, increased healthcare expenditure, and rising in pharmaceutical R&D expenditure.

Companies in the gene and cell therapy for oncology market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises.

While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

Further, the market was restrained by inadequate reimbursements, challenges due to regulatory changes, low healthcare access, and limited number of treatment centers.

Going forward increasing prevalence of cancer and chronic diseases, rising geriatric population, rising geriatric population, rising focus on cell and gene therapy, and rise in healthcare expenditure will drive the growth in the gene and cell therapy market.

Factors that could hinder the growth of the market in the future include high costs of therapy, stringent regulations, reimbursement challenges, and coronavirus pandemic.

The report tracks competitive developments, strategies, mergers and acquisitions and new product development. The companies analysed in the report include F. Hoffman-La Roche Ltd., Novartis, Sanofi, Alnylam Pharmaceuticals Inc., Pfizer, BlueBird Inc., Sarepta Therapeutics, Voyager Therapeutics, Orchard Therapeutics Plc, AnGes Inc.

Story continues

Key Topics Covered:

1. Report Scope and Methodology

1.1 Scope of the Report

1.2 Research Methodology

1.3 Executive Summary

2. Strategic Recommendations

3. Gene and Cell Therapy Market: Product Outlook

4. Global Gene and Cell Therapy Market: Sizing and Forecast

4.1 Global Gene and Cell Therapy Market Size, By Value, Year 2016-2026

5. Global Gene and Cell Therapy Market Segmentation - By Vector, By Application

5.1 Competitive Scenario of Global Gene and Cell Therapy Market: By Vector

5.1.1 Lentivirus - Market Size and Forecast (2016-2026)

5.1.2 AAV - Market Size and Forecast (2016-2026)

5.1.3 Retrovirus & Gammaretrovirus - Market Size and Forecast (2016-2026)

5.1.4 Others - Market Size and Forecast (2016-2026)

5.2 Competitive Scenario of Global Gene and Cell Therapy Market: By Application

5.2.1 Oncology - Market Size and Forecast (2016-2026)

5.2.2 Neurological Disorders - Market Size and Forecast (2016-2026)

5.2.3 Cardiovascular disorders - Market Size and Forecast (2016-2026)

5.2.4 Others - Market Size and Forecast (2016-2026)

6. Global Gene and Cell Therapy Market: Regional Analysis

6.1 Competitive Scenario of Global Gene and Cell Therapy Market: By Region

7. North America Gene and Cell Therapy Market: An Analysis (2016-2026)

7.1 North America Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value

7.2 North America Gene and Cell Therapy Market - Prominent Companies

7.3 Market Segmentation By Vector (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others)

7.4 Market Segmentation By Application (Oncology, Neurological Disorders, cardiovascular disorders and Others)

7.5 North America Gene and Cell Therapy Market: Country Analysis

7.6 Market Opportunity Chart of North America Gene and Cell Therapy Market - By Country, By Value, 2026

7.7 Competitive Scenario of North America Gene and Cell Therapy Market: By Country

7.8 United States Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value

7.9 United States Gene and Cell Therapy Market Segmentation - By Vector, By Application (2016-2026)

7.10 Canada Gene and Cell Therapy Market: Size and Forecast (2016-2026), By Value

7.11 Canada Gene and Cell Therapy Market Segmentation - By Vector, By Application (2016-2026)

8. Europe Gene and Cell Therapy Market: Segmentation: An Analysis (2016-2026)

9. Asia Pacific Gene and Cell Therapy Market:: An Analysis (2016-2026)

10. Global Gene and Cell Therapy Market Dynamics

10.1 Drivers

10.2 Restraints

10.3 Trends

11. Market Attractiveness

11.2 Market Attractiveness Chart of Global Gene and Cell Therapy Market - By Vector, 2026

11.3 Market Attractiveness Chart of Global Gene and Cell Therapy Market - Application, 2026

11.4 Market Attractiveness Chart of Global Gene and Cell Therapy Market - By Region, 2026

12. Competitive Landscape

12.1 Major Technological Innovations, Mergers & Acquisitions and Role of Manufacturers

12.2 Product Pipeline of Leading Gene Therapy Companies

12.3 Market Share Analysis

13. Company Analysis

13.1 F. Hoffman-La Roche Ltd.

13.2 Novartis

13.3 Sanofi

13.4 Alnylam Pharmaceuticals Inc.

13.5 Pfizer

13.6 BlueBird Inc.

13.7 Sarepta Therapeutics

13.8 Voyager Therapeutics

13.9 Orchard Therapeutics Plc

13.10 AnGes Inc.

For more information about this report visit https://www.researchandmarkets.com/r/mw6ql7

View source version on businesswire.com: https://www.businesswire.com/news/home/20211005005880/en/

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Global Gene and Cell Therapy (GCT) Market 2021-2026: Insights, Pipeline, Forecast with Impact of COVID-19 - ResearchAndMarkets.com - Yahoo Finance

The $85 million Iovance Cell Therapy Center (ICTC) has opened its doors at the Philadelphia Navy Yard with capacity to supply thousands of patients per year.

Iovance celebrated the opening of its 136,000 square-foot commercial-scale production facility, commissioned in 2018 at a cost of around $85 million, this week.

Since Iovance was founded, we have been dedicated to advancing novel cell therapies for patients with solid tumor cancers, Iovance CEO Frederick Vogt said.

Image: Stock Photo Secrets

A little over two years after breaking ground, iCTC is now one of the largest cell therapy manufacturing facilities in the world and may ultimately house hundreds of employees.

He added: We now have the capacity to supply broad access to TIL therapies for patients.

Iovance is developing several tumor infiltrating lymphocyte (TIL) cell therapies for various cancers. Lead product lifileucel has US FDA regenerative medicine advanced therapy (RMAT) designation and is in pivotal trials for both melanoma and cervical cancer.

Its program LN-145 is looking at using lifileucel in patients with metastatic non-small cell lung cancer, and the first clinical batch has been successfully manufactured and delivered from the new facility, the firm said.

Moving forward, we are diversifying between internal and external TIL manufacturing for clinical studies, and iCTC remains on track to provide commercial supply upon potential product approval, COO Igor Bilinsky said. Establishing our internal manufacturing capabilities is a top priority at Iovance to ensure broad access to and reduce the costs of Iovance TIL cell therapy.

Iovance has previously inked deals with contract development and manufacturing organizations (CDMOs) for clinical supply of its TILs, specifically WuXI Apptec which produces TILs from its Commerce Center 3 facility within WuXi Advanced Therapies cell and gene therapy site in the Philadelphia Navy Yard.

TILs are based on a 22-day process. This involves the isolation of TIL cells from a patients tumor and then expanding them by stimulating them ex vivo. The cells are fragmented in a minimalized cell culture system for the first 11 days before rapid expansion begins. On day 16 the cells are split into multiple flasks, and day 22 they are harvested.

However, the firm has looked to the opening of the Philadelphia, Pennsylvania plant as an opportunity to improve the process further and reduce costs.

In January 2020, Michelle Simpson-Abelson, then principal scientist at Iovance, said: The thought process of having our own manufacturing facility and being able to tweak the process is to allow it to be as accessible to as many patients as we can.

Original post:
Iovance opens cell therapy plant; makes first batch ...

CARLSBAD, Calif., Oct. 5, 2021 /PRNewswire/ -- Thermo Fisher Scientific today announced the launch of Gibco Cell Therapy Systems (CTS) NK-Xpander Medium, a GMP-manufactured cell culture medium that supports large-scale growth and culture of functional natural killer (NK) cells with or without the use of feeder cells. This is the first medium from Thermo Fisher specifically designed to support expansion of NK cells for cell therapy applications and is supported by raw material traceability and regulatory documentation.

"Cell therapy developers are increasingly turning to NK cells because they do not elicit the kinds of active immune responses that trigger conditions such as graft versus host disease," said Mark Powers, vice president, research and development at Thermo Fisher Scientific. "With NK-Xpander Medium, manufacturers can reach the necessary scale they need for NK cell therapies while minimizing regulatory burden and risk."

NK cells grown in NK-Xpander Medium exhibit a greater rate of expansion when compared to NK cells grown in other media and demonstrate cell killing in in vitro and in vivo models. In addition to their low immunogenicity, the ease of availability and sourcing of NK cells, coupled with efficient in vitro expansion, make them ideally suited for the development of allogeneic cell therapies. Unlike autologous therapies, which are produced using a patient's own cells, allogeneic cell therapies are derived from healthy donor tissue, and are more conducive to cost-effective scale-up of cell therapy manufacturing.

"NK cell therapies hold promising applications in treating solid tumors, which haven't been well served by therapies derived from other cell types," said Richard Eckert, professor and chairman at University of Maryland School of Medicine. "To capitalize on the promise of these therapies, our lab used human NK cells grown in Gibco CTS NK-Xpander Medium to study their impact on solid tumor-derived cancer cells. The NK cells cultured in NK-Xpander Medium displayed robust and potent cancer cell killing activity."

NK-Xpander Medium is part of Thermo Fisher's CTS product line, a comprehensive portfolio of GMP-manufactured products backed by regulatory documentation and designed to work synergistically, from cell isolation/activation to gene transfer and cell expansion, to address cell therapy developers' manufacturing workflow needs.

To learn more about Thermo Fisher's cell and gene therapy solutions, please visit http://www.thermofisher.com/CGT. To learn more about Gibco CTS NK-Xpander Medium, please visit http://www.thermofisher.com/nkcelltherapy.

* For Research Use or Manufacturing of Cell, Gene, or Tissue-Based Products. This product is not intended for direct administration into humans or animals.

About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $35 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 90,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visitwww.thermofisher.com.

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New Culture Medium Supports Expansion of Natural Killer Cells for Cell and Gene Therapies - PRNewswire

Research and development of stem cell-based therapies, where a patients own cells, or those from a donor, are used to fight injury and disease, is one of the fastest growing areas in the biotech space. Longeveron Inc. (NASDAQ: LGVN), a clinical-stage biotechnology company in the thick of clinical development, continues to advance its investigational therapeutic, Lomecel-B, for chronic, aging-related and life-threatening conditions.

The company recently announced the results of its randomized, blinded and placebo-controlled Phase 2 trial to evaluate the safety and efficacy of its proprietary Lomecel-B infusion in frail, older patients between 70 and 85 years old. The trial, which was partially funded by the National Institute on Aging, evaluated a single intravenous infusion of 4 different doses of Lomecel-B cell therapy compared to placebo on the change in the distance a person could walk in 6 minutes (a test known as the 6-minute walk test). Results showed that a single infusion of Lomecel-B resulted in an increase in walk distance of approximately 50 meters (164 feet) at 6 and 9 months after infusion, while the placebo-treated subjects showed minimal improvement at 6 months and a deterioration by 9 months.

Lomecel-B is a proprietary allogeneic product comprised of medicinal signaling cells (MSCs) from the bone marrow of adult donors, which are culture-expanded in Longeverons current good manufacturing practice cell-processing facility. According to trial results so far, Lomecel-B, and MSCs in general, may be injected or infused into an unrelated recipient without triggering a harmful reaction (rejection) due to the biochemical properties of these specialized cells. This is in part what makes this class of biologic so intriguing for use as a regenerative therapeutic.

A growing global trend is for biotech companies to direct their services to the cell and gene therapy industry and moving to expand into a new branch of the pharmaceutical contract development and manufacturing organization world.

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The U.S. Food and Drug Administration (FDA) has approved a small number of cell and gene therapy drugs. Still, a new product pipeline is fighting for the agencys attention with approximately 1,200 experimental therapies more than half in Phase 2 clinical trials. The annual sales growth estimates for cell therapies are projected to reach 15%.

Longeveron has also initiated a Phase 2 trial evaluating the safety and efficacy of Lomecel-B injection into the heart of children born with hypoplastic left heart syndrome (HLHS), a rare and often fatal congenital heart defect.

Longeveron believes that using the same cells that promote tissue repair, organ maintenance and immune system function can develop safe and effective therapies for some of the most challenging diseases and conditions associated with aging.

We continue to make steady progress advancing our Lomecel-B clinical research programs forward, Longeveron CEO Geoff Green said. We have encouraging top-line results from our Aging Frailty program, and anticipate initiating a Phase 2 trial in Alzheimers disease later this year.

Longeveron shared their review of the Aging Frailty trial data with independent frailty experts, with the objective of planning the next steps for the program. The company presented clinical data at the 2021 International Conference on Frailty & Sarcopenia Research on Sept. 29 during a round-table presentation.

Learn more about Longeveron at http://www.longeveron.com.

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With a compound annual growth rate (CAGR) of 34.2%, the global CAR-T cell therapy market is projected to hit $15.4 billion by 2028. This hypergrowth is largely driven by the incredible promise of CAR-T cell therapy as an effective, lasting way to fight cancer. Historically, however, that growth has been hindered by obstacles to developing a fast, affordable and low side-effect version of the treatment.

Avalon GloboCare (NASDAQ: AVCO), a clinical-stage biotech company focused on immunotherapy, diagnostics and therapeutics, may have found the breakthrough researchers have been looking for the past decade with its leading drug candidate, AVA-011, an mRNA CAR-T platform that solves the key obstacles blocking the revolutionary cell therapy from becoming more widely available.

Heres why CAR-T therapy has taken the biotech industry by storm, and why Avalon is optimistic that AVA-011 could finally offer a safe, affordable version of the treatment to patients around the world.

What Is CAR-T Cell Therapy?

CAR-T cell therapy is an exciting cancer treatment thats made huge waves in the biotech community over the last 10 years. Using a blood sample from a patient, developers can isolate the patients T cells, reengineer them with chimeric antigen receptors (CAR) and then reintroduce those cells into the patients bloodstream through an infusion.

T cells are the workhorses of our immune system. They systematically destroy foreign substances that could cause harm. The reengineered CAR-T cells offer two benefits on top of what T cells already do. First, the CAR molecule acts as a kind of molecular GPS guiding the T cell to the tumor site. Second, the CAR molecule itself is an added weapon that can bind to the signature tumor it was engineered to target and trigger an immune-driven cancer killing effect.

When the infusion is made it acts as a living drug, multiplying and remaining in the body to suppress any recurrence of the cancer it was engineered to target.

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In human trials of various versions of CAR-T cell therapy, more than 80% of patients saw a complete remission or a partial response for their cancer. While long-term studies are scarce because the treatment is still so new, one 5-year study found that every single patient who showed a full or partial response to the CAR-T cell therapy maintained that response five years later meaning that those with a complete response were still cancer-free and those with a partial response saw no progression of the disease to worse stages.

While the results of CAR-T cell therapy are nothing short of impressive, key challenges have prevented it from becoming more widespread. Above all, theres a high risk of adverse reactions. Cytokine Release Syndrome (CRS) is the most common reaction, causing fever and hypotension in 13% to 49% of patients, depending on which treatment was used.

Moreover, designer CAR-T cells like those used in this powerful new treatment are difficult and expensive to produce. The conventional protocol takes 14 days from vein to vein that is, from the time its extracted from the patient to the time its reengineered and delivered to the patient again via infusion. It also requires a disarmed viral vector to deliver the CAR molecule to the T cell. This adds additional cost and time to the manufacturing of the treatment. On average, a single infusion produced using this conventional protocol costs $500,000 for the patient.

Avalons FLASH-CAR Platform Addresses Key Challenges in CAR-T Cell Therapy

To overcome these obstacles, Avalon developed its proprietary FLASH-CAR platform. Using messenger RNA (mRNA), the platform is able to bypass the need for a viral vector to introduce the CAR molecule to the T cell. That bypassing shortens the vein to vein time to just 1 to 2 days and significantly reduces the cost of manufacturing.

Moreover, the mRNA used is modified to come with a safety switch. If a patient experiences CRS or another adverse reaction, Avalon has developed an FDA-approved antidote that can target those CAR-T cells and deactivate them, stopping the adverse reaction. This built-in safety switch is a game changer that gives patients and their healthcare providers more control over their cancer treatments.

The mRNA also enhances the living drug-effect by signaling a more rapid proliferation of CAR-T cells. This could potentially make the treatment more effective and offer even more long-term protection for patients.

Finally, mRNA contains functional units that allow manufacturers to activate other immune systems cell types as well, including natural killer cells and dendritic cells. This ability to engineer more than just T cells expands the potential of the cell therapy beyond the domain of blood cancers the only kinds of cancers the T cell-based infusions have currently been able to target successfully.

AVA-011 Prepares for First Human Clinical Trials

Avalons leading FLASH-CAR drug candidate is AVA-011, which is indicated to treat B-cell acute lymphoblastic leukemia and non-Hodgkin lymphoma (both blood cancers). With the potential of the FLASH-CAR platform to engineer other immune cell types, Avalon is working to develop other drug candidates that could target solid tumors. The promising new treatment has completed preclinical testing and slated to begin its first human clinical trials in 2022.

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Avalon GloboCare Aims to Revolutionize Cell Therapy Market with a Faster, Safer CAR-T Treatment - Yahoo Finance

Oral presentation to discuss new data updates from the high- and low-dose cohorts from the ongoing Phase 1/2 trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis

NEW YORK and DURHAM, N.C., Oct. 04, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that it will present new clinical and preclinical data in two oral presentations and one poster presentation at the upcoming European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, to be held virtually from October 19-22, 2021.

Oral presentations will include an update on the Phase 1/2 trial of AXO-AAV-GM1, the companys adeno-associated viral vector (AAV)9-based gene therapy for the treatment of Type I (early infantile onset) and Type II (late infantile and juvenile onset) GM1 gangliosidosis. Presentation will include new data from the low- and high-dose cohorts. The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Oral Presentation Details:

Presentation Title: Phase 1/2 Trial of AXO-AAV-GM1 Gene Therapy for the Treatment of Infantile- and Juvenile-onset GM1 GangliosidosisPresentation Number: OR28Session: Session 4a: CNS & Sensory IIPresenting Author: Erica De Boever, Ph.D., DDS, MPH, Vice President of Clinical Development at Sio Gene TherapiesPresentation Date and Time: Thursday October 21, 2021; 9:00-11:00 AM CEST

Presentation Title: Bicistronic AAV Gene Therapy for Tay-Sachs and Sandhoff Diseases in a Sheep ModelPresentation Number: OR30Session: Session 4a: CNS & Sensory IIPresenting Author: Toloo Taghian, Ph.D., University of MassachusettsPresentation Date and Time: Thursday, October 21, 2021; 9:00-11:00 AM CEST

Poster Presentation Details:

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Presentation Title: Phase 1/2 Open-label Dose Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinsons Disease: Efficacy, Safety, and Tolerability Data up to 24 MonthsPoster Number: P254Presenting Author: Gavin Corcoran, MD, Chief R&D Officer of Sio Gene Therapies

Copies of the presentation materials will be made available under the Events and Presentations section of Sios website.

About AXO-AAV-GM1AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the Food and Drug Administration and is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis. In 2018, Sio licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About AXO-AAV-GM2AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors.

About AXO-Lenti-PDAXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinsons disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration. Axovant expects to dose the first patient in EXPLORE-PD, a randomized, sham controlled study in 2021.

About Sio Gene TherapiesSio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "expect," "estimate," "may" and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing and outcome of its upcoming clinical and manufacturing milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing; the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 12, 2021, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and AnalystsParag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officer investors@siogtx.com

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Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021 - Yahoo Finance