Biogen : Gene Therapy

Biogen Gene Therapy

Junghae Suh, Ph.D., Head of Gene Therapy Accelerator Unit (GTxAU)

R&D Day

September 21, 2021

Forward-looking statements

This presentation contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to: our strategy and plans; potential of, and expectations for, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development

programs, clinical trials, and data readouts and presentations; risks and uncertainties associated with drug development and commercialization; regulatory discussions, submissions, filings, and approvals and the timing thereof; the potential benefits, safety, and efficacy of our and our collaboration partners' products and investigational therapies; the anticipated benefits and potential of investments, collaborations, and business development activities; and our future financial and operating results. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "potential," "possible," "prospect," "will," "would," and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development

programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our dependence on sales from our products; uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; failure to compete effectively due to significant product competition in the markets for our products; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives;

difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; our dependence on collaborators, joint venture partners, and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential future healthcare reforms; failure to obtain, protect, and enforce our data, intellectual property, and other proprietary rights and the risks and uncertainties relating to intellectual property claims and challenges; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; risks relating to the distribution and sale by third parties of counterfeit or unfit versions of our products; risks relating to the use of social media for our business; risks relating to technology failures or breaches; risks relating to management and key personnel changes, including attracting and retaining key personnel; failure to comply with legal and regulatory requirements; the risks of doing business internationally, including currency exchange rate fluctuations; risks relating to investment in our manufacturing capacity; problems with our manufacturing processes; fluctuations in our effective tax rate; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; fluctuations in our operating results; risks related to investment in properties; the market, interest, and credit risks associated with our investment portfolio; risks relating to share repurchase programs; risks relating to access to capital and credit markets; risks related to indebtedness; change in control provisions in certain of our collaboration agreements; environmental risks; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission (SEC).

These statements are based on our current beliefs and expectations and speak only as of the date of this presentation. We do not undertake any obligation to publicly update any forward-looking statements.

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Key messages

We are building Biogen's

Gene Therapy (GTx) R&D Engine to drive innovation and to deliver a continuous pipeline of gene therapies

Gene Therapy Accelerator Unit (GTxAU)

External innovation		Biogen's internal gene
is an important part of		therapy manufacturing
our strategy		facility is underway

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Building Biogen's gene therapy (GTx) R&D engine with aim of delivering both near- and long-term assets with greater probability of success

	Technology Innovation		Bidirectional		Portfolio Delivery
					alignment
	Focused technology innovation to				Focused system innovation to move
	increase probability of success of GTx				GTx programs through pipeline
	GTx Pillars		Capsid		Cargo		Vector		Vector
				Performance		Production
	Tech	•	Broad CNS, intravitreal,	•	Regulated/inducible	•	Routes of	•	Increase overall yield
		muscle-tropic capsids		promoters		administration (ROA)	•	Increase scalability
	Innovation*
	•	Higher potency capsids	•	Gene editing tools	•	Immunogenicity, safety
	Portfolio	•	Capsid identification	•	Cargo optimization	•	In vitro and in vivo cores	•	Small-/mid-scale
	•	Structure-activity	•	Human genetic	•	Bioanalytics, PK/PD		production
	Delivery
	•	Process development
			mapping		evidence bioinformatics		modeling
						•	In vivo imaging	•	Analytical development
	*Goals and needs of gene therapy programs			CNS = central nervous system; PK/PD = pharmacokinetics/pharmacodynamics

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Gene Therapy Accelerator Unit (GTxAU)

Identify and solve key rate-limiting steps in our technologies, capabilities, and drug discovery and development processes to accelerate gene therapy programs through the pipeline.

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