As Long As We Are Having Fun and Speculating This Weekend

Speaker at ISBiotech, March 6-8, 2017 : (http://www.isbiotech.org/pdfs/Spring2017PAAG.pdf)

Martin A. Giedlin, PhD

Executive Director, Process Development Novartis Pharmaceuticals Corporation

Bench to Commercial-Scale Process Development for a CAR T-Cell Therapy Abstract Autologous gene-modified adoptive cell therapy is a rapidly emerging option for treatment of refractory/relapsing leukemias and lymphomas. Numerous clinical trials have demonstrated that the use of chimeric antigen receptors (CARs) or modified T-cell receptors (TcRs) targeting these malignancies have a remarkable ability to traffic, expand, and kill target tumor cells post-infusion. However, there are a number of challenges to overcome in order to ensure the safety and efficacy of these cellular-based immunotherapies in a commercial setting. Characterization and control of patient leukapheresis starting material, vector for delivering the target gene, optimal activation/expansion conditions, and in some cases, cryopreservation process and stability, all need to be defined and controlled. This presentation will review an approach utilized to progress these therapeutics from academia to commercial manufacturing and highlight the roles of process development, characterization, and validation, specifically, within the context of a CD19 targeted CAR T immunotherapy. Biography Now look at his former employer: Sangamo Biosciences http://podcasts.ibclifesciences.com/CellTherapy/2014/IBC_B14188_Giedlin.pdf

Sangamo Gene Therapy Study for Hemophilia A now Live on ClinicalTrials.gov

SB-525 for Hemophilia A

Sangamo has developed an adeno-associated virus (AAV) carrying a clotting Factor 8 gene construct driven by Sangamo's proprietary synthetic liver specific promoter, which in preclinical studies is more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A. The Company’s investigational new drug (IND) application for this program has been cleared by the U.S. Federal Drug Administration (FDA) enabling the company to begin a Phase 1/2 clinical trial in adults with severe hemophilia A.

https://clinicaltrials.gov/ct2/show/NCT03061201?term=sangamo&rank=4

Enhancing Deal Making Staff - Legal

Sangamo Therapeutics, Inc.
JOB DESCRIPTION

Position: Associate Counsel

Reporting to: Sr. Director of Legal

Position Summary

The Corporate Development Team at Sangamo Therapeutics, Inc. is looking for a highly motivated and experienced transactional attorney to join its legal group.  The Associate Counsel will be working cross-functionally with various departments to drive legal strategy and to provide legal support in the areas of procurement, research and development, clinical operations, and manufacturing, among others.  The Associate Counsel’s primary responsibility will be to negotiate and draft commercial and non-commercial contracts to support Sangamo’s research, clinical and commercial operations but will also get exposure to a broad range of other legal issues faced by the biopharmaceutical industry, and more specifically in the field of gene therapy.    

Primary responsibilities 

Include the following. Other duties may be assigned.

• Negotiating and drafting various commercial, non-commercial and government contracts (e.g. manufacturing, services, collaboration/partnership, licensing, commercialization, confidential disclosure, material transfer, sponsored research, clinical trial, letter of indemnification, grant application/award, sub-award, etc.).
• Updating and maintaining library of template contracts.
• Assisting with ongoing process improvements for contracts management and approval process.
• Communicating, educating, and providing training to clients on various legal issues, policies and processes, including internal contracts approval processes, SOX compliance, and monitoring compliance of the same.
• Driving legal strategy in the course of advising and supporting clients to meet strategic business goals.     
• Advising on clinical regulatory compliance issues (e.g. FDA regulations, informed consent forms, protocols, reporting obligations, etc.).
• Managing outside counsel.
• Serving as a legal representative on various review committees as needed.

Requirements:

• Juris Doctorate from a top accredited law school.
• Current bar admission in at least one jurisdiction; Admission in California preferred.
• Minimum of eight years of transactional experience; In-house or law firm experience preferred. 
• Experience in biotech and/or pharmaceutical industry, including experience in FDA regulatory compliance, clinical development, and GMP manufacturing. 
• Ability to draft and negotiate contracts independently with minimum supervision.
• Ability to prioritize and manage tasks efficiently, consistently and accurately in a high volume, fast paced environment.
• Ability to exercise good judgement and to provide sound legal advice under pressure.
• Ability to work in cross-functional teams.
• Proficient in MS Office applications (Excel, Word, PowerPoint, Outlook).

 

 

Chemical & Engineering News - Sangamo Mention

CRISPR’s breakthrough problem

If the CRISPR gene editing system is to live up to its disease-curing potential, researchers must devise a plan to deliver it into the body

http://cen.acs.org/articles/95/i7/CRISPRs-breakthrough-problem.html

(note aslo includes discussion of LNP delivery)

snip

Sangamo is using an older gene-editing tool called zinc finger nucleases, a complex protein structure designed to bind and cleave a specific region of DNA. And doctors at the Great Ormond Street Hospital in London recently reported using a similar gene-editing technique called TALENs, which also recognizes and cuts precise DNA sequences, to engineer immune cells for a therapy that may have cured two infants of leukemia.

Both technologies have been around for longer than CRISPR has, with zinc-finger-based editing being in the works for more than two decades. They also both suffer from a limitation that has inhibited their widespread adoption: Each is a cumbersome protein complex that needs to be individually engineered for every new DNA target.

CRISPR, meanwhile, is easily adaptable. The Cas9 cutting protein remains the same for all applications, and to make a new edit, researchers need only to switch out the guide RNA. If the DNA sequence that needs editing is known, securing the complementary guide RNA is as easy as clicking “Order” from a supplier.

“When CRISPR came along, everyone knew what to do with it,” Intellia’s Barnes says. “People had been going around in a go-kart and you gave them a Ferrari, so away they go.”

...
CRISPR isn’t the first gene-editing technology promising to cure thousands of diseases. In fact, multiple studies of treatments developed using older technologies are now under way. Drug delivery guru Daniel Anderson of Massachusetts Institute of Technology points out that one of the most advanced programs is Sangamo Biosciences’ ongoing clinical trial to remove T cells from patients, edit their DNA to make them resistant to HIV, and reinject the modified cells. “So presumably, there are some genome-edited people walking around in California that they helped create,” Anderson says.

Although researchers are excited about the potential to use CRISPR to create therapies from people’s own blood, immune, and stem cells, thousands more genetic conditions affect everything else. For those disorders, CRISPR needs to be delivered like more traditional medicines so it can work its wonders editing DNA inside the body. But the challenge of shuttling CRISPR directly to the diseased tissue, or “in vivo” gene editing, is so daunting it could stall CRISPR’s otherwise rapid advancement

Expect Increased Partnership/Licensing Activity

Title:  Sr. Director / Head of Business Development

Manager: SVP & Chief Business Officer

Department:  Corporate Development Team

 

JOB SUMMARY:   

 

Sangamo Therapeutics is seeking a senior business development leader to play a key role in partnering, in-licensing and other collaboration activities.  This position will be the functional head of business development and will work closely with the SVP & Chief Business Officer to define the business development & licensing strategy to execute on the corporate objectives.  The person will be directly responsible for sourcing, evaluating, and negotiating a large number of strategic partnerships across biopharma, contract organizations, and academia.

 

 

ESSENTIAL FUNCTIONS: 

 

• Work closely with CBO, Executive Team and R&D Leadership to establish strategic partnering objectives associated with business development and licensing activities.
• Establish strong working relationship with scientific team and functional leads, e.g. Program Management, CMC/TechOps, Clinical, Commercial, Intellectual Property, and Legal.
• Evaluates, structures, and negotiates both buy-side and sell-side transactions across technology, manufacturing, delivery, and therapeutic programs.
• Manages and mentors other team members on best-in-class business development processes and trains deal team members.
• Drives enterprise value by thoughtfully structuring and negotiating deal terms including scope, exclusivity, economics, legal and intellectual property considerations to protect Sangamo core technology platforms, while accelerating products to patients.
• Leads cross-functional business development workstreams and builds strong cross-functional teams, always ensuring ownership and accountability for operational execution.
• Analyzes internal business development processes and makes recommendations for process improvement to drive efficiencies and better deal execution and management.

 

EDUCATION, EXPERIENCE AND SKILLS REQUIREMENTS:

 

• Minimum of 10-years of biopharma industry experience with >7 years of direct business development and negotiation experience
• Proven ability to drive business development partnerships through deal evaluation to execution and alliance management
• Strategic thinker with good quantitative analytical abilities
• Strong business and financial acumen
• Formal operational business and financial training required; JD or MBA is a plus
• Ability to understand and simplify complex scientific concepts into core business and legal objectives
• Track record of advancement and promotion in organizations
• Excellent communication, relationship building, and calm negotiation skills
• Knowledge of biopharma product development and commercialization experience; rare disease and cell & gene therapy focus is a plus
• Brings strong integrity, intelligence and energy to the company and the role

 

 

The above reflects management’s definition of essential functions for this position, but does not restrict the tasks that may be assigned.The above duties are representative only; management may assign or reassign duties and responsibilities to this position at any time.

Expect Sangamo to Add International Clinical Trial Sites

Sangamo Therapeutics, Inc.

JOB DESCRIPTION

 

Title:  Operations Specialist III

Manager:  Associate Director, TechOps Project Management

Department:  TechOps Project Management

 

JOB SUMMARY:   

The Operations Specialist III will interface with internal resources/stakeholders and external vendors/contractors to develop, implement and manage clinical supply logistics processes and strategies in support of the company’s gene therapy and genome editing products.  Primary focus will be related to planning and logistics for products that are manufactured, stored and distributed by Contract Manufacturing Organizations (CMOs) with clinical studies executed in both the US and Europe.  Role will liaise with internal resources within Clinical Operations, Technical Operations and Finance in addition to CMO representatives and logistics vendors.

 

ESSENTIAL FUNCTIONS: 

 

1. Responsible for GMP inventory management including record keeping for all inventory generation, requests (both GMP use and non-GMP use), movement and disposition as required.
2. Works with vendors to ensure accurate counts (physical inventory vs. virtual inventory records).
3. Manages proposals, contracts, PO generation and invoicing approvals for storage and clinical kitting / distribution vendors.
4. Understands clinical trial protocol requirements and translates into logistics strategy that accounts for study design, clinical site requirements and geographic location.
5. Identifies risks and assumptions in plans, anticipates problems and plans for contingencies. Removes obstacles to move work forward and/or to get efforts back on track.
6. Provides input to and reviews vendor generated clinical distribution procedures to ensure compliance with study requirements.
7. Maintains current understanding of US import/export regulations, reviews upcoming or potential regulation changes and proposals, and supports awareness of these changes across applicable functions within Sangamo and applicable third parties.

 

 

EDUCATION, EXPERIENCE AND SKILLS REQUIREMENTS:

The ideal candidate for this position will have demonstrated in-depth experience of clinical inventory management and study logistics.

Candidate must possess:

• Minimum of a Bachelor's degree in Business/Operations Management, or Chemistry, Pharmacy or equivalent life science field
• Minimum of 3 years related experience in the biotechnology or pharma industry, ideally for Advanced-therapy Medicinal Products (ATMPs) including cell and gene-based therapies
• Minimum of 5 years’ experience in commercial or clinical supply logistics
• Knowledge/experience with trade and customs regulations and global import export requirements
• Demonstrated skills in project management and working with vendors
• Proven ability to effectively develop, communicate, and gain support for execution plans with a wide range of stakeholders
• Experience with sourcing, contract distribution, supplier management and supply chain management is highly desirable
• Strong background in FDA, ISO, EMA, GMP and ICH requirements ideally for ATMP's a plus
• Advanced knowledge of drug development process, experience with cell-based, gene-based and rAAV-based therapies a plus

 

OTHER REQUIREMENTS:

• Excellent interpersonal skills
• Outstanding organizational skills
• Excellent written and oral communication skills
• Strong focus on customer service
• Ability to work in a fast paced and dynamic environment that will require management of several competing priorities while driving all projects forward and meeting program/project deliverables

The successful candidate will enjoy a competitive base salary and the opportunity to participate in incentive compensation programs, including bonus and stock option plans. Sangamo offers a comprehensive benefits program, including:  medical, dental and vision care; paid vacation and holiday time; access to a voluntary 401(k) and Employee Stock Purchase Plan. Sangamo is an equal opportunity employer.

Please send your resume/CV and a cover letter that specifically addresses this job posting as email attachments to: jobs@sangamo.com

The above reflects management’s definition of essential functions for this position, but does not restrict the tasks that may be assigned.The above duties are representative only; management may assign or reassign duties and responsibilities to this position at any time.