Stem Cell Clinic

Cell Therapy Manufacturing Market (2017-2027): Near-Term Demand Driven by Clincial Candidates – Research and … – PR Newswire (press release)

The Cell Therapy Manufacturing Market, 2017-2027 report provides an extensive study of the rapidly growing market of cell therapy manufacturing and focuses both on contract manufacturers and cell therapy developers with in-house manufacturing facilities. These therapies are anticipated to emerge as viable alternatives to conventional treatment options.

The scope of this report primarily includes manufacturing of advanced therapy medicinal products (ATMPs) that involve the use of immune cells such as T-cells, Tregs, dendritic cells, tumor cells and NK cells, and stem cells such as adult stem cells, human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs).

Several players, including cell therapy developers, research institutes, contract manufacturing organizations, and government and non-profit organizations, are playing a critical role in the development and manufacturing of these cell therapies. In fact, a number of these players have made heavy investments to expand their existing capabilities and establish new facilities for cell therapy products in order to meet the increasing demand.

Additionally, stakeholders have received significant support from governments worldwide, in terms of funding and establishment of consortiums to accelerate the transition of these therapies from laboratories to clinics. It is important to highlight that companies that offer logistics and operational services have developed systems / tools for safer and quicker delivery of therapies from manufacturing sites to patients; this has been identified as one of the key challenges in the overall development process.

The near-term demand for manufacturing of cell-based therapies will primarily be driven by clinical candidates. In the longer term, the currently approved therapies and late-stage therapies (that are likely to get commercialized in future) will act as key drivers of the market. Our outlook is highly promising; we expect the market for cell therapy manufacturing to grow at an annualized growth rate of -42% over the course of next ten years and be worth over USD 4 billion in 2027.

Companies Mentioned

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. CELL THERAPY MANUFACTURING: INTRODUCTION

4. MARKET OVERVIEW

5. ROADMAPS: POTENTIAL STRATEGIES TO OVERCOME EXISTING CHALLENGES

6. CELL THERAPY MANUFACTURING: IN-HOUSE MANUFACTURERS

7. CELL THERAPY MANUFACTURING: INDUSTRY PLAYERS

8. CELL THERAPY MANUFACTURING: NON-INDUSTRY PLAYERS

9. ROLE OF NON-PROFIT ORGANIZATIONS

10. RECENT DEVELOPMENTS

11. MARKET SIZING AND FORECAST

12. SWOT ANALYSIS

13. CONCLUSION

14. SURVEY ANALYSIS

15. INTERVIEW TRANSCRIPT

For more information about this report visit http://www.researchandmarkets.com/research/z9w7mt/cell_therapy

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Cell Therapy Manufacturing Market (2017-2027): Near-Term Demand Driven by Clincial Candidates - Research and ... - PR Newswire (press release)

Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says – KQED

A small stem cell trial in which patients with severe spinal injuriesappeared to make remarkable progress is still showing excellent results, according to the company conductingthe research.

One of the patients in the trial is 21-year-old Kris Boesen, from Bakersfield, California, whose story we reported on last year.A car crash had left theBakersfield, California native with three crushed vertebrae, almost no feeling below his neck, and a grimprognosis. Doctors believed he would live the rest of his life as a paraplegic.

Enter stem cell therapy. Most treatments for serious spinal injuries concentrate on physical therapy to expand the range of the patients remainingmotor skills and to limit further injury, not to reverse the actual damage. But last April, as part of an experimental phase 2 clinical trial called SCiStar, researchers injected Boesen with 10 million stem cells. By July, hehad recovered use of his hands to the point where he could use a wheelchair, a computer and a cellphone, and could take care of most of his daily living needs.In recent months his progress has continued, says his father.

Boesen is not the only patient to have improved in the trial, according toAsterias Biotherapeutics, which is conducting the research. Boesen is part of a cohort of six patients who were experiencing various levels of paralysis and were injected with the 10 million stem cell dose. In a Jan. 24update, the company saidfive of those patientshad improved either one or twolevels on a widely used scale to measuremotor function in spinal injury patients.

On Tuesday, Asterias issued a newupdate, announcingthat the sixth patient in the cohort has experienced a similar improvement.

While spontaneous recovery for spinal injury patients does occur,the likelihood of all six patients recovering to the degree they haveis less likely, researchers say.

This is as good as you could hope at this point, said Charles Liu, Boesens neurosurgeon and director of the USC Neurorestoration Center. So far all the evidence is pointing in the right direction.

To measure improvement in spinal injury patients, researchers use two yardsticks: the Upper Extremity Motor Scale, or UEMS, and the International Standards for Neurological Classification of Spinal Cord Injury, or ISNCSCI. On the UEMS scale,patients are scored from 0 to 5 on theirability to use five key muscles in the wrists, elbows and fingers. The ISNCSCI scale assesses where damage has occurred along the different levels of the cervical vertebrae, which generally determines the scope of impairment to the body and the level of care needed.

For instance, if a patient has sustained damage at the fourth cervical vertebra down, known as C-4, at the base of the neck, it generally means that person is paralyzed from the neck down, requiring round-the-clock care and a ventilator to breathe.A patient with a C-5 injury may not be able to move her arms or hands, requiring about 6 to 12 hours per day of assisted care; and at the C-6 level, better motor function mayallow a patient to take care of most of herdaily living needs on her own.

Which is all to say that even one level of recovery could substantially improve the daily life ofa spinal injury patient.

According to Asterias, all six patients in the 10million-cell cohort have improved their general UEMS scores, and jumped at least one motor level on the ISNCSCI scale on one or both sides of their body.

Two patients have improvedtwo motor levels on one side; and one patient,Boesen, has improved two motor levels on both sides.

Steve Cartt, president and CEO of Asterias, said anotherpatient, Jake Javier of Danville, California, has gonefrom partial paralysis to being able to use his hands well enough to considerpursuing a computer science career.

Throws Like a Regular Throw

In September, Boesens father, Rod Boesen, told us how excited he wasthat his son had regained some feeling in one of his feet. Last week, at11 months post-injection, the elder Boesensaid Kris has continued to improve.

Now he can move his toe and his knee together at the same time, Boesen said. Theyre about to give him a manual wheelchair now [instead of a motorized one]. He can grip with his hands enough to use a manual one.

Boesen said the movement in his sons arms and hands has greatlyimproved since September.Kris, a formerhigh school pitcher, had beenflinging a ball to his dog like people throw hand grenades, Boesen said. They kind of cradle them and thats how Kris would do it. But now he throws like a regular throw, tosses that ball down the hall, has that release point down, and just wings it.

Asterias is currently recruiting patients for a trial in which theyll receive 20 million stem cells, the optimal dose, according to company researchers. Two patients have already started the 20 million stem cell therapy, and six-month results from those patients will be released in the fall, Cartt said.

Patients who received 2 million stem cells in an earlier phase of the study have not shown much change in their condition, according to the Jan. 24 update.

Guarded Optimism

While Boesens father is impressed with the results, the optimism of researchers inside and outside the studyhas been guarded.The trial is still in its early stages, and the sample size is small, said Paul Knoepfler, a cell biology professor and stem cell researcher at UC Davis, who is not involved in the SCiStar study.

As a scientist, I still would want to wait for more data, Knoepfler said. Its certainly interesting, but its still early. Its a phase 2 trial.

To address the issue of small sample size, Asterias islooking at historical data to determinethe level of improvement for patients in similar circumstances who did not receive stem cell therapy. The company has said it found a meaningful difference in the recovery of its study patients compared to the norm.

Liu said one of the most importantresults is the lack of significant side effects or other negative outcomes resulting from the treatment to date.

Thats very significant to me, Liu said. Thats the first thing you look for, is anyone hurt from this therapy.

There was also a concern, he said, that some patients might regress over time, once the initial injection of stem cells wore off. Thathasyet to occur.

No one has lost anything theyve gained, Liu said. We were very happy to see that. This is all very promising.

The next step for the SCiStartrial will be to establish a control group, Cartt said.

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Strong Progress for Paralyzed Patients After Stem Cell Therapy, Company Says - KQED

Caladrius: ‘Hitachi will unlock full potential of cell therapy CDMO PCT’ – BioPharma-Reporter.com

Hitachi Chemical Co. America will increase its presence in the cell therapy development and manufacturing space through the $75m acquisition of Caladrius Biosciences subsidiary PCT.

The US off-shoot of Japanese firm Hitachi Chemical has entered an agreement to up its position in cell therapy contract development and manufacturing organisation (CDMO) PCT by buying out majority shareholder Caladrius Biosciences for a total of $75m in a deal expected to close in May.

Im delighted to report the entry into an agreement for the acquisition of our remaining 80.1% interest in PCT by Hitachi Chemical, Caladrius CEO David Mazzo said on a conference call to discuss his firms Q4 results this week. This transaction has the potential to unlock the tremendous value of our PCT asset in a way that was unimaginable just a few years ago.

He explained Caladrius has been increasingly challenged by the tens of millions of dollars of additional capital investment need over the next several years for PCT to fully realise its cell therapy commercial manufacturing growth goals, and added Hitachi is in a position to deploy the capital and engineering capabilities needed to achieve these.

Caladrius will continue to use PCT for the manufacturing and development of its own cell therapy candidates including CLBS03, a T-regulatory (Treg) cell-based therapy for the treatment of type 1 diabetes mellitus without the burden of having to try to support and grow that business due to the very, very large capital needs necessary to remain competitive, he told stakeholders.

The deal will include the transfer to Hitachi of cGMP-compliant facilities in Allendale, New Jersey and Mountain View, California offering quality systems, streamlined technology transfer, storage and logistics, and cell and tissue processing services.

In a statement following the announced acquisition, PCT said: Our relationship with our clients does not change based on this announcement, nor will it change based on the finalization of this transaction.

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Caladrius: 'Hitachi will unlock full potential of cell therapy CDMO PCT' - BioPharma-Reporter.com

Failed cell therapy study offers positives, raises new questions – ModernMedicine

Patients with a history of frequent anti-vascular endothelial growth factor (anti-VEGF) injections for the treatment of age-related macular degeneration (AMD) may not be the best potential candidates for encapsulated cell technology (ECT).

ECT contains human cell lines that are capable of producing a variety of proteins. After a single surgical implantation, they can produce proteins for up to two years or more, according to Szilard Kiss, MD, assistant professor of ophthalmology; director of clinical research at Weill Cornell Medical College, New York.

These cell lines survive even after being inside the human eye for six months, Dr. Kiss said.

A phase II study was initiated to compare the third generation of ECT (Neurotech, Cumberland, RI) to aflibercept (Eylea, Regeneron Pharmaceuticals). The study planned to enroll 90 subjects, with a primary outcome of non-inferiority to aflibercept alone after 108 weeks. Among the patient entry criteria, all patients had to have shown a response to aflibercept before randomization.

The patient profile was similar to other anti-VEGF treatment studies, with a best corrected visual acuity (BCVA) between 80 letters (20/25) and 35 letters (20/200) at baseline; limited pathology (such as fibrosis, scarring, or atrophy); good optical coherence tomography (OCT) response to injections; and having gone through at least three previous injections, with the last injection no more than four months before study enrollment.

The goal was to have fewer than 20% of patients needing a rescue injection during the follow-up period, Dr. Kiss said.

Study particulars

Unfortunately, there was not a significant difference between the implanted patients and those that received aflibercept. Those subjects that went on to 24 weeks may appear to have gained a little bit of vision, and one subject seemed to benefit, Dr. Kiss said. The OCT results show a saw-toothed pattern that we can see in patients who are treated with aflibercept every 8 weeks.

Although the responses look impressive, most subjects underwent rescue injections of aflibercept, Dr. Kiss said.

The time to rescue injections actually occurred as soon as 4 weeks after the implantation. And as such, there was early termination of the study, because it was not going to meet the primary endpoint of fewer than 20% needing injections, Dr. Kiss said.

However, the implantation itself was deemed successful, as the cells survived and produced the anti-VEGF molecule, but the amount that was produced was significantly below the 12 g/mL necessary.

There were two outlier subjects who had received an aflibercept injection alone about 10 days before the explant, and before measuring anti-VEGF activity. Dr. Kiss believes that to be the cause of the higher numbers compared to other subjects in the study.

None of the explants who did not receive aflibercept right before the explant produced enough anti-VEGF activity, he said.

Positive take-home

However, there had been indications the technology would be successful for this indicationa patient who had undergone six aflibercept injections during the previous year before enrollment did not need any injections (rescue therapy) during the 28 weeks before the AMD study terminated.

On the positive side, Dr. Kiss said the study results created new questions for the technologyincluding whether the approach could achieve better outcomes compared to real-world experience with other patient populations since the cell viability and stability was good.

The third generation ECT (NCT-503-3) did achieve the goal of improved VEGF-receptor production by at least 2-fold compared to double ECT (NT-503-2) implants. Further, while VEGF levels were not detectable, a complex formation was observed in the preliminary native gel.

While the AMD study has been halted, the company is investigating the technology in an ongoing phase II study in conjunction with the MacTel Project to investigate the long-term delivery of ciliary neurotrophic factor (CNTF) in people with macular telangiectasia (MacTel). A pilot study of neuro-enhancement in subjects with early visual impairment in collaboration with clinicians at Stanford University is also under way.

Most promising is that ECT is a unique and versatile drug delivery platform, with more than 1,000 patient years of safety data, Dr. Kiss added. The long-term continuous release of therapeutic proteins via ECT remains a viable and effective way to treat chronic ocular conditions.

Szilard Kiss, MD

E:[emailprotected]

This article is adapted from a presentation that Dr. Kiss presented at the Retina Subspecialty Day, prior to the 2016 American Academy of Ophthalmology meeting.

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Failed cell therapy study offers positives, raises new questions - ModernMedicine