By Alycea Wood and Kamran Zamanian, Ph.D., iData Research Inc.

When choosing a treatment option for orthopedic procedures, biomaterials have become widely popular. Biomaterials are biomedical materials that can be safely implanted or injected into the body and are, more often than not, a form of biologically active tissue themselves.1 Their prevalence in orthopedic procedures is largely attributed to their ability to mimic the structure or properties of osseous tissue. Many products can offer a number of beneficial properties, such as promoting bone growth within the body (osteoinduction), promoting bone growth on the biomaterials scaffold (osteoconduction), or inducing the differentiation of stem cells into osseous tissue (osteogenesis).2,3 The orthopedic biomaterials market includes bone graft substitutes, growth factors, cellular allografts, cell therapy, hyaluronic acid viscosupplementation, and even cartilage repair devices. The U.S. orthopedic biomaterials market saw a dramatic dip and subsequent rebound in market value in 2020 and 2021 as a result of the COVID-19 pandemic. After recovery, the market is projected to see a consistently steady growth in value within the next few years. This growth is expected to be seen across all market segments apart from cellular allograft devices (Figure 1).4

Figure 1: Orthopedic biomaterials market growth trends by market segment, U.S., 20192028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

Cellular allografts may consist of either allograft bone (donated bone tissue) in conjunction with adipose-derived adult stem cells or viable cells within a cortical cancellous bone matrix.4,5 In both scenarios, the devices provide osteoconduction, osteoinduction, and osteogenesis to the site of implantation. Historically, this market had seen promising growth because of the optimal environment for bone growth they can provide.6 The cellular allograft market is projected to see a much slower rate of growth in market value in the next few years despite its market potential due to increased constraints on the market itself. These include, but are not limited to, direct federal restriction on product research, cost of product development, and product recalls.4

There is a strong interest in the scientific community in embryonic stem cell (ESC) research, which is largely due to ESCs high differentiability when compared to adult stem cell (ASC) lines.7 The development of new cellular allograft products, and the resulting growth in the market, is dependent on continued research into realizing the full medical potential of stem cell use. In 2019, the Trump administration eliminated federal funding of research relying on ESC tissue and instituted the National Institutes of Health (NIH) Human Fetal Tissue Research Ethics Advisory Board. This negatively impacted a large number of studies in progress while restricting the ability of new projects to commence.8,9,10 While the board was in effect, it rejected all but one application for funding.11 In April 2021, the Biden administration removed both the board and the restrictions on current projects, allowing federally funded research using ESC to continue.12 This was not the first instance where restrictions were placed and then removed on ESC research. In March 2009, President Obama signed an executive order to overturn the Bush administrations restriction on ESC research.13

The repeated restrictions on ESC research have a number of long-term ramifications in the development and implementation of new, effective cellular allograft treatments. Scientists may need to divert their research efforts away from stem cells and into less turbulent fields, and the progress of product development slows down as studies have funding pulled; this may contribute to increased hesitancy by end users to use stem cell products. Reduced research efforts, funding, and faith in stem cell products will continue to limit the growth of the cellular allograft market.

Cellular allografts tend to be notably more expensive than others within the broader cell-based biomaterials market. When compared to the cell therapy market, which uses either concentrated platelet-rich plasma (PRP) or bone marrow aspirate concentrate (BMAC) in its treatment, the cellular allograft average selling price (ASP) sits over three times higher (Figure 2).3

Figure 2: The average selling price (ASP) of the cellular allograft & cell therapy markets, U.S., 20182028. Access iDatas U.S. Orthopedic Biomaterials report to view more granular data.

The ASP of the cellular allograft market is so high because of the prohibitively expensive cost of developing new products. During the development process, reliable efficacy of a new product is uncertain, and using protein markers to help distinguish stem cell types can be very challenging.4,14 The increased cost of product development acts as a significant barrier to parties looking to enter the U.S. cellular allograft market. The result is fewer products entering and rejuvenating the market, and existing products sit at prohibitively high prices as they have low direct competition.4 The high cost of cellular allograft products hinders new entrants from introducing products and prevents end users from being able to afford existing ones. A broader consequence of this is end users turning to more affordable orthopedic biomaterial types to reduce procedural costs.

Any product recalls within the U.S. orthopedic biomaterials market, especially within cell-based therapies, will negatively impact the use of cellular allografts. This impact is amplified when a recall occurs within the market segment itself, which was seen in the cellular allograft market as recently as June 2021. On June 2, 2021, Aziyo Biologics recalled its product FiberCel following a number of patients contracting tuberculosis.15 Recalls deter the use of cell-based products through increased distrust in the safety of the products themselves, potential public backlash against the specific product itself or, in the market more broadly, reduced reimbursement from health insurance providers as well as the introduction of more restrictive FDA protocols. This is another reason why effective, safe, cell-based products are necessary for the cellular allograft market to move forward.

Conclusion

Federal research restrictions, high development costs, and product recalls all negatively impact the growth of the cellular allograft market in the United States. These constraints contribute to the projected low growth rate in market value in the coming years despite the potential uses for stem cell therapies. To shift the tide back toward growth, the cellular allograft space will need consistent research progress through large-scale studies, more affordable product development, and strict enforcement of sanitization protocols for existing products to prevent future product recalls. The large therapeutic potential of stem cell therapy has been discussed extensively in scientific and popular literature, but it may take a while to realize it.

References

About The Authors:

Alycea Wood is a research analyst at iData Research. She develops and composes syndicated research projects regarding the medical device industry, and published the U.S. Orthopedic Biomaterials report series.

Kamran Zamanian, Ph.D., is CEO and founding partner of iData Research. He has spent over 20 years working in the market research industry with a dedication to the study of medical devices used in the health of patients all over the globe.

About iData Research

For 16 years, iData Research has been a strong advocate for data-driven decision-making within the global medical device, dental, and pharmaceutical industries. By providing custom research and consulting solutions, iData empowers its clients to trust the source of data and make important strategic decisions with confidence.

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The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online

The first cell therapies made from a patients own cells were cancer treatments T cells engineered to hit tumors. Immusoft is taking a similar approach with a different type of immune cell, aiming to deliver these cell therapies into the brain. The biotech startup is still preclinical, but Takeda Pharmaceutical sees enough promise to begin a research and development alliance that could yield new treatments for rare neurometabolic diseases.

Seattle-based Immusoft develops its treatments by reprogramming B cells, a type of white blood cell. The process for making this treatment is nearly identical to that of CAR T, the first autologous cancer cell therapies. Blood is collected from the patient and the desired immune cells are selected. Those cells are engineered, then multiplied in a lab. After the process produces enough cells, that treatment is infused into the patient.

Besides the type of cell that is used, the key difference between CAR T and Immusofts approach is the engineering step. In CAR T, this stage involves engineering T cells to recognize a particular antigen on tumors. For Immusoft, this step means programing B cells with DNA that enables them to produce large amounts of therapeutic protein. Immusoft calls its technology Immune System Programing, or ISP.

Takeda and Immusoft did not specify the diseases they aim to address under their new alliance, though the companies said the collaboration will focus on delivering therapies across the blood-brain barrier, the protective layer that keeps certain substances, including some drugs, from reaching the organ. By reaching the brain, these B cell therapies have the potential to address a range of neurological disorders.

We continue to build our internal capabilities as well as partner with innovative companies early on in the discovery process to advance our next-generation gene and cell therapy ambitions for rare genetic and hematologic diseases, Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan said in a prepared statement. Working together with Immusoft, we hope to validate their ISP technology for [central nervous system] delivery of innovative therapeutics for rare neurometabolic diseases.

Rare neurometabolic disease is already one of the areas of focus for Immusoft. The rare enzyme deficiency Hurler syndrome is the target for its most advanced internal program, ISP-001. That cell therapy candidate is on track for an investigational new drug application filing by the end of this year, according to the companys website.

According to deal terms announced Wednesday, Takeda will pay Seattle-based Immusoft an upfront payment as well as research funding. The specific amounts were not disclosed. When the drug candidates covered under the partnership reach preclinical development, Takeda may elect to choose an unspecified number of them to continue their development. The Japanese pharma giant would owe option fees plus milestone payments tied to the progress of those programs. The companies gave no specific breakdown of those payments, other than to say the total value could top $900 million if all options are exercised and all milestones are achieved.

Working with B cells offer several advantages over other approaches to treating disease. Gene therapies delivered via engineered viruses cant be re-dosed because the antibodies that patients develop to the virus will render subsequent doses ineffective. By using a patients own cells, Immusoft aims to produce therapies that can be dosed multiple times. Also, gene therapies made by collecting a patients stem cells require a preconditioning step that knocks out the immune system to make room for the transplanted stem cells to grow. This step opens the door to a range of potential complications. Takedas recent dealmaking shows the pharma giants interest in avoiding therapies that employ viral delivery. In unveiling a multi-program alliance with Poseida Therapeutics on Tuesday, the pharma giant cited the biotechs non-viral technologies.

There are other companies developing ways to engineer B cells into new therapies. South San Francisco-based Walking Fish Therapeutics unveiled a $50 million Series A round of funding last month to support its research, still preclinical. Be Biopharma of Boston emerged nearly a year ago with a $52 million Series A financing.

Immusofts research so far has produced a preclinical drug pipeline spanning both rare and common diseases. Besides the Hurler syndrome candidate, the other rare disease programs include potential treatments for muscle-wasting disorders amyotrophic lateral sclerosis and Duchenne muscular dystrophy; the metabolic disease Hunter syndrome; and Pompe and Gaucher diseases, both enzyme deficiency disorders. Immusofts common disease research is still in the discovery stage. For common diseases, Immusoft is developing treatments for cardiovascular disorders, rheumatoid arthritis, and Parkinsons disease.

Image by Jolygon via Getty Images

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Taking aim at the brain, Takeda strikes up cell therapy R&D alliance with Immusoft - MedCity News

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapyPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR049Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-SequencingPresenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: OR058Date/Time: Friday, October 22, 2021 at 12:46 CEST

Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD)Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, ItalyAbstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product ManufacturePresenting Author: Saranya Elavazhagan, Orchard TherapeuticsAbstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIAPresenting Author: Brian Bigger, Ph.D., University of ManchesterAbstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsPresenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease backgroundPresenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyAbstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapyPresenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapyPresenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene TherapyAbstract Number: P186

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaBenjamin NavonDirector, Corporate Communications+1 857-248-9454Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the - GlobeNewswire

15 October 2021

Perkins Coie LLP

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Key Takeaways:

On July 1, 2021, the FTC adopted aseries ofresolutionsauthorizing investigations into several areasdesignated as key law enforcement priorities during the next tenyears.One of thoseresolutionsdesignates "acts or practices affectinghealthcare markets" as an enforcement priority.

In the months after adopting this broad mandate, the FTC hasactively pursued false and deceptive advertising in healthcaremarkets.

For example, on August 16, 2021, the FTC and the GeorgiaAttorney Generalsuedthe co-founders ofthe Stem Cell Institute of America for false marketing inadvertising stem cell therapy to seniors. Specifically, thedefendants advertised their stem cell therapies as effectivelytreating orthopedic conditions and diseases. The FTC and theGeorgia Attorney General alleged that the defendants neverconducted any randomized clinical testing of the treatments tosupport these claims and that no other studies exist demonstratingthat stem cell treatments are effective in treating any orthopedicconditions or diseases.

Similarly, in early September 2021, theFTC sent cease-and-desistlettersto ten different companies demanding that they,among other things, stop selling unapproved diabetes products andmaking efficacy claims that are not supported by sufficientevidence.

These two actions, coming on the heels of the resolutionprioritizing enforcement in the healthcare industry, may portendaggressive FTC policing of healthcare-related advertising.Accordingly, companies offering healthcare services orproductsespecially those offering treatmentsshouldensure that their advertisements are backed up by competent andreliable scientific evidence.

The content of this article is intended to provide a generalguide to the subject matter. Specialist advice should be soughtabout your specific circumstances.

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