Category Archives: Gene therapy

DUBLIN, March 21, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Bioassay Services Market by Type of Therapy, Therapeutic Area, Scale of Operation and Geography: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape and the likely future potential of cell and gene therapy bioassay service providers, over the next decade. The study also features an in-depth analysis, highlighting the capabilities of various industry stakeholders engaged in this field.

Given the various advantages of cell and gene therapies and their ability to address the underlying causes of serious clinical conditions, the preference for such therapeutic modalities has increased over the years. As a result, several cell and gene therapies have been developed and approved for the treatment of a wide range of disease indications. In fact, at the time of this report's publication, more than 30 such therapies had been approved and over 1,200 therapeutic leads are under evaluation in different stages.

Owing to their rising popularity, these upcoming advanced therapeutic products are on the verge of becoming one of the highest valued therapeutic segments within the biopharmaceutical industry. Having said that, as is the case for all biological products, the development of cell and gene therapies is a complex, challenging and cost intensive process. Outsourcing is, therefore, a preferred operational model, with cell and gene therapy innovators relying heavily on specialty contract service providers to handle various aspects of their product development and manufacturing operations. In fact, over three quarters of the biopharmaceutical companies prefer to outsource their operations to the contract service providers and a large part of their expenditure goes to outsourcing services.

Amongst the various operations, the outsourcing of analytical testing services, including environment testing, bioanalytical testing, analytical testing, chemistry and stability testing, method development and validation, and product characterization, has witnessed significant rise in recent years.

The rising demand for outsourcing bioanalytical services has prompted the emergence of several contract research organizations that claim to offer bioassay services for cell and gene therapies. These companies usually have relatively more experience and are well equipped with the instruments and technological platforms to offer both pre-developed and customized bioassays.

In fact, these companies assist in bringing out the maximum efficiency of study design by offering several cost benefits and reducing the associated timelines. Given the benefits of outsourcing the bioassay services and the ongoing efforts of service providers to further improve / expand their respective offerings, we believe that the cell and gene therapy bioassay services market is likely to evolve at a steady pace, till 2030.

In addition to other elements, the study includes:

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. MARKET LANDSCAPE

5. COMPANY COMPETITIVENESS ANALYSIS

5.1. Chapter Overview

5.2. Methodology

5.3. Key Parameters

5.4. Competitiveness Analysis: Companies Offering Cell and Gene Therapy Bioassay Services in North America

5.5. Competitiveness Analysis: Companies Offering Cell and Gene Therapy Bioassay Services in Europe and Asia-Pacific

6. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN NORTH AMERICA: COMPANY PROFILES

6.1. Chapter Overview

6.2. CCRM

6.2.1. Company Overview

6.2.2. Cell and Gene Therapy Bioassay Service Portfolio

6.2.3. Recent Developments and Future Outlook

6.3. Nexelis

6.3.1. Company Overview

6.3.2. Cell and Gene Therapy Bioassay Service Portfolio

6.3.3. Recent Developments and Future Outlook

6.4. Pacific BioLabs

6.4.1. Company Overview

6.4.2. Cell and Gene Therapy Bioassay Service Portfolio

6.4.3. Recent Developments and Future Outlook

6.5. PPD Laboratories

6.5.1. Company Overview

6.5.2. Cell and Gene Therapy Bioassay Service Portfolio

6.5.3. Recent Developments and Future Outlook

6.6. WuXi Advanced Therapies

6.6.1. Company Overview

6.6.2. Cell and Gene Therapy Bioassay Service Portfolio

6.6.3. Recent Developments and Future Outlook

7. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN EUROPE AND ASIA-PACIFIC: COMPANY PROFILES

7.1. Chapter Overview

7.2. AnaBioTec

7.2.1. Company Overview

7.2.2. Cell and Gene Therapy Bioassay Service Portfolio

7.2.3. Recent Developments and Future Outlook

7.3. Intertek Pharmaceutical Services

7.3.1. Company Overview

7.3.2. Cell and Gene Therapy Bioassay Service Portfolio

7.3.3. Recent Developments and Future Outlook

7.4. Lonza

7.4.1. Company Overview

7.4.2. Cell and Gene Therapy Bioassay Service Portfolio

7.4.3. Recent Developments and Future Outlook

7.5. Porton Bio

7.5.1. Company Overview

7.5.2. Cell and Gene Therapy Bioassay Service Portfolio

7.5.3. Recent Developments and Future Outlook

8. MARKET TREND ANALYSIS

9. GLOBAL EVENT AND STRATEGIC INITIATIVE ANALYSIS

10. CASE STUDY: MARKET LANDSCAPE OF CELL AND GENE THERAPIES

10.1. Chapter Overview

10.2. Cell Therapy Market

10.2.1. T-Cell Immunotherapies: Development Pipeline

10.2.1.1. Analysis by Type of Product

10.2.2. CAR-T Cell Therapies: Development Pipeline

10.2.2.1. Analysis by Phase of Development

10.2.2.2. Analysis by Therapeutic Area

10.2.2.3. Analysis by Type of Developer

10.2.2.4. Key Industry Players: Analysis by Number of CAR-T Cell Therapies in Development

10.2.3. TCR-based Therapies: Development Pipeline

10.2.3.1. Analysis by Phase of Development

10.2.3.2. Analysis by Therapeutic Area

10.2.3.3. Analysis by Type of Developer

10.2.3.4. Key Industry Players: Analysis by Number of TCR-based Therapies in Development

10.2.4. TIL-based Therapies: Development Pipeline

10.2.4.1. Analysis by Phase of Development

10.2.4.2. Analysis by Therapeutic Area

10.2.4.3. Analysis by Type of Developer

10.2.4.4. Key Industry Players: Analysis by Number of TIL-based Therapies in Development

10.3. Gene Therapy Market

10.3.1. Gene Therapy Market: Clinical and Commercial Pipeline

10.3.1.1. Analysis by Phase of Development

10.3.1.2. Analysis by Therapeutic Area

10.3.2. Gene Therapy Market: Development Pipeline

10.3.2.1. Analysis by Phase of Development

10.3.2.2. Analysis by Therapeutic Area

11. LIKELY PARTNERS FOR CELL THERAPY BIOASSAY SERVICE PROVIDERS

11.1. Chapter Overview

11.2. Scope and Methodology

11.3. Potential Strategic Partners for Cell Therapy Bioassay Service Providers

11.3.1. Likely Partner Opportunities for Stem Cell Therapy Bioassay Service Providers

11.3.2. Likely Partner Opportunities for CAR-T Therapy Bioassay Service Providers

11.3.3. Likely Partner Opportunities for Dendritic Cell Therapy Bioassay Service Providers

11.3.4. Likely Partner Opportunities for Tumor Cell Therapy Bioassay Service Providers

11.3.5. Likely Partner Opportunities for NK Cell Therapy Bioassay Service Providers

12. LIKELY PARTNERS FOR GENE THERAPY BIOASSAY SERVICE PROVIDERS

12.1. Chapter Overview

Excerpt from:
Global Cell and Gene Therapy Bioassay Services Market to 2030 - by Type of Therapy, Therapeutic Area, Scale of Operation and Geography - 69News...

PHILADELPHIA (KYW Newsradio) For more than 40 years, researchers have been looking for a way to cure HIV. Now, that solution may finally be just around the corner.

Temple University researchers launched a clinical trial last month. The treatment removes HIV from infected cells using gene-editing technology, a therapy known as CRISPR.

When you do this CRISPR cleavage, you basically cut the virus on two ends, explained Dr. Tricia Burdo, associate professor in the Department of Microbiology, Immunology and Inflammation at Temples School of Medicine.

You cut a really big piece out, she said. The body has this natural ability to repair its own DNA, so once you remove that HIV from the DNA, your DNA will then come back together and repair itself.

That cut isnt made with a knife or scissors, but rather with molecules and proteins that enter the body during an infusion, which takes about an hour and a half.

Dr. Kamel Kalili, director of Temples Center for Neurovirology and Gene Editing, said the trials were done with animals.

The result of the intravenous inoculation of the CRISPR result in complete elimination of the virus from every single organ that we examined, he said.

And just a few weeks after the infusion, the virus was gone.

Clinical trials on humans are ongoing.

Go here to see the original:
Temple researchers using gene therapy to find a cure for HIV - KYW

DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy Bioassay Services Market by Type of Therapy, Therapeutic Area, Scale of Operation and Geography: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape and the likely future potential of cell and gene therapy bioassay service providers, over the next decade. The study also features an in-depth analysis, highlighting the capabilities of various industry stakeholders engaged in this field.

Given the various advantages of cell and gene therapies and their ability to address the underlying causes of serious clinical conditions, the preference for such therapeutic modalities has increased over the years. As a result, several cell and gene therapies have been developed and approved for the treatment of a wide range of disease indications. In fact, at the time of this report's publication, more than 30 such therapies had been approved and over 1,200 therapeutic leads are under evaluation in different stages.

Owing to their rising popularity, these upcoming advanced therapeutic products are on the verge of becoming one of the highest valued therapeutic segments within the biopharmaceutical industry. Having said that, as is the case for all biological products, the development of cell and gene therapies is a complex, challenging and cost intensive process. Outsourcing is, therefore, a preferred operational model, with cell and gene therapy innovators relying heavily on specialty contract service providers to handle various aspects of their product development and manufacturing operations. In fact, over three quarters of the biopharmaceutical companies prefer to outsource their operations to the contract service providers and a large part of their expenditure goes to outsourcing services.

Amongst the various operations, the outsourcing of analytical testing services, including environment testing, bioanalytical testing, analytical testing, chemistry and stability testing, method development and validation, and product characterization, has witnessed significant rise in recent years.

The rising demand for outsourcing bioanalytical services has prompted the emergence of several contract research organizations that claim to offer bioassay services for cell and gene therapies. These companies usually have relatively more experience and are well equipped with the instruments and technological platforms to offer both pre-developed and customized bioassays.

In fact, these companies assist in bringing out the maximum efficiency of study design by offering several cost benefits and reducing the associated timelines. Given the benefits of outsourcing the bioassay services and the ongoing efforts of service providers to further improve / expand their respective offerings, we believe that the cell and gene therapy bioassay services market is likely to evolve at a steady pace, till 2030.

Companies Mentioned

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. MARKET LANDSCAPE

5. COMPANY COMPETITIVENESS ANALYSIS

6. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN NORTH AMERICA: COMPANY PROFILES

7. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN EUROPE AND ASIA-PACIFIC: COMPANY PROFILES

8. MARKET TREND ANALYSIS

9. GLOBAL EVENT AND STRATEGIC INITIATIVE ANALYSIS

10. CASE STUDY: MARKET LANDSCAPE OF CELL AND GENE THERAPIES

11. LIKELY PARTNERS FOR CELL THERAPY BIOASSAY SERVICE PROVIDERS

12. LIKELY PARTNERS FOR GENE THERAPY BIOASSAY SERVICE PROVIDERS

13. MARKET FORECAST AND OPPORTUNITY ANALYSIS

14. CONCLUSION

15. EXECUTIVE INSIGHTS

16. APPENDIX 1: TABULATED DATA

17. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/3rvbdt

Original post:
Insights on the Cell and Gene Therapy Bioassay Services Market to 2030 - Industry Trends and Global Forecasts - ResearchAndMarkets.com - Business Wire

Mustang Bio, Inc.

WORCESTER, Mass., March 23, 2022 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the full year ended December 31, 2021.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, In 2021, we continued to make great strides in advancing our cell and gene therapy programs. Notably, the U.S. Food and Drug Administration (FDA) approved two Mustang Investigational New Drug (IND) applications, the first to initiate a pivotal multicenter Phase 2 clinical trial to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with X-linked severe combined immunodeficiency (XSCID), and the second to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-cell non-Hodgkin lymphomas (B-NHL) and chronic lymphocytic leukemia (CLL). Additionally, we received a $2 million grant from the National Cancer Institute to partially fund our MB-106 Phase 2 clinical trial. Interim data from the ongoing Phase 1/2 clinical trial of MB-106 presented at the 63rd American Society of Hematology Annual Meeting (ASH2021) by our collaborators at Fred Hutchinson Cancer Research Center (Fred Hutch) showed a 95% overall response rate, 65% complete response rate and favorable safety profile. We also expanded our product portfolio by executing an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID and enhanced our CAR T program with an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that has the potential to transform the administration of CAR T therapies and be used as an off-the-shelf therapy.

Story continues

Dr. Litchman continued, We anticipate another productive year in 2022, with several Mustang IND clinical trial initiations and data updates from our ongoing clinical programs at prominent medical conferences. In February, City of Hope presented Phase 1 data on MB-105, our prostate stem cell antigen (PSCA) CAR T-cell therapy for the treatment of PSCA-positive metastatic castration-resistant prostate cancer (mCRPC) at the 2022 American Society of Clinical Oncology (ASCO) Genitourinary (GU) Cancers Symposium that demonstrated its potential to treat patients with mCRPC. In April, our collaborators at Fred Hutch will present interim Phase 1/2 clinical trial data on MB-106 at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR) which show its compelling clinical activity and favorable safety profile. In the first half of 2022 we anticipate enrolling the first patient in Mustangs multicenter Phase 1/2 clinical trial to evaluate MB-106, and in the second half of 2022 we expect to enroll the first patient in Mustangs multicenter pivotal Phase 2 clinical trial to evaluate MB-107. We also plan to advance additional Mustang clinical candidates, including filing an IND for MB-109 (MB-101 autologous IL13R2-directed CAR T cells + MB-108 oncolytic virus) for the treatment of glioblastoma. With this steady progress across our clinical programs, and with our robust pipeline and strong cash position following completion of the debt facility with Runway Growth Capital, Mustang is poised to continue its success building an integrated cell and gene therapy company.

Financial Results:

As of December 31, 2021, Mustangs cash and cash equivalents and restricted cash totaled $110.6 million, compared to $98.8 million as of December 31, 2020, an increase of $11.8 million year-to-date.

Research and development expenses were $49.9 million for the year ended December 31, 2021. This compares to $37.2 million for 2020. Non-cash, stock-based compensation expenses included in research and development were $2.3 million for the year ended December 31, 2021, compared to $1.4 million for 2020.

Research and development expenses from license acquisitions totaled $5.8 million for the year ended December 31, 2021, compared to $10.1 million for 2020. Non-cash, stock-based compensation expenses included in research and development licenses acquired were $4.2 million for the year ended December 31, 2021, compared to $7.6 million for 2020.

General and administrative expenses were $11.0 million for the year ended December 31, 2021. This compares to $9.5 million for 2020. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.9 million for the year ended December 31, 2021, compared to $4.0 million for 2020.

Net loss attributable to common stockholders was $66.4 million, or $0.76 per share, for the year ended December 31, 2021, compared to a net loss attributable to common stockholders of $60.0 million, or $1.14 per share, for 2020.

2021 and Recent Corporate Highlights:

In February 2021, Mustang announced encouraging MB-107 and MB-207 clinical updates from its X-linked severe combined immunodeficiency (XSCID) investigator IND trials, as well as additional consistent safety and efficacy data. In the second half of 2022, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang Bios IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID, also known as bubble boy disease. Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with a hematopoietic stem cell transplantation (HSCT) and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from FDA and, based on feedback from the Agency, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.

In May 2021, Mustang announced that the FDA approved its IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-NHL and CLL. Mustang intends to dose the first patient in that trial in the first half of 2022.

Also in May 2021, Mustang announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13R2-directed CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).

In June 2021, Mustang announced that MB-106 CD20-targeted CAR T cell therapy data were presented at the European Hematology Association 2021 Virtual Congress. Dr. Mazyar Shadman of Fred Hutch presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL, which showed a favorable safety profile and compelling clinical activity, with a 93% overall response rate and 67% complete response rate in patients treated with a modified cell manufacturing process.

Also in June 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL.

Additionally in June 2021, Mustang announced that it was one of 28 recipients awarded tax incentives from the Massachusetts Life Sciences Center. The $300,000 tax incentive amount that Mustang was awarded was based on a hiring commitment of 20 net new full-time equivalent employees for calendar year 2021 and retaining that headcount level through 2025.

In August 2021, Mustang announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to MB-107, a lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants.

Also in August 2021, Mustang announced an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.

In October 2021, Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory and The Heritage Provider Network Professor in Immunotherapy at City of Hope, presented updated Phase 1 clinical data regarding MB-101 (IL13R2targeted CAR T cells) for the treatment of glioblastoma at two scientific conferences, the First Annual Conference on CNS Clinical Trials, co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology, and the American Association for Cancer Research Virtual Special Conference: Brain Cancer.

In November 2021, Mustang announced the execution of an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.

Also in November 2021, Mustang announced that it was awarded a grant of approximately $2 million from the National Cancer Institute of the National Institutes of Health. This two-year award will partially fund the Mustang-sponsored multicenter trial to assess the safety, tolerability and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL or CLL.

In December 2021, Mustang announced that MB-106 data were presented at ASH2021. Dr. Shadman of Fred Hutch presented updated interim data showing a 95% overall response rate, 65% complete response rate and favorable safety profile from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the poster can be viewed online here.

Also in December 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted, autologous CAR T cell therapy to treat B-NHL and CLL. A replay of the webinar can be found here.

Additionally in December 2021, Mustang was added to the NASDAQ Biotechnology Index.

In January 2022, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the ASTCT and CIBMTR, rescheduled to take place April 23-26, 2022, in Salt Lake City, Utah. A copy of the abstract can be viewed on the meeting website here.

In February 2022, Mustang was selected as the Bronze winner for the Central region in the Eighteenth Annual Team Massachusetts Economic Impact Awards presented by MassEcon. The award winners will be honored at Gillette Stadium on April 7, 2022.

Also in February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the 2022 American Society of Clinical Oncology Genitourinary Cancers Symposium. The data results indicated that PSCA-CAR T-cell therapy is feasible in patients with mCRPC with dose limiting toxicity (DLT) of cystitis and show preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells can proceed in the trial.

In March 2022, Mustang completed a $75 million long-term debt facility with Runway Growth Capital LLC ("Runway).

Also in March 2022, Mustang announced that an abstract reporting on Phase 1 trials being conducted at the University of Alabama at Birmingham (UAB) and City of Hope of Mustang Bios exclusively licensed oncolytic viral and CAR T-cell therapies for the treatment of patients with glioblastoma (GBM) was selected as a late-breaking poster presentation at the American Association for Cancer Research (AACR) Annual Meeting 2022, taking place April 8 13, 2022, in New Orleans, Louisiana. The abstract will also be published in the online Proceedings of the AACR.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

MUSTANG BIO, INC.Balance Sheets (in thousands, except for share and per share amounts)

December 31,

December 31,

2021

2020

ASSETS

Current Assets:

Cash and cash equivalents

$

109,618

$

97,804

Other receivables - related party

50

15

Prepaid expenses and other current assets

2,038

1,715

Total current assets

111,706

99,534

Property, plant and equipment, net

9,025

7,529

Fixed assets - construction in process

2,027

499

Restricted cash

1,000

1,000

Other assets

362

250

Operating lease right-of-use asset, net

1,050

1,088

Total Assets

$

125,170

$

109,900

LIABILITIES AND STOCKHOLDERS EQUITY

Current Liabilities:

Accounts payable and accrued expenses

$

9,744

$

8,747

Payables and accrued expenses - related party

723

490

Operating lease liabilities - short-term

348

278

Total current liabilities

10,815

9,515

Deferred income

270

Operating lease liabilities - long-term

1,685

1,950

Total Liabilities

Visit link:
Mustang Bio Reports Full-Year 2021 Financial Results and Recent Corporate Highlights - Yahoo Finance

theasis/E+ via Getty Images

You don't go to the leading edge of life sciences/bioproduction looking for bargains, as the strong growth and margins available to companies facilitating the rapid growth of new biologic treatment options like gene and cell therapies have fueled strong share price performance (and multiples) for larger, better-known companies like Danaher (DHR) and Thermo Fisher (TMO), as well as smaller players like Bio-Techne (NASDAQ:TECH).

I find a lot to like in Bio-Techne's leverage to cell and gene therapy-enabling products like GMP protein production and non-viral gene editing, as well as its leverage to life sciences/bioproduction research tools, spatial biology, and molecular diagnostics. I don't find nearly as much to like in the valuation, but stocks like these are a "you either get it, or you don't" sort of proposition where you're basically betting that the underlying growth of the market and the company's strategic decisions will eventually lead to enough revenue and profit growth down the line to redeem an eye-watering valuation today.

Through both M&A and leveraging internal manufacturing capabilities grounded in a long history of producing proteomic research reagents, Bio-Techne has built a business segment to leverage the exceptional underlying growth potential in cell and gene therapies over the coming years.

Bio-Techne's offerings include Cloudz cell activation kits that use non-magnetic beads to separate and activate cells, the TcBuster non-viral gene editing platform, and its GMP proteins, as well as complementary tools in spatial biology and immunocytochemistry. All told, management has sized its opportunity in CGT at over $3B today, growing at over 20%, and the company only has a small low-single-digit market share today.

GMP proteins are a major thematic driver right now, growing 180% in the fourth quarter and helping drive 80%-plus growth overall for the Cell and Gene Therapy (or CGT) business. GMP proteins are proteins produced in dedicated facilities and in accordance with strict quality management guidelines, ensuring biological activity, purity, and batch-to-batch consistency.

These proteins are not used directly in therapies (they're not infused or injected into patients), but they're used to produce cell and gene therapies that are practically in their infancy. As such, I think a handy shortcut for readers is to think of them as consumables used in the development and manufacture of cell and gene therapies like Novartis' (NVS) Kymriah (CAR-T) and Zolgensma (gene therapy).

Management recently opened a new GMP protein manufacturing facility and the ramp of commercial operations there should drive meaningful growth for several quarters, with the business scaling from around $30M to over $200M over the next several years. In addition to ramping up this facility, management has already started talking of broadening the business into GMP antibodies and media, as well as RNA and DNA products. As an aside, Bio-Techne tried to acquire Aldevron, a manufacturer of GMP-grade plasmid DNA, mRNA, and proteins back in 2021 but lost out to Danaher.

Bio-Techne's CGT operations are the "sizzle" today, but there's plenty of steak behind it. While the company's legacy proteomic reagents and diagnostics (calibrators and controls) operations aren't particularly sexy, they are still growing at a mid-single-digit clip and they generate good cash flow for the business.

Beyond this are interesting opportunities in analytical instrumentation, spatial biology, and molecular diagnostics.

The Analytical Solutions business is growing at a double-digit rate for Bio-Techne, with the company leveraging growing demand for automation in the lab. The Simple Western is the only automated tool for western blotting, a technique that identifies specific proteins in a sample and can require hours of hands-on time. The Maurice platform automates protein profiling while Ella, a benchtop immunoassay platform, can be used in a range of applications like monitoring cytokine storms (in CAR-T recipients or COVID-19 patients) and detecting impurities in finished biologics production runs.

Bio-Techne's Advanced Cell Diagnostics business offers RNAscope and DNAscope, and an in-situ RNA/DNA hybridization platform that detects target RNA or DNA within a single cell without disturbing the tissue morphology. This allows users to examine biomarker status, structural variation, and so on, and is useful in a range of applications from cancer diagnostics to ensuring whether reprogrammed T-cells for CAR-T are expressing the intended antigen receptors.

In molecular diagnostics, Bio-Techne hasn't seen the hoped-for adoption of its ExoDx Prostate IntelliScore liquid biopsy for prostate cancer (a rule-out test after ambiguous PSA results), but reimbursement has improved and post-pandemic normalization of office visits should drive more use. Beyond this, though, the company is building out a broader portfolio of biomarker-based screening products that can address a range of diseases/conditions, with a particular focus on genetic carrier screening and oncology diagnostics.

All told, Bio-Techne is targeting markets with combined addressable revenue of over $15 billion a year, including high-growth areas like analytical research tools, cell/gene therapy, spatial biology, liquid biopsy, and molecular diagnostics. Given that cell and gene therapies are practically in their infancy (or at least early toddlerhood), I see significant growth opportunities in tools and consumables that facilitate research and production, and I think Bio-Techne has a good starting position today.

I also doubt that the company is anywhere close to done with its M&A program. More than a decade ago, I followed Bio-Techne (then Techne) as a sell-side analyst, and the change in the company since then has been remarkable. Much of that change can be tied to a change in management and operating philosophy (actively targeting growth), but M&A has played a key enabling role, including deals for Asuragen (diagnostics kitting), B-MoGen (TcBuster gene editing), Quad Technologies (Cloudz), Exosome, CyVek (Ella) and ProteinSimple (automated equipment, including Simple Western).

Most recently, the company announced an unusual deal for Wilson Wolf, the manufacturer of G-Rex bioreactors (used to grow/produce proteins or cells like T-cells in volume). Bio-Techne isn't buying the company today but now has the option to buy 20% when the company reaches $100M in revenue and the whole company at $225M in revenue (or $135M in EBITDA), as well as another option if those targets aren't reached. No definitive terms have been reached, but at $225M in revenue, it seems likely that the deal would cost over $2B.

I'm looking for high-teens annualized revenue growth over the next decade and EBITDA margin expansion to over 42% in 2024 (as well as long-term FCF margins in the high 20%s), but those targets don't really matter. I don't think there's any way to derive a bottoms-up fair value that will look attractive today, though comparative analysis to other high-growth life sciences/bioproduction names suggests a current EV/forward revenue of around 16.5x isn't completely unprecedented (Danaher paid 17.6x forward revenue for Aldevron).

Investing in highly-valued "hyper-growth" stocks has never really been in my wheelhouse; I've done it and made money doing it, but I'm always reluctant to make "forget the valuation and buy it'll all work out eventually" recommendations to others. I do like Bio-Techne's leverage to growth in bioproduction generally and cell/gene therapy research and production more specifically, but this is definitely not a stock for investors who can't accept high risk.

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Bio-Techne - Exceptional Leverage To Biopharma Growth, And Priced Accordingly - Seeking Alpha

"Throughout his career, Rayne has demonstrated strong leadership in multiple roles across the globe, including most recently as chief manufacturing officer leading the development of our state-of-the-art manufacturing facility in Thousand Oaks," said Peter Anastasiou, president and CEO of Capsida. "Rayne's appointment to COO will ensure Capsida achieves its goal of moving our technology from the lab and to patients, and will help Capsida realize the promise of gene therapy."

"I'm honored to take on the role of COO at Capsida, a company that always puts patients first," said Mr. Waller. "Throughout my career in the biotechnology industry, I've worked to focus on the end goal of bringing therapies to patients who have no other options. As COO, I look forward to working with the Capsida leadership team and our board to bring the promise of gene therapy to life."

Gene therapy is still in its infancy and has yet to achieve its full potential. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous administration.

Prior to joining Capsida, Mr. Waller spent 27 years at Amgen in increasing roles of responsibility across manufacturing and supply-chain management. His most recent roles at Amgen included vice president and site head for Amgen's largest manufacturing site in Puerto Rico, vice president of regional manufacturing, responsible for overseeing site operations for manufacturing facilities in Ireland and The Netherlands, and vice president of global supply-chain management with responsibility for contract manufacturing, global supply management, operations strategic planning, and risk management.

Mr. Waller holds a bachelor's degree in business administration from the University of Arizona.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of rare and more common genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us atwww.capsida.comto learn more.

SOURCE Capsida Biotherapeutics

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Capsida Biotherapeutics Announces Appointment of Rayne Waller to Chief Operating Officer - PR Newswire

Wet age-related macular degeneration (wAMD) has recently witnessed a series of approvals thanks to a degree of product differentiation, and this has drawn enthusiasm from multiple stakeholders in the field. This interest is expected to be widened with the anticipated approvals of more products, particularly biosimilars. Despite this, a lack of treatment options for other sub-types of the disease means that opportunities are still available for pharmaceutical companies to make a difference to patients affected by AMD.

Product differentiation has been a key strategy employed by companies whose products have recently received approvals for wAMD. Consider for example Roches Susvimo (ranibizumab), which received US Food and Drug Administration (FDA) approval in October last year. Patients implanted with Susvimo are expected to visit their doctor only twice yearly to have the implant refilled so that the medicine can be delivered to their eyes continuously. That represents a significant alleviation of treatment burden when compared with the current standards of care such as Lucentis (ranibizumab), Eylea (aflibercept) and Beovu (brolucizumab), which need to be administered more frequently.

Vabysmo (faricimab), also from Roche, received FDA approval in January this year for wAMD and for diabetic macular oedema. This therapy is administered every four weeks for the first four doses, following which, depending on the results of optical coherence tomography and visual acuity evaluations, there is potential for the frequency of administration to be reduced to as few as twice a year, resulting in a lower cost of treatment compared to standard of care. While it remains to be seen to what extent these new therapies will be commercially successful, being associated with lesser frequency of administration already makes such therapies an attractive option for patients and physicians in this field.

Looking ahead, anticipated launches of new biosimilars and innovator molecules are expected to widen interest in the field of AMD. According to GlobalDatas Pharmaceutical Intelligence Centre (PIC), there are currently 15 products in late-stage development exclusively for wAMD in the seven major markets (7MM: the US, France, Germany, Italy, Spain, UK and Japan). Five of these are aflibercept biosimilars and four are ranibizumab biosimilars. While both groups of biosimilars are expected to directly target patient shares from their branded counterparts, Eylea (aflibercept) and Lucentis (ranibizumab), the latter two also face threats from innovator molecules including RGX-314 from RegenxBio and AbbVie. This is a gene therapy that has the potential to be used as a one-time treatment option for patients with wAMD and, if approved, has the potential to disrupt current market dynamics for this sub-type of AMD.

In addition, there are gaps that exist in the market, particularly for therapies that can help patients with dry AMD and geographic atrophy, for which there are currently no approved therapies available. According to GlobalDatas PIC, there are three therapies in late-stage development for geographic atrophy: Alkeus Pharmaceuticals ALK-001, Apellis Pharmaceuticals pegcetacoplan, and IVERIC bios avacincaptad pegol sodium. Dobecure SLs therapy ethamsylate is also in development for both dry AMD (dAMD) and wAMD. It remains to be seen, however, how effective these therapies will be in addressing the disease at an earlier stage, as it is crucial to treat these diseases before they progress further.

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Product approvals and unmet treatment needs expected to widen interest in AMD - Pharmaceutical Technology

LONDON--(BUSINESS WIRE)--Ixaka Ltd, an integrated cell and gene therapy company, today announces an expansion of its IP portfolio to allow a broad range of applications for its polymeric targeted nanoparticle (TNP) gene delivery platform across multiple therapeutic areas. The extended IP enables the development of therapies encapsulating any cargo including mRNA, plasmids and adenovirus associated virus (AAV), and gene editing technologies as well as lentiviral vector-based therapies.

Ixakas TNP composed of proprietary polymer capsule (OM-PBAE polymers (oligopeptide end-modified poly -amino-ester polymers)) directed to specific cells through targeting agents attached the capsule (an aptamer-based targeting moiety) and cell targeted efficient transduction (use of lentiviral vector cell specific promoter). This targeting enables the targeted nanoparticles to be directed to specific cells allowing beneficial gene transduction to occur directly within a patients body. The technology is currently being applied as a gene delivery platform to generate CAR T-cell therapies in vivo for haematological malignancies, with the potential for improved efficacy and safety compared to similar products currently marketed or in development. The expanded IP allows for a broad range of cargos to be encapsulated providing greater flexibility to engineer new therapies which are optimized to specific diseases.

A new agreement extends the use of its OM-PBAE polymers for encapsulation of a broad range of cargos for use in any therapeutic area including mRNA, plasmids, and any other vectors in addition to lentiviral vectors and AAV.

The extension of the license agreement opens the door to expansion of Ixakas TNP platform, enabling the use of OM-PBAE polymers capsules to be used for delivery of numerous cargos with the choice of genetic modification technology tailored for each disease. Potential applications include drug delivery for oligonucleotides such as DNA, RNA and siRNA, plasmids, small molecules, and gene editing using tools such as CRISPRCas9, zinc finger or megaTALS enabling Ixaka to broaden its therapeutic pipeline.

Joe Dupere, CEO at Ixaka, commented:

This IP agreement further strengthens Ixakas rapidly growing IP portfolio, highlighting the pioneering nature and broad potential of our targeted nanoparticle technology. The platform is already showing great potential in generating CAR-T cells in vivo for CD19 blood cancers. With an array of other possible applications, we will now be seeking collaborations for our future pipeline, which could encompass solid tumours, rare genetic disorders, autoimmune diseases, broader immunotherapy applications, gene editing, immunodiagnostics and vaccines.

IP portfolio overview

Building a robust and broad IP portfolio is at the heart of Ixakas development strategy. The Companys ongoing R&D and upcoming clinical data will allow filing of additional patent applications across multiple territories.

Ixakas current IP portfolio contains 1 patent family for its multi-cell therapy platform and 11 patent families for its TNP platform, covering all key components of the technology, including a proprietary polymer, bald engineered lentiviral vector, T-cell specific promoter and aptamer-based targeting agent. The portfolio also provides protection across a wide geographic range (including Europe, the US, Canada, Mexico, Brazil, China, India, Korea, Japan, Australia).

The patent is for products developed under a licence agreement between Ixaka (previously aratinga.bio), Sagetis Biotech (Sagetis) and universities (Institut Quimic de Sarria CETS Fundacio Privada and Institut dInvestigacions Biomediques).

About Ixaka

Ixaka is a cell and gene therapy company focused on using the natural powers of the body to cure disease.

Ixakas proprietary technologies enhance the naturally therapeutic power of cells by increasing the presence of curative cells at the site of disease, or by directly modifying cells within the body to improve disease targeting and boost their restorative effect.

Ixakas technologies concentrated multi-cell therapies and nanoparticle therapeutics demonstrate potential for the treatment of a broad range of serious diseases across oncology, cardiovascular, neurological and ocular diseases, and genetic disorders.

Ixaka has offices in London, UK with R&D and manufacturing operations in Seville, Spain and Paris, France and additional manufacturing capability in Frankfurt, Germany.

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

Connect with us: Twitter: https://twitter.com/Ixaka_Ltd ; LinkedIn: https://www.linkedin.com/company/ixaka-limited

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Ixaka Expands IP Portfolio to Enable Use of Targeted Nanoparticle in vivo Gene Delivery Technology With Any Cargo in Any Therapeutic Area - Business...

Today, you could estimate that one family per day is being treated with and impacted by gene therapy. We want to see that increase to 10s, 100s, 1,000s per day, and reaching that goal comes from investing in research, clinical trials and manufacturing, said Ken Mills, CEO of clinical stage US biotechnology company, Regenxbio.

Regenxbio, said its CEO, has played a pivotal role in the gene therapy industry since its founding in 2008, as a result of research from the lab of gene therapy pioneer, Jim Wilson, University of Pennsylvania.

The company's NAV technology platform consists of over 100 novel adeno-associated virus (AAV) vectors, one of which was used in the US Food and Drug Administration (FDA) approved gene therapy, ZOLGENSMA, for spinal muscular atrophy in children under two years old.

The developer has also licensed out its technology to a growing list of partners and licensees that includes Novartis, Eli Lily and Pfizer and has a robust in-house pipeline of candidates for retinal and rare diseases.

Looking at the US gene therapy regulatory landscape, what are the current roadblocks?

AAV-mediated gene therapies offer the possibility of a one-time administration that could address the underlying disease and change the way critical medicine is delivered to patients, but the regulatory landscape has not evolved as quickly as the innovations of AAV gene therapy.

Weve seen that the FDA is open to working with industry and gene therapy stakeholders to determine the appropriate path forward, to streamline clinical development and get medicines to patients faster. Leveraging accelerated approvals and surrogate endpoints in clinical trials, such as a biomarker, may play a large role, Mills told BioPharma-Reporter.

The Pathway Development Consortium (PDC) launched in 2021 by Regenxbio and Solid Biosciences is working to advance opportunities to leverage the FDAs accelerated approval pathway for gene therapy candidates.

Our mission to bring together patients, industry, regulators, academia, payers and other stakeholders for meaningful scientific and policy discussion, said the CEO.

There is a significant strain on manufacturing capabilities in the gene therapy sector both capacity and reproducibility, but more importantly, talent, commented Mills.

As the sector has grown rapidly and expanded broadly, we have seen these rate-limiting factors continue to persist. In addition, significant strain on supply chains is likely to continue into 2022 and will impact pharma and biotech. Consistent, reliable manufacturing is critical to gene therapy trial development, product approval, and commercialization, so it is crucial to overcome these capabilities challenges.

Contract development and manufacturing organizations (CDMOs) are critical partners, said the executive, and he anticipates continued investment in manufacturing capabilities through in-house facilities and CDMOs next year.

And as the field continues to advance, we will start to see more and more efficiencies that companies like Regenxbio can capitalize upon to allow for rapid manufacturing and formulation development.

Regenxbio has invested in the establishment of a robust suspension cell culture-based manufacturing process and new manufacturing facility at its headquarters in Rockville, Maryland.

We have also invested to ensure the hiring of the right people to make this possible. Five to 10 years ago, you did not see a lot of process development teams, and now they are crucial to drive the scalability of capabilities across clinical and commercial strategy.

Through the expansion of its expert manufacturing team and facility build out, he said the companys researchers and process development team have been able to work side by side to mitigate potential issues early in the development process.

The goal is always to get therapies approved and to patients as quickly as possible, and a reliable, scalable chemistry, manufacturing and controls (CMC) process is crucial in accomplishing this, said Mills.

Our philosophy initially was to develop the best process platform that could be utilized across multiple programs with a highly similar process that could be easily transferred to a CDMO. We also have a platform downstream process developed that works across our programs, giving consistent downstream yields that are appropriate for the current phase of development.

We have developed proprietary formulations that are indication-specific. The formulations are stable at the intended storage conditions over several years and we have ongoing monitoring of product quality during that period to ensure consistent performance.

In terms of the highlights for the biotech this year, Mills said it was a fast paced, high-achieving 12 months for the company.

In September, we announced a partnership with AbbVie to develop and commercialize RGX-314, our gene therapy for the treatment of wet AMD, diabetic retinopathy, and other chronic retinal diseases. Under the terms of the agreement, AbbVie will provide Regenxbio a US$370m upfront payment with the potential for the company to receive up to US$1.38bn in additional development, regulatory and commercial milestones.

We are currently running a pivotal program of RGX-314 for the treatment of wet AMD, and we expect to file a BLA in 2024. We are also conducting additional trials evaluating RGX-314 delivered directly to the suprachoroidal space of the eye for the treatment of wet AMD and diabetic retinopathy. In 2021, we reported initial data from both of those trials.

Regenxbio also announced, early in 2021, a new pipeline candidate for treating Duchenne muscular dystrophy (Duchenne) - RGX-202. That is designed, said Mills, to deliver an optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne.

We received Orphan Drug Designation from the FDA in November and shared that we expect to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021.

Commercial-scale cGMP material has already been produced at 1,000 liter capacity using our suspension cell culture manufacturing process, and the company's internal cGMP facility is expected to allow for production up to 2,000 liters for the clinical development of RGX-202.

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A look at prospects for the US gene therapy industry - BioPharma-Reporter.com

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been added to the ICE Biotechnology Index (NYSE:ICEBIO) in accordance with the annual reconstitution of the index, effective prior to the U.S. market open on Monday, December 20, 2021.

Tayshas inclusion in this key biotechnology index provides important validation of our platform and value proposition as a company, said RA Session II, President, Founder and CEO of Taysha. We remain focused on executing our near-term clinical and regulatory milestones, which we believe will continue to increase our visibility within the investment community.

The ICE Biotechnology Index tracks the performance of qualifying U.S.-listed biotechnology companies classified within the Biotechnology Sub-Industry Group of the ICE Uniform Sector Classification schema, which is a multi-asset class industry classification taxonomy developed by ICE. The index includes companies that are engaged in the research and development of therapeutic treatments but are not focused on the commercialization and mass production of pharmaceutical drugs. The index also includes companies that are engaged in the production of tools or systems that enable biotechnology processes.

About Taysha Gene Therapies

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

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Taysha Gene Therapies Added to the ICE Biotechnology Index - Business Wire